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

Achromatopsia Clinical Trial

Official title:

Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3 and AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3 or CNGA3

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03278873
• Other study ID #: MGT007
• Secondary ID: 2016-003856-59
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: June 29, 2017
• Est. completion date: January 15, 2026

Study information

• Verified date: March 2024
• Source: MeiraGTx UK II Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 34
• Est. completion date: January 15, 2026
• Est. primary completion date: January 15, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 100 Years

Eligibility

Inclusion in the study will be limited to individuals who:

1. Are able to give informed consent or assent, with or without the guidance of their parent(s)/guardian(s) where appropriate

2. 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)

3. Are willing to adhere to the protocol and long-term follow-up Individuals will be excluded who: Are unwilling or unable to meet with the requirements of the study

Related Conditions & MeSH terms

• Achromatopsia
• Color Vision Defects

Intervention

Biological:
• either AAV - CNGB3 or AAV - CNGA3
Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3

Locations

Country	Name	City	State
United Kingdom	Moorfields Eye Hospital NHS Foundation Trust	London
United States	Kellog Eye Center	Ann Arbor	Michigan

Sponsors (3)

Lead Sponsor	Collaborator
MeiraGTx UK II Ltd	EMAS Pharma, Syne Qua Non Limited

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Adverse Events related to the treatment	longer term safety as the absence of ATIMP-related safety events	5 Years
Secondary	Improvement in the visual function	Improvements in visual function as assessed by visual assessment	5 years
Secondary	Improvement in retinal function	Improvement in the retinal function as assessed by visual assessment	5 years
Secondary	Improvement in quality of life	Quality of life will be measured by QoL questionnaires	5 years