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
Overview
- Phase
- Phase 1
- Intervention
- Prior exposure to AAV-CNGA3 or AAV-CNGB3
- Conditions
- Achromatopsia
- Sponsor
- MeiraGTx UK II Ltd
- Enrollment
- 34
- Locations
- 2
- Primary Endpoint
- Incidence of Adverse Events Related to the Treatment
- Status
- Terminated
- Last Updated
- 10 months ago
Overview
Brief Summary
This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia associated with defects in CNGB3 who participated in a clinical trial in which they received AAV-CNGB3 retinal gene therapy.
Detailed Description
The follow-up study is designed to collect data on the longer-term safety and efficacy of AAV-CNGA3 retinal gene therapy and AAV-CNGB3 retinal gene therapy.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Low dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single low dose of AAV-CNGA3 or AAV-CNGB3
Intervention: Prior exposure to AAV-CNGA3 or AAV-CNGB3
Intermediate dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single intermediate dose of AAV-CNGA3 or AAV-CNGB3
Intervention: Prior exposure to AAV-CNGA3 or AAV-CNGB3
Other dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single other dose (between the intermediate and high dose) of AAV-CNGA3 or AAV-CNGB3
Intervention: Prior exposure to AAV-CNGA3 or AAV-CNGB3
High dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single high dose of AAV-CNGA3 or AAV-CNGB3
Intervention: Prior exposure to AAV-CNGA3 or AAV-CNGB3
Outcomes
Primary Outcomes
Incidence of Adverse Events Related to the Treatment
Time Frame: 5 Years
The primary outcome measure is the longer-term safety of treatment with AAV-CNGA3 or AAV-CNGB3, assessed by the absence of IMP-related adverse events.
Secondary Outcomes
- Improvements in Visual Function as Assessed by Visual Acuity at Month 12(12 months)
- Improvements in Visual Function as Assessed by Visual Acuity at Month 60(60 months)
- Improvements in Retinal Function as Assessed by Static Perimetry at Month 12(12 months)
- Improvements in Retinal Function as Assessed by Static Perimetry at Month 60(60 months)
- Quality of Life at Month 12 Measured by QoL Questionnaires in Children and Adolescents(12 months)
- Quality of Life at Month 60 Measured by QoL Questionnaires in Children and Adolescents(60 months)
- Quality of Life at Month 12 Measured by QoL Questionnaires in Adults(12 months)
- Quality of Life at Month 60 Measured by QoL Questionnaires in Adults(60 months)