A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Treated Previously with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study
- Conditions
- 10047060choroideremia (CHM)retinal degeneration
- Registration Number
- NL-OMON54647
- Lead Sponsor
- ightstaRx Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 8
Inclusion Criteria - Participants are eligible for study participation if they
meet all of the following inclusion criteria:
CHM Participants:
a. Are willing and able to give informed consent for participation in the study
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
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.
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary endpoint of this study is safety of AAV2-REP1 and AAV8-RPGR, which<br /><br>will be evaluated through adverse event (AE) reporting and full ophthalmic<br /><br>examinations.</p><br>
- Secondary Outcome Measures
Name Time Method