Gene Therapy for Achromatopsia (CNGA3)
- Conditions
- Achromatopsia
- Interventions
- Biological: adeno-associated virus vector AAV- CNGA3
- Registration Number
- NCT03758404
- Lead Sponsor
- MeiraGTx UK II Ltd
- 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
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 11
- Are aged years or over
- Have achromatopsia confirmed by a retinal specialist investigator
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Low dose adeno-associated virus (AAV) CNGA3 adeno-associated virus vector AAV- CNGA3 Subretinal administration of a single low dose AAV CNGA3 High dose adeno-associated virus (AAV) CNGA3 adeno-associated virus vector AAV- CNGA3 Subretinal administration of a single high dose AAV CNGA3 Intermediate dose adeno-associated virus (AAV) CNGA3 adeno-associated virus vector AAV- CNGA3 Subretinal administration of a single intermediate dose AAV CNGA3
- Primary Outcome Measures
Name Time Method 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. 6 Weeks 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
Name Time Method Improvements in Visual Function as Assessed by Visual Acuity 6 Months 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.
Quality of Life Measured by QoL Questionnaires in Children and Adolescents 6 Months 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.
Improvements in Retinal Function as Assessed by Static Perimetry 6 Months 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 Adults 6 Months 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.
Trial Locations
- Locations (2)
Kellogg Eye Center
🇺🇸Ann Arbor, Michigan, United States
Moorfields Eye Hospital NHS Foundation Trust
🇬🇧London, United Kingdom