A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent study
- Conditions
- Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP)MedDRA version: 20.1Level: LLTClassification code 10008791Term: ChoroideremiaSystem Organ Class: 100000004853MedDRA version: 20.0Level: PTClassification code 10038914Term: Retinitis pigmentosaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Eye Diseases [C11]
- Registration Number
- EUCTR2017-003104-42-FI
- Lead Sponsor
- ightstaRx Ltd (A Biogen Company)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Male
- Target Recruitment
- 440
CHM Participants
a. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of BIIB111 for CHM.
XLRP Participants
a. Have received a sub-retinal injection of BIIB112 for XLRP and have exited an antecedent study.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 440
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Participants are not eligible for study participation if they meet
the following exclusion criterion.
a. In the opinion of the investigator and/or the Sponsor, it is not in the participant’s best interest to participate in the study.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method