A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated in an antecedent study
Phase 1
- Conditions
- Choroideremia (CHM)Therapeutic area: Diseases [C] - Eye Diseases [C11]
- Registration Number
- EUCTR2017-003104-42-NL
- 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
- 167
Inclusion Criteria
• Are willing and able to give informed consent for participation in the study, and
• Have received a sub-retinal injection of AAV2-REP1 for CHM 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 167
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion Criteria
Subjects 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 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 AAV2-REP1 in subjects with choroideremia who have been treated previously with AAV2-REP1 and who have exited an antecedent study.;Secondary Objective: Not applicable;Primary end point(s): The primary endpoint of this study is safety of AAV2-REP1, which will be evaluated through adverse event (AE) reporting, full ophthalmic examinations, and laboratory assessments of immunogenicity.;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): Baseline is defined as the last available measurement prior to administration of AAV2-REP1 for the treated eye(s) and the associated untreated eye(s), if applicable.<br>The following are both efficacy and safety assessments.<br>• 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);Timepoint(s) of evaluation of this end point: Visual assessments will be performed as per protocol