Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
- Conditions
- Achromatopsia
- Interventions
- Biological: Prior exposure to AAV-CNGA3 or AAV-CNGB3
- Registration Number
- NCT03278873
- Lead Sponsor
- MeiraGTx UK II Ltd
- 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 34
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Low dose of AAV-CNGA3 or AAV-CNGB3 Prior exposure to AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single low dose of AAV-CNGA3 or AAV-CNGB3 Intermediate dose of AAV-CNGA3 or AAV-CNGB3 Prior exposure to AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single intermediate dose of AAV-CNGA3 or AAV-CNGB3 Other dose of AAV-CNGA3 or AAV-CNGB3 Prior exposure to AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single other dose (between the intermediate and high dose) of AAV-CNGA3 or AAV-CNGB3 High dose of AAV-CNGA3 or AAV-CNGB3 Prior exposure to AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single high dose of AAV-CNGA3 or AAV-CNGB3
- Primary Outcome Measures
Name Time Method Incidence of Adverse Events Related to the Treatment 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 Outcome Measures
Name Time Method Improvements in Retinal Function as Assessed by Static Perimetry at Month 12 12 months Change from baseline to Month 12 in contrast sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.
Improvements in Retinal Function as Assessed by Static Perimetry at Month 60 60 months Change from baseline to Month 60 in contrast sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.
Quality of Life at Month 12 Measured by QoL Questionnaires in Children and Adolescents 12 months Change from baseline to Month 12 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 at Month 60 Measured by QoL Questionnaires in Children and Adolescents 60 months Change from baseline to Month 60 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 at Month 12 Measured by QoL Questionnaires in Adults 12 months Change from baseline to Month 12 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.
Improvements in Visual Function as Assessed by Visual Acuity at Month 12 12 months Change from baseline to Month 12 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 Visual Function as Assessed by Visual Acuity at Month 60 60 months Change from baseline to Month 60 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.
Quality of Life at Month 60 Measured by QoL Questionnaires in Adults 60 months Change from baseline to Month 60 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.
Trial Locations
- Locations (2)
Kellogg Eye Center
🇺🇸Ann Arbor, Michigan, United States
Moorfields Eye Hospital NHS Foundation Trust
🇬🇧London, United Kingdom
Kellogg Eye Center🇺🇸Ann Arbor, Michigan, United States