| Description |
1 online resource (1 PDF file (xx, 106 pages)) : illustrations |
| Contents |
Intro -- FrontMatter -- Reviewers -- Acknowledgments -- Contents -- Boxes, Figures, and Table -- Acronyms and Abbreviations -- 1 Introduction and Overview -- 2 Developing First-in-Human Gene Therapy Clinical Trials -- 3 Understanding the Complexities of Patient Selection, Enrollment, and the Consent Process -- 4 Developing Endpoints for Gene Therapy Clinical Trials -- 5 Integrating Gene-Based Therapies into Clinical Practice: Exploring Long-Term Clinical Follow-Up of Patients -- 6 Reflections on the Workshop and Potential Opportunities for Next Steps -- References |
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Appendix A: Workshop Agenda -- Appendix B: Speaker Biographical Sketches -- Appendix C: Statement of Task -- Appendix D: Registered Attendees |
| Summary |
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 |
| Bibliography |
Includes bibliographical references |
| Notes |
This activity was supported by contracts between the National Academy of Sciences and Advanced Regenerative Manufacturing Institute; Akron Biotech; Alliance for Regenerative Medicine; American Society of Gene Cell Therapy; Burroughs Wellcome Fund; California Institute for Regenerative Medicine; Centre for Commercialization of Regenerative Medicine; Department of Veterans Affairs (Contract No. VA268-16-C-0051); Food and Drug Administration (Grant #1R13FD0066--14-01); GE Healthcare; International Society for Cell Gene Therapy; International Society for Stem Cell Research; Johnson Johnson; The Michael J. Fox Foundation for Parkinson's Research; National Institute of Standards and Technology; National Institutes of Health: National Institute of Dental and Craniofacial Research (Contract No. HHSN263201800029I; Order No. 75N98019F00847), National Institute of Diabetes and Digestive and Kidney Diseases (PO No. 75N94019P00304); The New York Stem Cell Foundation; Parkinson's Foundation; Sanofi (Contract No. 55630791); and United Therapeutics Corporation (Contract No. 10003921). Any opinions, findings, conclusions, or recommendations expressed in this publication do not necessarily reflect the views of any organization or agency that provided support for the project |
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Online resource; title from PDF title page (viewed November 10, 2020) |
| Subject |
Gene therapy.
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Regenerative medicine -- Congresses
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Genetic Therapy
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Clinical Trials as Topic
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Gene therapy
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Regenerative medicine
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United States |
| Genre/Form |
proceedings (reports)
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Conference papers and proceedings
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Conference papers and proceedings.
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Actes de congrès.
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| Form |
Electronic book
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| Author |
Hackmann, Meredith, rapporteur
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Alper, Joe, rapporteur
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Beachy, Sarah H., rapporteur
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National Academies of Sciences, Engineering, and Medicine (U.S.). Forum on Regenerative Medicine, sponsoring body.
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Exploring Novel Clinical Trial Designs for Gene-Based Therapies: a Workshop (2019 : Washington, D.C.)
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| ISBN |
9780309672993 |
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0309672996 |
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9780309673013 |
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0309673011 |
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