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

• Sponsor: MeiraGTx UK II Ltd (Industry)
• Overall Status: Active, not recruiting
• CT.gov ID: NCT03278873
• Collaborator: Syne Qua Non Limited (Industry), EMAS Pharma (Industry)
• Enrollment: 34
• Locations: 2
• Arms: 3
• Anticipated Duration (Months): 102.6
• Patients Per Site: 17
• Patients Per Site Per Month: 0.2

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Achromatopsia	Biological: either AAV - CNGB3 or AAV - CNGA3	Phase 1/Phase 2

Detailed Description

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

Study Design

• Study Type: Interventional
• Actual Enrollment: 34 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Intervention Model Description: Follow-up of patients from two separate trials but there is no difference in follow-up assessments for the patients therefore they can be considered a "single group"Follow-up of patients from two separate trials but there is no difference in follow-up assessments for the patients therefore they can be considered a "single group"
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: Jun 29, 2017
• Anticipated Primary Completion Date: Jan 15, 2026
• Anticipated Study Completion Date: Jan 15, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3 Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3	Biological: either AAV - CNGB3 or AAV - CNGA3; Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3
Experimental: Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3 Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3	Biological: either AAV - CNGB3 or AAV - CNGA3; Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3
Experimental: Biological-high dose of either AAV - CNGB3 or AAV - CNGA3 Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3	Biological: either AAV - CNGB3 or AAV - CNGA3; Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3

Outcome Measures

Primary Outcome Measures

1. Incidence of Adverse Events related to the treatment [5 Years]

   longer term safety as the absence of ATIMP-related safety events

Secondary Outcome Measures

1. Improvement in the visual function [5 years]

   Improvements in visual function as assessed by visual assessment

2. Improvement in retinal function [5 years]

   Improvement in the retinal function as assessed by visual assessment

3. Improvement in quality of life [5 years]

   Quality of life will be measured by QoL questionnaires

Eligibility Criteria

Criteria

• Ages Eligible for Study: 3 Years to 100 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Kellog Eye Center	Ann Arbor	Michigan	United States	48105
2	Moorfields Eye Hospital NHS Foundation Trust	London		United Kingdom

Sponsors and Collaborators

• MeiraGTx UK II Ltd
• Syne Qua Non Limited
• EMAS Pharma

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: MeiraGTx UK II Ltd
• ClinicalTrials.gov Identifier: NCT03278873
• Other Study ID Numbers: MGT007; 2016-003856-59
• First Posted: Sep 12, 2017
• Last Update Posted: Jan 14, 2022
• Last Verified: Jan 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by MeiraGTx UK II Ltd: Achromatopsia; CNGB3; CNGA3
• Additional relevant MeSH terms: Color Vision Defects

Study Results

No Results Posted as of Jan 14, 2022