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-DK
- 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
- 330
CHM Participants:
a. Are willing and able to give informed consent for participation in the study, and
b. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of AAV2-REP1 for CHM
XLRP Participants:
a. Are willing and able to give informed consent for participation in the study
b. Have received a sub-retinal injection of AAV8-RPGR 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 330
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
In the opinion of the investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study.
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