Title
Gene Therapy for Achromatopsia (CNGB3)
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
Phase
Phase 1/Phase 2Lead Sponsor
MeiraGTxStudy Type
InterventionalStatus
Completed Results PostedIndication/Condition
AchromatopsiaIntervention/Treatment
AAV - CNGB3Study Participants
23A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia
CNGB3 retinal gene therapy for patients with achromatopsia
Comparison of different dosages of AAV-CNGB3
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
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
| Event Type | Organ System | Event Term | Low Dose AAV - CNGB3 | Intermediate Dose AAV-CNGB3 | Other Dose - AAV - CNGB3 | High Dose AAV - CNGB3 | Total |
|---|
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)
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.
Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.
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.
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.
