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
- MedDRA version: 20.1Level: LLTClassification code 10008791Term: ChoroideremiaSystem Organ Class: 100000004853Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP)Therapeutic area: Diseases [C] - Eye Diseases [C11]MedDRA version: 20.0Level: PTClassification code 10038914Term: Retinitis pigmentosaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
- Registration Number
- EUCTR2017-003104-42-GB
- Lead Sponsor
- ightstaRx Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Male
- Target Recruitment
- 440
CHM Subjects:
a. Are willing and able to give informed consent for participation in the study, and
b. Have received a sub-retinal injection of AAV2-REP1 for CHM and have exited an antecedent study
XLRP Subjects:
a. Are willing and able to give informed consent (or legal guardian consent and subject assent, if applicable) 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? yes
Number of subjects for this age range: 9
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 167
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 subject’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 Main Objective: To evaluate the long-term safety and efficacy of a sub retinal injection of:<br>• AAV2-REP1 in subjects with choroideremia (CHM) who have been previously treated with AAV2-REP1 and who have exited an antecedent study.<br>• AAV8-RPGR in subjects with X-linked retinitis pigmentosa (XLRP) who have been previously treated with AAV8-RPGR and who have exited an antecedent study.;Secondary Objective: Not applicable;Primary end point(s): Long-term safety of AAV2-REP1 and AAV8-RPGR;Timepoint(s) of evaluation of this end point: Safety assessments will be performed as per protocol
- Secondary Outcome Measures
Name Time Method Secondary end point(s): • Change from Baseline in best-corrected visual acuity (BCVA) as measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart<br>• Change from Baseline in fundus autofluorescence<br>• Change from Baseline in fundus photography<br>• Change from Baseline in spectral-domain optical coherence tomography (SD-OCT)<br>• Change from Baseline in microperimetry<br>• Change from Baseline in the 25-Item Visual Function Questionnaire (VFQ-25)<br>• Change from Baseline in visual field (in AAV8-RPGR-treated subjects only);Timepoint(s) of evaluation of this end point: Visual assessments will be performed as per protocol