Status:
TERMINATED
Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
Lead Sponsor:
MeiraGTx UK II Ltd
Conditions:
Achromatopsia
Eligibility:
All Genders
3-100 years
Brief Summary
This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of ...
Detailed Description
The follow-up study is designed to collect data on the longer-term safety and efficacy of AAV-CNGA3 retinal gene therapy and AAV-CNGB3 retinal gene therapy.
Eligibility Criteria
Inclusion
- Inclusion in the study will be limited to individuals who:
- Are able to give informed consent or assent, with or without the guidance of their parent(s)/guardian(s), where appropriate
- Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or EudraCT 2018-003431-29)
- Are willing to adhere to the protocol and long-term follow-up
- Individuals will be excluded who:
- Are unwilling or unable to meet the requirements of the study
Exclusion
Key Trial Info
Start Date :
June 29 2017
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
April 4 2024
Estimated Enrollment :
34 Patients enrolled
Trial Details
Trial ID
NCT03278873
Start Date
June 29 2017
End Date
April 4 2024
Last Update
June 11 2025
Active Locations (2)
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1
Kellogg Eye Center
Ann Arbor, Michigan, United States, 48105
2
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom