drugset / Trial / NCT03507686
A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111
NaSingle-groupOpen-labelTreatment
Summary
The objective of the study is to evaluate the safety of bilateral, sequential sub-retinal administration of a single dose of BIIB111 in adult male participants with Choroideremia (CHM).
Timeline
- Start
- 2017-11-29
- Primary completion
- 2022-06-29
- Completion
- 2022-06-29
Publications
- MacLaren RE, Audo I, Fischer MD, Huckfeldt RM, Lam BL, Pennesi ME, Sisk R, Gow JA, Li J, Zhu K, Tsang SF. An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study. Hum Gene Ther. 2024 Aug;35(15-16):564-575. doi: 10.1089/hum.2024.017. Epub 2024 Jul 27.
- Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV2-REP1 | Gene therapy (AAV / viral vector) | 0.1 ml | Other |
| Subject | AAV2-REP1 | Gene therapy (AAV / viral vector) | 1e+11 vg | Other |