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Gene Therapy for Achromatopsia (CNGA3) (CNGA3)

Primary Purpose

Achromatopsia

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
adeno-associated virus vector AAV- CNGA3
Sponsored by
MeiraGTx UK II Ltd
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Achromatopsia

Eligibility Criteria

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

Inclusion Criteria:

  • Are aged years or over
  • Have achromatopsia confirmed by a retinal specialist investigator

Exclusion Criteria:

  • Are females who are pregnant or breastfeeding
  • Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
  • Have any other condition that the investigator considers makes them inappropriate for entry into the trial

Sites / Locations

  • Kellogg Eye Center
  • Moorfields Eye Hospital NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

Low dose adeno-associated virus (AAV) CNGA3

Intermediate dose adeno-associated virus (AAV) CNGA3

High dose adeno-associated virus (AAV) CNGA3

Arm Description

Subretinal administration of a single low dose AAV CNGA3

Subretinal administration of a single intermediate dose AAV CNGA3

Subretinal administration of a single high dose AAV CNGA3

Outcomes

Primary Outcome Measures

Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone.
The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone: Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences Severe unresponsive inflammation Infective endophthalmitis Ocular malignancy Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)

Secondary Outcome Measures

Improvements in Visual Function as Assessed by Visual Acuity
Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time.
Improvements in Retinal Function as Assessed by Static Perimetry
Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.
Quality of Life Measured by QoL Questionnaires in Children and Adolescents
Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.
Quality of Life Measured by QoL Questionnaires in Adults
Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.

Full Information

First Posted
November 27, 2018
Last Updated
November 3, 2022
Sponsor
MeiraGTx UK II Ltd
Collaborators
Janssen Research & Development, LLC
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1. Study Identification

Unique Protocol Identification Number
NCT03758404
Brief Title
Gene Therapy for Achromatopsia (CNGA3)
Acronym
CNGA3
Official Title
An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Children and Adults With Achromatopsia Owing to Defects in CNGA3
Study Type
Interventional

2. Study Status

Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
August 12, 2019 (Actual)
Primary Completion Date
June 10, 2021 (Actual)
Study Completion Date
June 10, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MeiraGTx UK II Ltd
Collaborators
Janssen Research & Development, LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia
Detailed Description
CNGA3 retinal gene therapy for patients with achromatopsia

6. Conditions and Keywords

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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Low dose adeno-associated virus (AAV) CNGA3
Arm Type
Experimental
Arm Description
Subretinal administration of a single low dose AAV CNGA3
Arm Title
Intermediate dose adeno-associated virus (AAV) CNGA3
Arm Type
Experimental
Arm Description
Subretinal administration of a single intermediate dose AAV CNGA3
Arm Title
High dose adeno-associated virus (AAV) CNGA3
Arm Type
Experimental
Arm Description
Subretinal administration of a single high dose AAV CNGA3
Intervention Type
Biological
Intervention Name(s)
adeno-associated virus vector AAV- CNGA3
Intervention Description
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene
Primary Outcome Measure Information:
Title
Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone.
Description
The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone: Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences Severe unresponsive inflammation Infective endophthalmitis Ocular malignancy Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)
Time Frame
6 Weeks
Secondary Outcome Measure Information:
Title
Improvements in Visual Function as Assessed by Visual Acuity
Description
Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time.
Time Frame
6 Months
Title
Improvements in Retinal Function as Assessed by Static Perimetry
Description
Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.
Time Frame
6 Months
Title
Quality of Life Measured by QoL Questionnaires in Children and Adolescents
Description
Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.
Time Frame
6 Months
Title
Quality of Life Measured by QoL Questionnaires in Adults
Description
Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.
Time Frame
6 Months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Are aged years or over Have achromatopsia confirmed by a retinal specialist investigator Exclusion Criteria: Are females who are pregnant or breastfeeding Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months Have any other condition that the investigator considers makes them inappropriate for entry into the trial
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
James Bainbridge
Organizational Affiliation
Chief Investigator
Official's Role
Principal Investigator
Facility Information:
Facility Name
Kellogg 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
ZIP/Postal Code
EC1V 2PD
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

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Gene Therapy for Achromatopsia (CNGA3)

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