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Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page 77
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page 78
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page 79
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
Page 80
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
Page 81
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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82 NOVEL CLINICAL TRIAL DESIGNS FOR GENE-BASED THERAPIES Verhaart, I. E. C., A. Robertson, I. J. Wilson, A. Aartsma-Rus, S. Cameron, C. C. Jones, S. F. Cook, and H. Lochmüller. 2017. Prevalence, incidence and carrier frequency of 5q- linked spinal muscular atrophy—A literature review. Orphanet Journal of Rare Diseases 12(1):124. Wang, R. T., F. Barthelemy, A. S. Martin, E. D. Douine, A. Eskin, A. Lucas, J. Lavigne, H. Peay, N. Khanlou, L. Sweeney, R. M. Cantor, M. C. Miceli, and S. F. Nelson. 2018. DMD genotype correlations from the Duchenne registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype. Human Mutation 39(9):1193–1202. Wendler, D., L. Belsky, K. M. Thompson, and E. J. Emanuel. 2005. Quantifying the federal minimal risk standard: Implications for pediatric research without a prospect of direct benefit. JAMA 294(7):826–832. Xu, L., I. Irony, W. W. Bryan, and B. Dunn. 2017. Development of gene therapies-lessons from nusinersen. Gene Therapy 24(9):527–528. Zarin, D. A., S. N. Goodman, and J. Kimmelman. 2019. Harms from uninformative clinical trials. JAMA 322(9):813–814. PREPUBLICATION COPY—Uncorrected Proofs

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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.

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