Gene Therapy for Achromatopsia (CNGB3) (CNGB3)
Primary Purpose
Achromatopsia
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
AAV - CNGB3
Sponsored by
About this trial
This is an interventional treatment trial for Achromatopsia
Eligibility Criteria
Inclusion Criteria:
- Are aged 3 years or older
- Have achromatopsia confirmed by a retinal specialist (CI or PI)
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 CI/PI considers makes them inappropriate for entry into the trial
Sites / Locations
- University of Michigan Kellog Eye Centre
- Moorfields Eye Hospital NHS Foundation Trust
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
Biological-Low dose AAV - CNGB3
Biological-Medium dose AAV - CNGB3
Biological-High dose AAV - CNGB3
Arm Description
Subretinal administration of a single low dose of AAV - CNGB3
Subretinal administration of a single intermediate dose of AAV - CNGB3
Subretinal administration of a single high dose of AAV - CNGB3
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 in the treated eye. 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-5D-Y 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 change from baseline reflects worsening.
Quality of Life Measured by QoL Questionnaires in Adults
Change from baseline to Week 24 in EuroQol-5D-5L 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 change from baseline reflects worsening.
Full Information
NCT ID
NCT03001310
First Posted
November 22, 2016
Last Updated
February 9, 2023
Sponsor
MeiraGTx UK II Ltd
Collaborators
Janssen Research & Development, LLC
1. Study Identification
Unique Protocol Identification Number
NCT03001310
Brief Title
Gene Therapy for Achromatopsia (CNGB3)
Acronym
CNGB3
Official Title
An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Completed
Study Start Date
January 16, 2017 (Actual)
Primary Completion Date
October 25, 2019 (Actual)
Study Completion Date
October 25, 2019 (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 AAV - CNGB3 retinal gene therapy for patients with achromatopsia
Detailed Description
CNGB3 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
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
23 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Biological-Low dose AAV - CNGB3
Arm Type
Experimental
Arm Description
Subretinal administration of a single low dose of AAV - CNGB3
Arm Title
Biological-Medium dose AAV - CNGB3
Arm Type
Experimental
Arm Description
Subretinal administration of a single intermediate dose of AAV - CNGB3
Arm Title
Biological-High dose AAV - CNGB3
Arm Type
Experimental
Arm Description
Subretinal administration of a single high dose of AAV - CNGB3
Intervention Type
Biological
Intervention Name(s)
AAV - CNGB3
Intervention Description
Comparison of different dosages of AAV-CNGB3
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 in the treated eye. 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-5D-Y 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 change from baseline 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-5D-5L 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 change from baseline 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 3 years or older
Have achromatopsia confirmed by a retinal specialist (CI or PI)
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 CI/PI considers makes them inappropriate for entry into the trial
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
James Bainbridge, Prof
Organizational Affiliation
Chief Investigator
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Michigan Kellog Eye Centre
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
MI 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
No
Learn more about this trial
Gene Therapy for Achromatopsia (CNGB3)
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