Clinical trials: Choroideremia
Choroideremia, CHM (Choroideremia) Gene Mutations
Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
This clinical study evaluates the safety and tolerability of AAV2-hCHM in participants with Choroideremia gene mutations.
- Trial name
- NCT02341807
- Testing
- AAV2-hCHM
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Spark Therapeutics, Inc.
Choroideremia
A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111
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).
- Trial name
- GEMINI (NCT03507686)
- Testing
- BIIB111
- Where
- 6 sites in United States, France and 1 more
- Volunteers
- 66 people
- Sponsor
- Biogen
Choroideremia
Efficacy and Safety of BIIB111 for the Treatment of Choroideremia
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).
- Trial name
- STAR (NCT03496012)
- Testing
- BIIB111
- Where
- 18 sites in United States, Canada and 6 more
- Volunteers
- 169 people
- Sponsor
- Biogen
Choroideremia
Choroideremia Gene Therapy Clinical Trial
Phase II gene therapy study, involving a total of 6 male patients with choroideremia. The study will be conducted at the Bascom Palmer Eye Institute, University of Miami.
- Trial name
- NCT02553135
- Testing
- Injection of AAV2-REP1 (10e11 vg)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Byron Lam