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Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium.

Publication ,  Journal Article
Thompson, DA; Iannaccone, A; Ali, RR; Arshavsky, VY; Audo, I; Bainbridge, JWB; Besirli, CG; Birch, DG; Branham, KE; Cideciyan, AV; Daiger, SP ...
Published in: Transl Vis Sci Technol
June 2020

Major advances in the study of inherited retinal diseases (IRDs) have placed efforts to develop treatments for these blinding conditions at the forefront of the emerging field of precision medicine. As a result, the growth of clinical trials for IRDs has increased rapidly over the past decade and is expected to further accelerate as more therapeutic possibilities emerge and qualified participants are identified. Although guided by established principles, these specialized trials, requiring analysis of novel outcome measures and endpoints in small patient populations, present multiple challenges relative to study design and ethical considerations. This position paper reviews recent accomplishments and existing challenges in clinical trials for IRDs and presents a set of recommendations aimed at rapidly advancing future progress. The goal is to stimulate discussions among researchers, funding agencies, industry, and policy makers that will further the design, conduct, and analysis of clinical trials needed to accelerate the approval of effective treatments for IRDs, while promoting advocacy and ensuring patient safety.

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Published In

Transl Vis Sci Technol

DOI

ISSN

2164-2591

Publication Date

June 2020

Volume

9

Issue

7

Start / End Page

2

Location

United States

Related Subject Headings

  • Retinal Diseases
  • Retina
  • Precision Medicine
  • Humans
  • 3212 Ophthalmology and optometry
  • 1113 Opthalmology and Optometry
  • 0903 Biomedical Engineering
 

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Thompson, D. A., Iannaccone, A., Ali, R. R., Arshavsky, V. Y., Audo, I., Bainbridge, J. W. B., … Monaciano Consortium, . (2020). Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium. Transl Vis Sci Technol, 9(7), 2. https://doi.org/10.1167/tvst.9.7.2
Thompson, Debra A., Alessandro Iannaccone, Robin R. Ali, Vadim Y. Arshavsky, Isabelle Audo, James W. B. Bainbridge, Cagri G. Besirli, et al. “Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium.Transl Vis Sci Technol 9, no. 7 (June 2020): 2. https://doi.org/10.1167/tvst.9.7.2.
Thompson DA, Iannaccone A, Ali RR, Arshavsky VY, Audo I, Bainbridge JWB, et al. Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium. Transl Vis Sci Technol. 2020 Jun;9(7):2.
Thompson, Debra A., et al. “Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium.Transl Vis Sci Technol, vol. 9, no. 7, June 2020, p. 2. Pubmed, doi:10.1167/tvst.9.7.2.
Thompson DA, Iannaccone A, Ali RR, Arshavsky VY, Audo I, Bainbridge JWB, Besirli CG, Birch DG, Branham KE, Cideciyan AV, Daiger SP, Dalkara D, Duncan JL, Fahim AT, Flannery JG, Gattegna R, Heckenlively JR, Heon E, Jayasundera KT, Khan NW, Klassen H, Leroy BP, Molday RS, Musch DC, Pennesi ME, Petersen-Jones SM, Pierce EA, Rao RC, Reh TA, Sahel JA, Sharon D, Sieving PA, Strettoi E, Yang P, Zacks DN, Monaciano Consortium. Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium. Transl Vis Sci Technol. 2020 Jun;9(7):2.

Published In

Transl Vis Sci Technol

DOI

ISSN

2164-2591

Publication Date

June 2020

Volume

9

Issue

7

Start / End Page

2

Location

United States

Related Subject Headings

  • Retinal Diseases
  • Retina
  • Precision Medicine
  • Humans
  • 3212 Ophthalmology and optometry
  • 1113 Opthalmology and Optometry
  • 0903 Biomedical Engineering