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| first in human study: Exploring Novel Clinical Trial Designs for Gene-Based Therapies National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, Forum on Regenerative Medicine, 2020-08-27 Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop. |
| first in human study: The Prevention and Treatment of Missing Data in Clinical Trials National Research Council, Division of Behavioral and Social Sciences and Education, Committee on National Statistics, Panel on Handling Missing Data in Clinical Trials, 2010-12-21 Randomized clinical trials are the primary tool for evaluating new medical interventions. Randomization provides for a fair comparison between treatment and control groups, balancing out, on average, distributions of known and unknown factors among the participants. Unfortunately, these studies often lack a substantial percentage of data. This missing data reduces the benefit provided by the randomization and introduces potential biases in the comparison of the treatment groups. Missing data can arise for a variety of reasons, including the inability or unwillingness of participants to meet appointments for evaluation. And in some studies, some or all of data collection ceases when participants discontinue study treatment. Existing guidelines for the design and conduct of clinical trials, and the analysis of the resulting data, provide only limited advice on how to handle missing data. Thus, approaches to the analysis of data with an appreciable amount of missing values tend to be ad hoc and variable. The Prevention and Treatment of Missing Data in Clinical Trials concludes that a more principled approach to design and analysis in the presence of missing data is both needed and possible. Such an approach needs to focus on two critical elements: (1) careful design and conduct to limit the amount and impact of missing data and (2) analysis that makes full use of information on all randomized participants and is based on careful attention to the assumptions about the nature of the missing data underlying estimates of treatment effects. In addition to the highest priority recommendations, the book offers more detailed recommendations on the conduct of clinical trials and techniques for analysis of trial data. |
| first in human study: Clinical Pharmacology: Current Topics and Case Studies Markus Müller, 2011-02-04 Today we witness an eventful time in which the powerful new forces of genomics, information technology and economics are rapidly changing the science and art of medicine. This will require more specialization than ever before. However, there is also an increasing demand for an integrated approach, which is provided by the discipline of Clinical Pharmacology (CP). CP pursues a scientific goal by studying drug action in patients and volunteers, a clinical goal by administering appropriate drug therapy and a regulatory goal by assessing the risk/benefit ratio of drug candidates in drug development and reimbursement. This introduction to current topics of CP covers traditional topics of clinical drug research and trial methodology but also provides insight in current topics like genomics, imaging technology and issues in drug reimbursement. A number of concrete case studies in clinical drug research and development help to give a better understanding of the general principles of CP. |
| first in human study: Small Clinical Trials Institute of Medicine, Board on Health Sciences Policy, Committee on Strategies for Small-Number-Participant Clinical Research Trials, 2001-01-01 Clinical trials are used to elucidate the most appropriate preventive, diagnostic, or treatment options for individuals with a given medical condition. Perhaps the most essential feature of a clinical trial is that it aims to use results based on a limited sample of research participants to see if the intervention is safe and effective or if it is comparable to a comparison treatment. Sample size is a crucial component of any clinical trial. A trial with a small number of research participants is more prone to variability and carries a considerable risk of failing to demonstrate the effectiveness of a given intervention when one really is present. This may occur in phase I (safety and pharmacologic profiles), II (pilot efficacy evaluation), and III (extensive assessment of safety and efficacy) trials. Although phase I and II studies may have smaller sample sizes, they usually have adequate statistical power, which is the committee's definition of a large trial. Sometimes a trial with eight participants may have adequate statistical power, statistical power being the probability of rejecting the null hypothesis when the hypothesis is false. Small Clinical Trials assesses the current methodologies and the appropriate situations for the conduct of clinical trials with small sample sizes. This report assesses the published literature on various strategies such as (1) meta-analysis to combine disparate information from several studies including Bayesian techniques as in the confidence profile method and (2) other alternatives such as assessing therapeutic results in a single treated population (e.g., astronauts) by sequentially measuring whether the intervention is falling above or below a preestablished probability outcome range and meeting predesigned specifications as opposed to incremental improvement. |
| first in human study: Sharing Clinical Trial Data Institute of Medicine, Board on Health Sciences Policy, Committee on Strategies for Responsible Sharing of Clinical Trial Data, 2015-04-20 Data sharing can accelerate new discoveries by avoiding duplicative trials, stimulating new ideas for research, and enabling the maximal scientific knowledge and benefits to be gained from the efforts of clinical trial participants and investigators. At the same time, sharing clinical trial data presents risks, burdens, and challenges. These include the need to protect the privacy and honor the consent of clinical trial participants; safeguard the legitimate economic interests of sponsors; and guard against invalid secondary analyses, which could undermine trust in clinical trials or otherwise harm public health. Sharing Clinical Trial Data presents activities and strategies for the responsible sharing of clinical trial data. With the goal of increasing scientific knowledge to lead to better therapies for patients, this book identifies guiding principles and makes recommendations to maximize the benefits and minimize risks. This report offers guidance on the types of clinical trial data available at different points in the process, the points in the process at which each type of data should be shared, methods for sharing data, what groups should have access to data, and future knowledge and infrastructure needs. Responsible sharing of clinical trial data will allow other investigators to replicate published findings and carry out additional analyses, strengthen the evidence base for regulatory and clinical decisions, and increase the scientific knowledge gained from investments by the funders of clinical trials. The recommendations of Sharing Clinical Trial Data will be useful both now and well into the future as improved sharing of data leads to a stronger evidence base for treatment. This book will be of interest to stakeholders across the spectrum of research-from funders, to researchers, to journals, to physicians, and ultimately, to patients. |
| first in human study: Early Drug Development Mitchell N. Cayen, 2011-02-25 The focus of early drug development has been the submission of an Investigational New Drug application to regulatory agencies. Early Drug Development: Strategies and Routes to First-in-Human Trials guides drug development organizations in preparing and submitting an Investigational New Drug (IND) application. By explaining the nuts and bolts of preclinical development activities and their interplay in effectively identifying successful clinical candidates, the book helps pharmaceutical scientists determine what types of discovery and preclinical research studies are needed in order to support a submission to regulatory agencies. |
| first in human study: A Comprehensive Guide to Toxicology in Preclinical Drug Development Ali S. Faqi, 2012-10-18 A Comprehensive Guide to Toxicology in Preclinical Drug Development is a resource for toxicologists in industry and regulatory settings, as well as directors working in contract resource organizations, who need a thorough understanding of the drug development process. Incorporating real-life case studies and examples, the book is a practical guide that outlines day-to-day activities and experiences in preclinical toxicology. This multi-contributed reference provides a detailed picture of the complex and highly interrelated activities of preclinical toxicology in both small molecules and biologics. The book discusses discovery toxicology and the international guidelines for safety evaluation, and presents traditional and nontraditional toxicology models. Chapters cover development of vaccines, oncology drugs, botanic drugs, monoclonal antibodies, and more, as well as study development and personnel, the role of imaging in preclinical evaluation, and supporting materials for IND applications. By incorporating the latest research in this area and featuring practical scenarios, this reference is a complete and actionable guide to all aspects of preclinical drug testing. - Chapters written by world-renowned contributors who are experts in their fields - Includes the latest research in preclinical drug testing and international guidelines - Covers preclinical toxicology in small molecules and biologics in one single source |
| first in human study: Improving and Accelerating Therapeutic Development for Nervous System Disorders Institute of Medicine, Board on Health Sciences Policy, Forum on Neuroscience and Nervous System Disorders, 2014-02-06 Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials. |
| first in human study: To Err Is Human Institute of Medicine, Committee on Quality of Health Care in America, 2000-03-01 Experts estimate that as many as 98,000 people die in any given year from medical errors that occur in hospitals. That's more than die from motor vehicle accidents, breast cancer, or AIDSâ€three causes that receive far more public attention. Indeed, more people die annually from medication errors than from workplace injuries. Add the financial cost to the human tragedy, and medical error easily rises to the top ranks of urgent, widespread public problems. To Err Is Human breaks the silence that has surrounded medical errors and their consequenceâ€but not by pointing fingers at caring health care professionals who make honest mistakes. After all, to err is human. Instead, this book sets forth a national agendaâ€with state and local implicationsâ€for reducing medical errors and improving patient safety through the design of a safer health system. This volume reveals the often startling statistics of medical error and the disparity between the incidence of error and public perception of it, given many patients' expectations that the medical profession always performs perfectly. A careful examination is made of how the surrounding forces of legislation, regulation, and market activity influence the quality of care provided by health care organizations and then looks at their handling of medical mistakes. Using a detailed case study, the book reviews the current understanding of why these mistakes happen. A key theme is that legitimate liability concerns discourage reporting of errorsâ€which begs the question, How can we learn from our mistakes? Balancing regulatory versus market-based initiatives and public versus private efforts, the Institute of Medicine presents wide-ranging recommendations for improving patient safety, in the areas of leadership, improved data collection and analysis, and development of effective systems at the level of direct patient care. To Err Is Human asserts that the problem is not bad people in health careâ€it is that good people are working in bad systems that need to be made safer. Comprehensive and straightforward, this book offers a clear prescription for raising the level of patient safety in American health care. It also explains how patients themselves can influence the quality of care that they receive once they check into the hospital. This book will be vitally important to federal, state, and local health policy makers and regulators, health professional licensing officials, hospital administrators, medical educators and students, health caregivers, health journalists, patient advocatesâ€as well as patients themselves. First in a series of publications from the Quality of Health Care in America, a project initiated by the Institute of Medicine |
| first in human study: Ethics Dumping Doris Schroeder, Julie Cook, François Hirsch, Solveig Fenet, Vasantha Muthuswamy, 2017-12-04 This open access book provides original, up-to-date case studies of “ethics dumping” that were largely facilitated by loopholes in the ethics governance of low and middle-income countries. It is instructive even to experienced researchers since it provides a voice to vulnerable populations from the fore mentioned countries. Ensuring the ethical conduct of North-South collaborations in research is a process fraught with difficulties. The background conditions under which such collaborations take place include extreme differentials in available income and power, as well as a past history of colonialism, while differences in culture can add a new layer of complications. In this context, up-to-date case studies of unethical conduct are essential for research ethics training. |
| first in human study: Toxicokinetics , 1995 |
| first in human study: The Immortal Life of Henrietta Lacks Rebecca Skloot, 2010-02-02 #1 NEW YORK TIMES BESTSELLER • “The story of modern medicine and bioethics—and, indeed, race relations—is refracted beautifully, and movingly.”—Entertainment Weekly NOW A MAJOR MOTION PICTURE FROM HBO® STARRING OPRAH WINFREY AND ROSE BYRNE • ONE OF THE “MOST INFLUENTIAL” (CNN), “DEFINING” (LITHUB), AND “BEST” (THE PHILADELPHIA INQUIRER) BOOKS OF THE DECADE • ONE OF ESSENCE’S 50 MOST IMPACTFUL BLACK BOOKS OF THE PAST 50 YEARS • WINNER OF THE CHICAGO TRIBUNE HEARTLAND PRIZE FOR NONFICTION NAMED ONE OF THE BEST BOOKS OF THE YEAR BY The New York Times Book Review • Entertainment Weekly • O: The Oprah Magazine • NPR • Financial Times • New York • Independent (U.K.) • Times (U.K.) • Publishers Weekly • Library Journal • Kirkus Reviews • Booklist • Globe and Mail Her name was Henrietta Lacks, but scientists know her as HeLa. She was a poor Southern tobacco farmer who worked the same land as her slave ancestors, yet her cells—taken without her knowledge—became one of the most important tools in medicine: The first “immortal” human cells grown in culture, which are still alive today, though she has been dead for more than sixty years. HeLa cells were vital for developing the polio vaccine; uncovered secrets of cancer, viruses, and the atom bomb’s effects; helped lead to important advances like in vitro fertilization, cloning, and gene mapping; and have been bought and sold by the billions. Yet Henrietta Lacks remains virtually unknown, buried in an unmarked grave. Henrietta’s family did not learn of her “immortality” until more than twenty years after her death, when scientists investigating HeLa began using her husband and children in research without informed consent. And though the cells had launched a multimillion-dollar industry that sells human biological materials, her family never saw any of the profits. As Rebecca Skloot so brilliantly shows, the story of the Lacks family—past and present—is inextricably connected to the dark history of experimentation on African Americans, the birth of bioethics, and the legal battles over whether we control the stuff we are made of. Over the decade it took to uncover this story, Rebecca became enmeshed in the lives of the Lacks family—especially Henrietta’s daughter Deborah. Deborah was consumed with questions: Had scientists cloned her mother? Had they killed her to harvest her cells? And if her mother was so important to medicine, why couldn’t her children afford health insurance? Intimate in feeling, astonishing in scope, and impossible to put down, The Immortal Life of Henrietta Lacks captures the beauty and drama of scientific discovery, as well as its human consequences. |
| first in human study: Novel Designs of Early Phase Trials for Cancer Therapeutics Shivaani Kummar, Chris Takimoto, 2018-05-26 Novel Designs of Early Phase Trials for Cancer Therapeutics provides a comprehensive review by leaders in the field of the process of drug development, the integration of molecular profiling, the changes in early phase trial designs, and endpoints to optimally develop a new generation of cancer therapeutics. The book discusses topics such as statistical perspectives on cohort expansions, the role and application of molecular profiling and how to integrate biomarkers in early phase trials. Additionally, it discusses how to incorporate patient reported outcomes in phase one trials. This book is a valuable resource for medical oncologists, basic and translational biomedical scientists, and trainees in oncology and pharmacology who are interested in learning how to improve their research by using early phase trials. |
| first in human study: Stem Cell and Gene Therapy for Cardiovascular Disease Emerson c. Perin, Leslie W. Miller, Doris Taylor, James T. Willerson, 2015-08-21 Stem Cell and Gene Therapy for Cardiovascular Disease is a state-of-the-art reference that combines, in one place, the breadth and depth of information available on the topic. As stem cell and gene therapies are the most cutting-edge therapies currently available for patients with heart failure, each section of the book provides information on medical trials from contributors and specialists from around the world, including not only what has been completed, but also what is planned for future research and trials. Cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals will find this book an invaluable resource for further study on the topic. - Provides information on stem and gene therapy medical trials from contributors and specialists around the world, including not only what has been completed, but also what is planned for future research and trials - Presents topics that can be applied to allogeneic cells, mesenchymal cells, gene therapy, cardiomyoctyes, iPS cells, MAPC's, and organogenesis - Covers the three areas with the greatest clinical trials to date: chronic limb ischemia, chronic angina, and acute MI - Covers the prevailing opinions on how to harness the body's natural repair mechanisms - Ideal resource for cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals |
| first in human study: Principles and Practice of Clinical Trial Medicine Richard Chin, Bruce Y Lee, 2008-07-25 Clinical trials are an important part of medicine and healthcare today, deciding which treatments we use to treat patients. Anyone involved in healthcare today must know the basics of running and interpreting clinical trial data. Written in an easy-to-understand style by authors who have considerable expertise and experience in both academia and industry, Principles and Practice of Clinical Trial Medicine covers all of the basics of clinical trials, from legal and ethical issues to statistics, to patient recruitment and reporting results. - Jargon-free writing style enables those with less experience to run their own clinical trials and interpret data - Book contains an ideal mix of theory and practice so researchers will understand both the rationale and logistics to clinical trial medicine - Expert authorship whose experience includes running clinical trials in an academic as well as industry settings - Numerous illustrations reinforce and elucidate key concepts and add to the book's overall pedagogy |
| first in human study: Gene Transfer and the Ethics of First-in-Human Research Jonathan Kimmelman, 2010 Examines the ethical and policy dimensions of testing novel medical interventions in human beings for the first time. |
| first in human study: FDA in the Twenty-First Century Holly Fernandez Lynch, I. Glenn Cohen, 2015-09-08 In its decades-long effort to assure the safety, efficacy, and security of medicines and other products, the Food and Drug Administration has struggled with issues of funding, proper associations with industry, and the balance between consumer choice and consumer protection. Today, these challenges are compounded by the pressures of globalization, the introduction of novel technologies, and fast-evolving threats to public health. With essays by leading scholars and government and private-industry experts, FDA in the Twenty-First Century addresses perennial and new problems and the improvements the agency can make to better serve the public good. The collection features essays on effective regulation in an era of globalization, consumer empowerment, and comparative effectiveness, as well as questions of data transparency, conflicts of interest, industry responsibility, and innovation policy, all with an emphasis on pharmaceuticals. The book also intervenes in the debate over off-label drug marketing and the proper role of the FDA before and after a drug goes on the market. Dealing honestly and thoroughly with the FDA's successes and failures, these essays rethink the structure, function, and future of the agency and the effect policy innovations may have on regulatory institutions abroad. |
| first in human study: The Belmont Report United States. National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research, 1978 |
| first in human study: Phase I Cancer Clinical Trials Elizabeth A. Eisenhauer, Chris Twelves, Marc E. Buyse, 2014-06 Phase I trials are a critical first step in the study of novel cancer therapeutic approaches. As this title is the only comprehensive book on this topic, it is a useful resource for oncology trainees or specialists interested in understanding cancer drug development. New to this edition are chapters on Phase 0 Trials and Immunotherapeutics, and updated information on the process, pitfalls, and logistics of Phase I Trials. |
| first in human study: Transforming Clinical Research in the United States Institute of Medicine, Board on Health Sciences Policy, Forum on Drug Discovery, Development, and Translation, 2010-10-22 An ideal health care system relies on efficiently generating timely, accurate evidence to deliver on its promise of diminishing the divide between clinical practice and research. There are growing indications, however, that the current health care system and the clinical research that guides medical decisions in the United States falls far short of this vision. The process of generating medical evidence through clinical trials in the United States is expensive and lengthy, includes a number of regulatory hurdles, and is based on a limited infrastructure. The link between clinical research and medical progress is also frequently misunderstood or unsupported by both patients and providers. The focus of clinical research changes as diseases emerge and new treatments create cures for old conditions. As diseases evolve, the ultimate goal remains to speed new and improved medical treatments to patients throughout the world. To keep pace with rapidly changing health care demands, clinical research resources need to be organized and on hand to address the numerous health care questions that continually emerge. Improving the overall capacity of the clinical research enterprise will depend on ensuring that there is an adequate infrastructure in place to support the investigators who conduct research, the patients with real diseases who volunteer to participate in experimental research, and the institutions that organize and carry out the trials. To address these issues and better understand the current state of clinical research in the United States, the Institute of Medicine's (IOM) Forum on Drug Discovery, Development, and Translation held a 2-day workshop entitled Transforming Clinical Research in the United States. The workshop, summarized in this volume, laid the foundation for a broader initiative of the Forum addressing different aspects of clinical research. Future Forum plans include further examining regulatory, administrative, and structural barriers to the effective conduct of clinical research; developing a vision for a stable, continuously funded clinical research infrastructure in the United States; and considering strategies and collaborative activities to facilitate more robust public engagement in the clinical research enterprise. |
| first in human study: Women and Health Research Institute of Medicine, Committee on Ethical and Legal Issues Relating to the Inclusion of Women in Clinical Studies, 1994-02-01 In the nineteenth century some scientists argued that women should not be educated because thinking would use energy needed by the uterus for reproduction. The proof? Educated women had a lower birth rate. Today's researchers can only shake their heads at such reasoning. Yet professional journals and the popular press are increasingly criticizing medical research for ignoring women's health issues. Women and Health Research examines the facts behind the public's perceptions about women participating as subjects in medical research. With the goal of increasing researchers' awareness of this important topic, the book explores issues related to maintaining justice (in its ethical sense) in clinical studies. Leading experts present general principles for the ethical conduct of research on womenâ€principles that are especially important in the light of recent changes in federal policy on the inclusion of women in clinical research. Women and Health Research documents the historical shift from a paternalistic approach by researchers toward women and a disproportionate reliance on certain groups for research to one that emphasizes proper access for women as subjects in clinical studies in order to ensure that women receive the benefits of research. The book addresses present-day challenges to equity in four areas: Scientificâ€Do practical aspects of scientific research work at cross-purposes to gender equity? Focusing on drug trials, the authors identify rationales for excluding people from research based on demographics. Social and Ethicalâ€The authors offer compelling discussions on subjectivity in science, the evidence for male bias, and issues related to race and ethnicity, as well as the recruitment, retention, and protection of research participants. Legalâ€Women and Health Research reviews federal research policies that affect the inclusion of women and evaluates the basis for researchers' fears about liability, citing court cases. Riskâ€The authors focus on risks to reproduction and offspring in clinical drug trials, exploring how risks can be identified for study participants, who should make the assessment of risk and benefit for participation in a clinical study, and how legal implications could be addressed. This landmark study will be of immediate use to the research community, policymakers, women's health advocates, attorneys, and individuals. |
| first in human study: Adverse Events Jill A. Fisher, 2020-05-12 Winner, 2022 Donald W. Light Award for Applied Medical Sociology, given by the Medical Sociology Section of the American Sociological Association Winner, 2021 Robert K. Merton Book Award, given by the Science, Knowledge, and Technology Section of the American Sociological Association 2021 Outstanding Academic Title, Choice Magazine Explores the social inequality of clinical drug testing and its effects on scientific results Imagine that you volunteer for the clinical trial of an experimental drug. The only direct benefit of participating is that you will receive up to $5,175. You must spend twenty nights literally locked in a research facility. You will be told what to eat, when to eat, and when to sleep. You will share a bedroom with several strangers. Who are you, and why would you choose to take part in this kind of study? This book explores the hidden world of pharmaceutical testing on healthy volunteers. Drawing on two years of fieldwork in clinics across the country and 268 interviews with participants and staff, it illustrates how decisions to take part in such studies are often influenced by poverty and lack of employment opportunities. It shows that healthy participants are typically recruited from African American and Latino/a communities, and that they are often serial participants, who obtain a significant portion of their income from these trials. This book reveals not only how social inequality fundamentally shapes these drug trials, but it also depicts the important validity concerns inherent in this mode of testing new pharmaceuticals. These highly controlled studies bear little resemblance to real-world conditions, and everyone involved is incentivized to game the system, ultimately making new drugs appear safer than they really are. Adverse Events provides an unprecedented view of the intersection of racial inequalities with pharmaceutical testing, signaling the dangers of this research enterprise to both social justice and public health. |
| first in human study: Handbook for Clinical Research Flora Hammond, MD, James F. Malec, Todd Nick, Ralph Buschbacher, MD, 2014-08-26 With over 80 information-packed chapters, Handbook for Clinical Research delivers the practical insights and expert tips necessary for successful research design, analysis, and implementation. Using clear language and an accessible bullet point format, the authors present the knowledge and expertise developed over time and traditionally shared from mentor to mentee and colleague to colleague. Organized for quick access to key topics and replete with practical examples, the book describes a variety of research designs and statistical methods and explains how to choose the best design for a particular project. Research implementation, including regulatory issues and grant writing, is also covered. The book opens with a section on the basics of research design, discussing the many ways in which studies can be organized, executed, and evaluated. The second section is devoted to statistics and explains how to choose the correct statistical approach and reviews the varieties of data types, descriptive and inferential statistics, methods for demonstrating associations, hypothesis testing and prediction, specialized methods, and considerations in epidemiological studies and measure construction. The third section covers implementation, including how to develop a grant application step by step, the project budget, and the nuts and bolts of the timely and successful completion of a research project and documentation of findings: procedural manuals and case report forms collecting, managing and securing data operational structure and ongoing monitoring and evaluation and ethical and regulatory concerns in research with human subjects. With a concise presentation of the essentials for successful research, the Handbook for Clinical Research is a valuable addition to the library of any student, research professional, or clinician interested in expanding the knowledge base of his or her field. Key Features: Delivers the essential elements, practical insights, and trade secrets for ensuring successful research design, analysis, and implementation Presents the nuts and bolts of statistical analysis Organized for quick access to a wealth of information Replete with practical examples of successful research designs Û from single case designs to meta-analysis - and how to achieve them Addresses research implementation including regulatory issues and grant writing |
| first in human study: Beyond the HIPAA Privacy Rule Institute of Medicine, Board on Health Care Services, Board on Health Sciences Policy, Committee on Health Research and the Privacy of Health Information: The HIPAA Privacy Rule, 2009-03-24 In the realm of health care, privacy protections are needed to preserve patients' dignity and prevent possible harms. Ten years ago, to address these concerns as well as set guidelines for ethical health research, Congress called for a set of federal standards now known as the HIPAA Privacy Rule. In its 2009 report, Beyond the HIPAA Privacy Rule: Enhancing Privacy, Improving Health Through Research, the Institute of Medicine's Committee on Health Research and the Privacy of Health Information concludes that the HIPAA Privacy Rule does not protect privacy as well as it should, and that it impedes important health research. |
| first in human study: Principles of Safety Pharmacology Michael K. Pugsley, Michael J Curtis, 2015-06-19 This book illustrates, in a comprehensive manner, the most current areas of importance to Safety Pharmacology, a burgeoning unique pharmacological discipline with important ties to academia, industry and regulatory authorities. It provides readers with a definitive collection of topics containing essential information on the latest industry guidelines and overviews current and breakthrough topics in both functional and molecular pharmacology. An additional novelty of the book is that it constitutes academic, pharmaceutical and biotechnology perspectives for Safety Pharmacology issues. Each chapter is written by an expert in the area and includes not only a fundamental background regarding the topic but also detailed descriptions of currently accepted, validated models and methods as well as innovative methodologies used in drug discovery. |
| first in human study: Reviewing Clinical Trials Chinese University of Hong Kong, Chinese University of Hong Kong. Clinical Trials Centre, Washington, DC. Association for the Accreditation of Human research Protection Programs, Inc, 2010 The idea for this manual came from Pfizer in the US, which provided the Clinical Trials Centre at The University of Hong Kong, Hong Kong SAR, PR China with a nonbinding grant for its development. The general project layout protocol was accepted by Pfizer in July 2009. Pfizer has not in any way interfered with the project, except for providing nonbinding comments to the final product. The entire text of this manual was written by Johan PE Karlberg. Marjorie A Speers provided considerable and essential comments on the contents and the first and subsequent drafts. A group of international human research protection experts mostly working in non-profit institutions or organisations - see Contributors for details - reviewed and provided important comments on the contents and final draft. It was solely created with the intention to promote human research protection of participants in clinical trials. This manual will be translated into numerous languages and is provided free of charge as an electronic file over the Internet (http://www.ClinicalTrialMagnifier.com) and offered in print for a fee. The objective beyond this project is to establish educational activities, developed around the manual, and jointly organised with leading academic institutions worldwide. |
| first in human study: A Clinical Trials Manual From The Duke Clinical Research Institute Margaret Liu, Kate Davis, 2011-08-24 The publication of the second edition of this manual comes at an important juncture in the history of clinical research. As advances in information technology make it possible to link individuals and groups in diverse locations in jointly seeking the answers to pressing global health problems, it is critically important to remain vigilant about moral and ethical safeguards for every patient enrolled in a trial. Those who study this manual will be well aware of how to ensure patient safety along with fiscal responsibility, trial efficiency, and research integrity. —Robert Harrington, Professor of Medicine, Director, Duke Clinical Research Institute, Durham, North Carolina, USA The Duke Clinical Research Institute (DCRI) is one of the world's leading academic clinical research organizations; its mission is to develop and share knowledge that improves the care of patients around the world through innovative clinical research. This concise handbook provides a practical nuts and bolts approach to the process of conducting clinical trials, identifying methods and techniques that can be replicated at other institutions and medical practices. Designed for investigators, research coordinators, CRO personnel, students, and others who have a desire to learn about clinical trials, this manual begins with an overview of the historical framework of clinical research, and leads the reader through a discussion of safety concerns and resulting regulations. Topics include Good Clinical Practice, informed consent, management of subject safety and data, as well as monitoring and reporting adverse events. Updated to reflect recent regulatory and clinical developments, the manual reviews the conduct of clinical trials research in an increasingly global context. This new edition has been further expanded to include: In-depth information on conducting clinical trials of medical devices and biologics The role and responsibilities of Institutional Review Boards, and Recent developments regarding subject privacy concerns and regulations. Ethical documents such as the Belmont Report and the Declaration of Helsinki are reviewed in relation to all aspects of clinical research, with a discussion of how researchers should apply the principles outlined in these important documents. This graphically appealing and eminently readable manual also provides sample forms and worksheets to facilitate data management and regulatory record retention; these can be modified and adapted for use at investigative sites. |
| first in human study: Pharmaceutical Medicine and Translational Clinical Research Divya Vohora, Gursharan Singh, 2017-11-14 Pharmaceutical Medicine and Translational Clinical Research covers clinical testing of medicines and the translation of pharmaceutical drug research into new medicines, also focusing on the need to understand the safety profile of medicine and the benefit-risk balance. Pharmacoeconomics and the social impact of healthcare on patients and public health are also featured. It is written in a clear and straightforward manner to enable rapid review and assimilation of complex information and contains reader-friendly features.As a greater understanding of these aspects is critical for students in the areas of pharmaceutical medicine, clinical research, pharmacology and pharmacy, as well as professionals working in the pharmaceutical industry, this book is an ideal resource. - Includes detailed coverage of current trends and key topics in pharmaceutical medicine, including biosimilars, biobetters, super generics, and - Provides a comprehensive look at current and important aspects of the science and regulation of drug and biologics discovery |
| first in human study: The Turnaway Study Diana Greene Foster, 2021-06 Now with a new afterword by the author--Back cover. |
| first in human study: The Oxford Textbook of Clinical Research Ethics Ezekiel J. Emanuel, Christine C. Grady, Robert A. Crouch, Reidar K. Lie, Franklin G. Miller, David D. Wendler, 2011-02 The Oxford Textbook of Clinical Research Ethics is the first comprehensive and systematic reference on clinical research ethics. Under the editorship of experts from the U.S. National Institutes of Health of the United States, the book's 73 chapters offer a wide-ranging and systematic examination of all aspects of research with human beings. Considering the historical triumphs of research as well as its tragedies, the textbook provides a framework for analyzing the ethical aspects of research studies with human beings. Through both conceptual analysis and systematic reviews of empirical data, the contributors examine issues ranging from scientific validity, fair subject selection, risk benefit ratio, independent review, and informed consent to focused consideration of international research ethics, conflicts of interests, and other aspects of responsible conduct of research. The editors of The Oxford Textbook of Clinical Research Ethics offer a work that critically assesses and advances scholarship in the field of human subjects research. Comprehensive in scope and depth, this book will be a crucial resource for researchers in the medical sciences, as well as teachers and students. |
| first in human study: Critical Appraisal of Epidemiological Studies and Clinical Trials Mark Elwood, 2007-02-22 This book presents a logical system of critical appraisal, to allow readers to evaluate studies and to carry out their own studies more effectively. This system emphasizes the central importance of cause and effect relationships. Its great strength is that it is applicable to a wide range of issues, and both to intervention trials and observational studies. This system unifies the often different approaches used in epidemiology, health services research, clinical trials, and evidence-based medicine, starting from a logical consideration of cause and effect. The author's approach to the issues of study design, selection of subjects, bias, confounding, and the place of statistical methods has been praised for its clarity and interest. Systematic reviews, meta-analysis, and the applications of this logic to evidence-based medicine, knowledge-based health care, and health practice and policy are discussed. Current and often controversial examples are used, including screening for prostate cancer, publication bias in psychiatry, public health issues in developing countries, and conflicts between observational studies and randomized trials. Statistical issues are explained clearly without complex mathematics, and the most useful methods are summarized in the appendix. The final chapters give six applications of the critical appraisal of major studies: randomized trials of medical treatment and prevention, a prospective and a retrospective cohort study, a small matched case-control study, and a large case-control study. In these chapters, sections of the original papers are reproduced and the original studies placed in context by a summary of current developments. |
| first in human study: Clinical Trials in Oncology, Third Edition Stephanie Green, Jacqueline Benedetti, Angela Smith, John Crowley, 2012-05-09 The third edition of the bestselling Clinical Trials in Oncology provides a concise, nontechnical, and thoroughly up-to-date review of methods and issues related to cancer clinical trials. The authors emphasize the importance of proper study design, analysis, and data management and identify the pitfalls inherent in these processes. In addition, the book has been restructured to have separate chapters and expanded discussions on general clinical trials issues, and issues specific to Phases I, II, and III. New sections cover innovations in Phase I designs, randomized Phase II designs, and overcoming the challenges of array data. Although this book focuses on cancer trials, the same issues and concepts are important in any clinical setting. As always, the authors use clear, lucid prose and a multitude of real-world examples to convey the principles of successful trials without the need for a strong statistics or mathematics background. Armed with Clinical Trials in Oncology, Third Edition, clinicians and statisticians can avoid the many hazards that can jeopardize the success of a trial. |
| first in human study: Face Value Alexander Todorov, 2017-05-30 The scientific story of first impressions—and why the snap character judgments we make from faces are irresistible but usually incorrect We make up our minds about others after seeing their faces for a fraction of a second—and these snap judgments predict all kinds of important decisions. For example, politicians who simply look more competent are more likely to win elections. Yet the character judgments we make from faces are as inaccurate as they are irresistible; in most situations, we would guess more accurately if we ignored faces. So why do we put so much stock in these widely shared impressions? What is their purpose if they are completely unreliable? In this book, Alexander Todorov, one of the world's leading researchers on the subject, answers these questions as he tells the story of the modern science of first impressions. Drawing on psychology, cognitive science, neuroscience, computer science, and other fields, this accessible and richly illustrated book describes cutting-edge research and puts it in the context of the history of efforts to read personality from faces. Todorov describes how we have evolved the ability to read basic social signals and momentary emotional states from faces, using a network of brain regions dedicated to the processing of faces. Yet contrary to the nineteenth-century pseudoscience of physiognomy and even some of today's psychologists, faces don't provide us a map to the personalities of others. Rather, the impressions we draw from faces reveal a map of our own biases and stereotypes. A fascinating scientific account of first impressions, Face Value explains why we pay so much attention to faces, why they lead us astray, and what our judgments actually tell us. |
| first in human study: Cardiovascular Thrombus On Topaz, 2018-08-18 Cardiovascular Thrombus: From Pathology and Clinical Presentations to Imaging, Pharmacotherapy and Interventions provides a comprehensive, up-to-date presentation of the research and clinical practices as related to the contemporary aspects of the diagnosis and management of cardiovascular thrombosis. The formation, identification and management of cardiovascular thrombus is of paramount importance for researchers and practicing physicians, yet it remains one of the most challenging diagnostic and clinical scenarios. This important reference connects between research, up-to-date clinical knowledge, and the technological tools available for diagnosis and management of thrombus in cardiovascular medicine. The book includes comprehensive descriptions and review of pathology, clinical presentations, diagnosis, pharmacotherapy, interventions and future trends. It is the perfect reference for basic science students and researchers in general and interventional cardiology, general and interventional radiology, vascular medicine specialists, and vascular, general and cardiac surgeons. Provides comprehensive presentation of the pathophysiology, clinical presentations and diagnosis of cardiovascular thrombosis Includes the most up-to-date information on the practical management of patients with thrombus related conditions Written by the leading experts in the field Describes the current and upcoming pharmacotherapy and technology available for thrombus research and treatment |
| first in human study: Volunteers in Research and Testing Bryony Close, R. Coomber, Anthony Hubbard, John Illingworth, 1997-02-01 This text discusses the use of volunteers, either healthy or undergoing treatment, in the research and testing of medicinal and non-medicinal products. The extent to which the improved use of such volunteers could reduce the need for animal tests is |
| first in human study: Responsible Conduct of Research Adil E. Shamoo, David B. Resnik, 2009-02-12 Recent scandals and controversies, such as data fabrication in federally funded science, data manipulation and distortion in private industry, and human embryonic stem cell research, illustrate the importance of ethics in science. Responsible Conduct of Research, now in a completely updated second edition, provides an introduction to the social, ethical, and legal issues facing scientists today. |
| first in human study: Anatomy and Physiology J. Gordon Betts, Peter DeSaix, Jody E. Johnson, Oksana Korol, Dean H. Kruse, Brandon Poe, James A. Wise, Mark Womble, Kelly A. Young, 2013-04-25 |
| first in human study: Safety Evaluation of Pharmaceuticals and Medical Devices Shayne C. Gad, 2010-10-26 The inspiration for this text was the 1988 volume by Alder and Zbinden, written before the ICH harmonization process for drug safety evaluation (or its ISO analog for device biocompatibility evaluation) had been initiated or come to force. Since then, much has changed in both the world and practice of medicine and the regulation of drugs. The intent of this volume is to provide similar guidance as to what nonclinical safety assessment tests need to be performed to move a drug into man, through development and to market approved (this intent was subsequently extended to cover the closely related medical device biotechnology, and combination product fields) in a concise, abbreviated manner for all the major world market countries. |
| first in human study: International Ethical Guidelines for Health-Related Research Involving Humans Council for International Organizations of Medical Sciences (CIOMS), 2017-01-31 In the new 2016 version of the ethical guidelines, CIOMS provides answers to a number of pressing issues in research ethics. The Council does so by stressing the need for research having scientific and social value, by providing special guidelines for health-related research in low-resource settings, by detailing the provisions for involving vulnerable groups in research and for describing under what conditions biological samples and health-related data can be used for research.--Page 4 de la couverture. |
| first in human study: Science, Medicine, and Animals National Research Council, Division on Earth and Life Studies, Institute for Laboratory Animal Research, 2006-02-19 Science, Medicine, and Animals explains the role that animals play in biomedical research and the ways in which scientists, governments, and citizens have tried to balance the experimental use of animals with a concern for all living creatures. An accompanying Teacher's Guide is available to help teachers of middle and high school students use Science, Medicine, and Animals in the classroom. As students examine the issues in Science, Medicine, and Animals, they will gain a greater understanding of the goals of biomedical research and the real-world practice of the scientific method in general. Science, Medicine, and Animals and the Teacher's Guide were written by the Institute for Laboratory Animal Research and published by the National Research Council of the National Academies. The report was reviewed by a committee made up of experts and scholars with diverse perspectives, including members of the U.S. Department of Agriculture, National Institutes of Health, the Humane Society of the United States, and the American Society for the Prevention of Cruelty to Animals. The Teacher's Guide was reviewed by members of the National Academies' Teacher Associates Network. Science, Medicine, and Animals is recommended by the National Science Teacher's Association NSTA Recommends. |
Design and Conduct Considerations for First‐in‐Human Trials
First‐in‐human (FIH) trials serve as the link to advance new promising drug candidates and are conducted primarily to determine the safe dose range for further clinical development. …
Revised guideline on first-in-human clinical trials
Jul 25, 2017 · This document addresses non-clinical and clinical issues for consideration prior to the first administration of an investigational medicinal product in humans. It also addresses the …
Phase I and first-in-human Clinical Trials and FDA's
When an investigational medicinal product (IMP) that is developed and assessed through in-vitro or animal testing, is tested on human subjects for the first time, it is called Phase I or First-in …
Expansion Cohorts: Use in First-In-Human Clinical Trials to Expedite
Mar 2, 2022 · The purpose of this guidance is to provide advice to sponsors regarding the design and conduct of first-in-human (FIH) clinical trials intended to efficiently expedite the clinical …
First-In-Human Clinical Trial Requirement -BioPharma Services
First-in-human clinical trials, also known as FIH trials, are studies during which a drug is administered to humans for the first time after it’s testing in in vitro and in vivo preclinical, …
Definition of first-in-human study - NCI Dictionary of Cancer Terms
A type of clinical trial in which a new drug, procedure, or treatment is tested in humans for the first time. First-in-human studies take place after the new treatment has been tested in laboratory …
First in Human Studies - Association of the British …
First in Human (FIH) clinical trials are part of the exploratory phase of drug development and represent a significant milestone in the clinical development of new medicines.
A brief survey of first-in-human studies - PubMed
First-in-human (FIH) studies are a critical step in the drug development process and typically aim to characterize a compound's pharmacokinetics, potential effective concentration or dose, and …
First-in-Human Trials | Single and Multiple Ascending Dose Study
The purpose of First-in-Human (FIH) trials is to determine a safe dose range for further clinical development, and to develop a better understanding of the investigational product’s …
Chapter 6. Phase I Trials: First in Human - McGraw Hill Medical
The first step in human drug development involves Phase I or “first-in-human” studies. These trials are an exciting milestone in the drug development process, because this is the first time an …
Phase I, two-part, multicenter, first-in-human (FIH) study of …
Phase I, two-part, multicenter, first-in-human (FIH) study of DS-6000a in subjects with advanced renal cell carcinoma (RCC) and ovarian tumors (OVC). ... Conclusions: The interim data from …
First-in-human study of the safety, pharmacokinetics, and ...
2.1. Study design This first-in-human study of TVB-2640 employed a multi-center, open-label, dose-escalation and cohort expansion design. This study was conducted in accordance with …
Izokibep: Preclinical development and first-in-human study …
A Phase 1 first-in-human study was conducted to establish the safety, pharmacokinetics, and preliminary efficacy of izokibep, when administered intravenously and subcutaneously as …
Hyperbaric Oxygen Therapy: First Human Study Shows …
For the first-time a human study shows the reversal in biology of aging, with hyperbaric oxygen therapy (HBOT) TEL AVIV – November 18, 2020: In a scientifically verified approach, …
First human safety and effectiveness study of defibrillation …
First human safety and effectiveness study of defibrillation with a novel patch wearable cardioverter-defibrillator Milan Chovanec1, Jan Petrů1, Pavel Hála 1, Stepan Kralovec1, Anjali …
Efficiency and Target Derepression of Anti-miR-92a: Results …
Results of a First in Human Study Wesley Tyler Abplanalp, 1,2, *Ariane Fischer, 1, David John,1 Andreas M. Zeiher, 2,3 Willy Gosgnach, 4 Helene Darville, 4 Rusty Montgomery, 5 Linda …
A first-in-human study of the fibroblast activation protein
May 10, 2023 · This first-in-human study evaluated RO7122290, a bispecific fusion protein carrying a split trimeric 4-1BB (CD137) ligand and a fibroblast activation protein α (FAP) …
Safety and Efficacy of Menin Inhibition in Patients with MLL
Safety and Efficacy of Menin Inhibition in Patients with MLL-Rearranged and NPM1 Mutant Acute Leukemia: A Phase 1, First-in-Human Study of SNDX-5613 Author: Hope Qamoos Created …
First-in-human study of ABBV-400, a novel c-Met targeting …
First-in-human study of ABBV-400, a novel c-Met–targeting antibody-drug con-jugate, in advanced solid tumors: Results in colorectal cancer. ... The study is also evaluating ABBV-400 …
First‐in‐human study evaluating safety, pharmacokinetics, …
First-in Human Study of Lorundrostat, a Potent and Highly Selective Aldosterone Synthase Inhibitor. Presented at the American College of Cardiology's 72nd Annual Scientific Session …
A phase I, first-in-human study to evaluate the safety and …
A phase I, first-in-human study to evaluate the safety and tolerability, pharmacokinetics, and pharmacodynamics of MRG-001 in healthy subjects Ali R. Ahmadi,1,4,* George Atiee,2 Bart …
1073 A phase 1, first-in-human, open-label study evaluating …
1073 A PHASE 1, FIRST-IN-HUMAN, OPEN-LABEL STUDY EVALUATING THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND PRELIMINARY …
Intense pulsed light: The early years - clderm.com
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The first in‐human study to evaluate the antiplatelet …
The first in-human study to evaluate the antiplatelet properties of the clopidogrel conjugate DT-678 in acute coronary syndrome patients and healthy volunteers ... human subjects. This …
First-in-human study of dosimetry, safety and efficacy for
[29]. In this first-in-human study, we investigate the phar-macokinetics, dosimetry, biodistribution, and toxicity of [177Lu]Lu-P15-073 as a RLT agent for bone metastasis. Methods Preparation …
Preliminary results from PIKture-01, a First-in-Human Study …
• Efficacy data are immature in this ongoing dose-escalation study • 13/14 patients at doses ≥600 mg BID remain on study • 2/3 patients at 300 mg showed prolonged stable disease • …
APSF Vision - Anesthesia Patient Safety Foundation
First Human Study of the Investigational Sedative and Anesthetic Drug AZD3043: A Dose-Escalation Trial to Assess the Safety, Pharmacokinetics, and Efficacy of a 30-Minute Infusion …
Estimating the “First in human” dose – a revisit with …
Dec 16, 2013 · first clinical study in humans. In this mini review, we will discuss the no observed adverse effect level (NOAEL) and the minimum anticipated biological effect level (MABEL) …
Safety, tolerability, pharmacokinetic, and pharmacodynamic ...
first dose of the SAD study. In addition, the maximum toler - ated dose of the preclinical study was found to be 1000mg/ kg in beagle dogs, and safety factors of 10 and 30 were ap-plied, …
First Results of a Phase 1, First-in-Human, Dose-Escalation …
First Results of a Phase 1, First-in-Human, Dose-Escalation Study of ISB 2001, a BCMAxCD38xCD3 Targeting Trispecific Antibody in Patients with Relapsed/Refractory …
PROTOCOL Doc #: 104-010990-00 First-in-Human Study …
First-in-Human Study Protocol – Monarch Urology (Protocol No.: 2021-URO-0001) Rev: C Page: 2 of 48 Printed copies are considered UNCONTROLLED documents after the date printed. …
Translational findings for odronextamab: From preclinical …
Study Highlights WHAT IS THE CURRENT KNOWLEDGE ON THE TOPIC? Odronextamab is an IgG4-based CD20xCD3-targeting bispecific antibody being evaluated in a first-in-human (FIH) …
Preliminary results of a first-in-human study on ESG206, a …
Preliminary results of a first-in-human study on ESG206, a novel anti-BAFF-R monoclonal antibody, in relapsed or refractory B-cell malignancies. Yuqin Song, Ningjing Lin, Hui Liu, Qing …
First-in-Human, Multicenter, Phase I Dose-Escalation and …
Mar 26, 2020 · First-in-Human, Multicenter, Phase I Dose-Escalation and Expansion Study of Anti-Mesothelin Antibody–Drug Conjugate ... PURPOSE This phase I study, which to our …
First-in-human study of ZGGS15, a dual specific antibody …
First-in-human study of ZGGS15, a dual specific antibody targeting LAG-3 and TIGIT, as monotherapy in patients with advanced solid tumors. Ji Zhu, Zhen Wang, Xiaoli Chai, Lihua …
First-in-Human study of JNJ-55920839 in healthy volunteers …
Added value of this study. In this first-in-human, phase 1 clinical trial, we showed that JNJ-55920839, a fully human monoclonal antibody directly . targeting multiple type I IFNs, was …
First-in-human, phase I study of CBP-1008, a first-in-class …
First-in-human, phase I study of CBP-1008, a first-in-class bispecific ligand drug conjugate (Bi-XDC), in patients with advanced solid tumors. Subject: JCO 2024.42:5556-5556 Created Date:
STEADFAST: A First-in-Human Study Assessing HLA-A*02 …
• In this first-in-human (FIH) phase 1/2study, HLA-A*02–negative patients with ESRD awaiting a kidney graft from an HLA-A*02– ... • This FIH study provided the setting for the first-ever …
First-in-human study of THB335, an oral small molecule …
673First-in-human study of THB335, an oral small molecule inhibitor of Wild Type KIT Receptor Tyrosine Kinase: Initial Safety, Pharmacokinetics (PK) and Pharmacodynamics (PD) Results …
First-in-human, phase 1/2 study of GSK4524101, an oral …
and PARPi, this first-in-human study investigates treatment with GSK4524101, an investiga-tionalPOLQi,withorwithoutthePARPiniraparib,inpatientswithsolidtumors.Methods:This open …
FIRST IN HUMAN STUDY OF AUTO4, A TRBC1-TARGETING …
• Here we present first in human clinical findings of AUTO4, a TRBC1 directed autologous CAR T cell therapy, tested against relapsed/refractory (r/r) TRBC1+ PTCL. NCT03590574 is a multi …
Workshop on the Guideline for First-In-Man Clinical Trials for ...
human safety Translate the science to humans and account for differences in relative potency Estimate the clinical starting dose for FTIH study using both toxicology AND pharmacology …
First- in- human study of the safety, tolerability, …
A first-in-human study evaluated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ALPN-101 in healthy adult subjects. ALPN- 101 was generally well …
First-in-human phase 1/2a study of the first-in-class, next …
First-in-human phase 1/2a study of the first-in-class, next-generation CDK4-selective inhibitor PF-07220060 + endocrine therapy (ET): Updated safety data in patients with HR+/HER22 mBC. …
First-in-human study of 225actinium mti-201 (225Ac-MTI …
Methods: This is a single institution first-in-human phase I study (NCT05496686) of 225Ac-MTI-201 in metastatic UM. The primary objective is to determine the safety of a single intravenous …
A Phase 1, First-In-Human, Randomized, Double-Blind, …
Design of the Study: This is a Phase 1, first-in-human, randomized, double-blind, placebo-controlled, single ascending dose (SAD) study, in six (6) cohorts (five fasted cohorts, one fed …
A First-in-Human Study of ARO-RAGE, a Novel Inhaled RNA …
A First-in-Human Study of ARO -RAGE, a Novel Inhaled RNA-Interference Therapy for Asthma. O’Carroll M. 1, Huetsch J. 2, Hamilton J. 2, Ta A. 2, Moser L. 2, Alagarsamy S. 2, Salathe M. …
First-in-human phase 1 study of CC-94676, a first-in-class …
First-in-human phase 1 study of CC-94676, a first-in-class androgen receptor (AR) ligand-directed degrader (LDD), in patients (pts) with metastatic castration-resistant prostate cancer …
A First-in-Human Phase I Study to Assess the …
A First-in-Human Phase I Study to Assess the Pharmacodynamic Profile of a Novel Potassium Channel Opener (XEN1101) on Human Cortical Excitability with TMS-EEG and TMS-EMG ... • …
How to choose the MRSD for FTIH clinical trials
First-Time-In-Human Clinical Trials Howard Lee, MD, PhD Associate Adjunct Professor Director, Center for Drug Development Science Department of Biopharmaceutical Sciences ...
First-in-Human Study of 18F-Labeled PET Tracer for …
The clinical study protocol was approved by Yokohama City Uni-versity Human Investigation Committee in accordance with the ethical guidelines for medical and health research involving …
51. A First-In-Human Phase I Study of LYT-300, a First-In …
A First-In-Human Phase I Study of LYT-300, a First-In-Class Orally Bioavailable Prodrug of the Neuro-steroid Allopregnanolone That is Absorbed via the Lymphatic System James Shipley 1, …
A first in human study in healthy volunteers to assess the …
Mar 25, 2022 · A first in human study in healthy volunteers to assess the safety, tolerability, and pharmacokinetics of BMS-986238 in healthy participants Submission date 25/03/2022 …
P505 First in Human, Single Ascending Dose Study in …
First in Human, Single Ascending Dose Study in Healthy Volunteers of SNDX-6352, a Humanized IgG4 Monoclonal Antibody Targeting Colony Stimulating Factor-1 Receptor (CSF-1R) ... • The …
A new resorbable magnesium scaffold (DREAMS 3G): 12
IOMAG-I is a European, multicentre first-in-human study assessing the safety and performance of a third-generation sirolimus-eluting resorbable magnesium scaf-fold (DREAMS 3G, …
A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, …
a randomized, double-blind, placebo-controlled, first-in-human study to assess safety, tolerability, and pharmacokinetics of single and multiple ascending doses of sbp-9330 (with a nested food …
A first-in-human study of IK-595, an oral MEK/RAF …
A first-in-human study of IK-595, an oral MEK/RAF molecular glue, in patients with RAS- or RAF-altered advanced solid tumors. Nehal J. Lakhani, Alexander I. Spira, Anthony W. Tolcher, …
An open-label, first-in-human study of BAY2927088 in …
Study Phase I first-in-human trial (NCT05099172) Status Enrolling patients globally into dose-escalation and backfill cohorts Participants Patients with advanced EGFR/HER2 mutation-
First-in-human study of Sirona: a study to determine safety ...
Mar 16, 2022 · Study information Scientific Title First-in-human study of Sirona, an expanding hydrogel tablet, designed to promote weight loss in humans: a study to determine safety, …
A phase 1a/b, multi-regional, first-in-human study of …
A phase 1a/b, multi-regional, first-in-human study of CS5001, a novel anti-ROR1 ADC, in patients with advanced solid tumors and lymphomas. Subject: JCO 2024.42:3023-3023 Created Date:
