Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3

Phase 1

Completed

• Conditions: Achromatopsia
• Interventions: Biological: either AAV - CNGB3 or AAV - CNGA3

• Registration Number: NCT03278873
• Lead Sponsor: MeiraGTx UK II Ltd

Brief Summary

This study is a longer-term follow-up study for patients who participated in one of the clinical trials: AAV - CNGB3 retinal gene therapy for patients with achromatopsia, or AAV - CNGA3 retinal gene therapy for patients with achromatopsia.

Detailed Description

The follow up study is designed to collect data on longer-term safety and efficacy.

Study Timeline

• Study Start: 2017-06-29
• Study First Submitted: 2017-08-16
• Study First Submitted QC: 2017-09-07
• Study First Posted: 2017-09-12
• Primary Completion: 2024-04-04
• Study Completion: 2024-04-04
• Disposition First Posted: 2024-11-21
• Status Verified: 2025-03
• Disposition First Submitted: 2025-03-31
• Last Update Submitted: 2025-03-31
• Last Update Posted: 2025-04-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 34

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3	either AAV - CNGB3 or AAV - CNGA3	Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3
Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3	either AAV - CNGB3 or AAV - CNGA3	Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3
Biological-high dose of either AAV - CNGB3 or AAV - CNGA3	either AAV - CNGB3 or AAV - CNGA3	Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3

Primary Outcome Measures

Name	Time	Method
Incidence of Adverse Events related to the treatment	5 Years	longer term safety as the absence of ATIMP-related safety events

Secondary Outcome Measures

Name	Time	Method
Improvement in retinal function	5 years	Improvement in the retinal function as assessed by visual assessment
Improvement in the visual function	5 years	Improvements in visual function as assessed by visual assessment
Improvement in quality of life	5 years	Quality of life will be measured by QoL questionnaires

Trial Locations

Locations (2)

Kellog Eye Center; 🇺🇸 Ann Arbor, Michigan, United States

Moorfields Eye Hospital NHS Foundation Trust; 🇬🇧 London, United Kingdom