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

Primary Purpose

Achromatopsia

Status

Active

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

either AAV - CNGB3 or AAV - CNGA3

About this trial

This is an interventional treatment trial for Achromatopsia focused on measuring Achromatopsia, CNGB3, CNGA3

Eligibility Criteria

3 Years - 100 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 with the requirements of the study

Sites / Locations

Kellog Eye Center
Moorfields Eye Hospital NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3

Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3

Biological-high dose of either AAV - CNGB3 or AAV - CNGA3

Arm Description

Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3

Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3

Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3

Outcomes

Primary Outcome Measures

Incidence of Adverse Events related to the treatment

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

Secondary Outcome Measures

Improvement in the visual function

Improvements in visual function as assessed by visual assessment

Improvement in retinal function

Improvement in the retinal function as assessed by visual assessment

Improvement in quality of life

Quality of life will be measured by QoL questionnaires

Full Information

NCT ID

NCT03278873

First Posted

August 16, 2017

Last Updated

February 13, 2023

Sponsor

MeiraGTx UK II Ltd

Collaborators

Syne Qua Non Limited, EMAS Pharma

1. Study Identification

Unique Protocol Identification Number

NCT03278873

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

June 29, 2017 (Actual)

Primary Completion Date

January 15, 2026 (Anticipated)

Study Completion Date

January 15, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

MeiraGTx UK II Ltd

Collaborators

Syne Qua Non Limited, EMAS Pharma

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. 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.

Detailed Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Achromatopsia

Keywords

Achromatopsia, CNGB3, CNGA3

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

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"

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

34 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3

Arm Type

Experimental

Arm Description

Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3

Arm Title

Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3

Arm Type

Experimental

Arm Description

Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3

Arm Title

Biological-high dose of either AAV - CNGB3 or AAV - CNGA3

Arm Type

Experimental

Arm Description

Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3

Intervention Type

Biological

Intervention Name(s)

either AAV - CNGB3 or AAV - CNGA3

Intervention Description

Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3

Primary Outcome Measure Information:

Title

Incidence of Adverse Events related to the treatment

Description

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

Time Frame

5 Years

Secondary Outcome Measure Information:

Title

Improvement in the visual function

Description

Improvements in visual function as assessed by visual assessment

Time Frame

5 years

Title

Improvement in retinal function

Description

Improvement in the retinal function as assessed by visual assessment

Time Frame

5 years

Title

Improvement in quality of life

Description

Quality of life will be measured by QoL questionnaires

Time Frame

5 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Years

Maximum Age & Unit of Time

100 Years

Accepts Healthy Volunteers

Eligibility Criteria

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 with the requirements of the study

Facility Information:

Facility Name

Kellog Eye Center

City

Ann Arbor

State/Province

Michigan

ZIP/Postal Code

48105

Country

United States

Facility Name

Moorfields Eye Hospital NHS Foundation Trust

City

London

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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