Efficacy and Safety of BIIB111 for the Treatment of Choroideremia

Summary

The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM).

Timeline

Start

2017-12-11

Primary completion

2020-12-01

Completion

2020-12-01

Outcome

Missed primary endpoint

registry analysis (superiority test); Untreated Control Group vs BIIB111 Low Dose; p =0.354 NCT03496012 ↗

registry analysis (superiority test); Untreated Control Group vs BIIB111 High Dose; p =0.245 NCT03496012 ↗

Publications

• 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	BIIB111	Gene therapy (AAV / viral vector)	—	Other

Indications