Achromatopsia caused by mutations in the CNGA3 gene MedDRA version: 20.0 Level: LLT Classification code 10000454 Term: Achromatopsia System Organ Class: 100000004850
Conditions
Interventions
Product Name: AAV2/8-hG1.7p.coCNGA3
Pharmaceutical Form: Solution for injection
Concentration unit: vector genomes (vg)/mL
Concentration type: not less
Sponsors
MeiraGTx UK II Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Key Inclusion Criteria: • Aged 3 to 15 years old • Achromatopsia caused by mutations in CNGA3 • Evidence of preservation of photoreceptors at the macula • Able to undertake age-appropriate clinical assessments • Willing to give consent for the use of blood and blood components collected throughout the trial for the investigation of immune response to Advanced Therapy Investigational Product (ATIMP). Are the trial subjects under 18? yes Number of subjects for this age range: 36 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Key Exclusion Criteria: • Female adolescents who are pregnant or breastfeeding • Intra-ocular surgery within 6 months of screening Ocular or systemic disorder that may preclude subretinal surgery and/or interfere with interpretation of the study results. • Participated in another research study involving an investigational therapy for ocular disease within the last 6 months • Have any other condition that the Principal Investigator (PI) considers makes them inappropriate for entry into the trial • Are unwilling to consider the possibility of entry into a subsequent longer term follow up study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary research objective is to assess the safety of a AAV2/8 vector for hCNGA gene replacement in the retina. Safety is defined as the absence of ATIMP-related event including, reduction in visual acuity by 15 ETDRS letters, Severe unresponsive inflammation, Infective endophthalmitis, Ocular malignancy, Grade III or above non-ocular SUSAR ;Secondary Objective: The secondary research objective is to determine whether an AAV2/8 vector for hCNGA3 gene replacement in the retina can improve retinal function, visual function and quality of life.; Primary end point(s): The primary outcome is safety of subretinal administration of AAV2/8-hG1.7p.cohCNGA3. Safety is defined as the absence of ATIMP-related: • Reduction in visual acuity by 15 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 SUSAR Safety will be assessed for 6 months after the intervention in this study, and a further 4.5 years in a separate subsequent study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary outcomes are measures of the efficacy of the intervention, which will be performed on an individual participant basis and will be descriptive in nature. Efficacy will be assessed at several time points between 3 to 6 months after the intervention: 1) Any improvement in visual function from baseline that is greater than the baseline variation for that test and is sustained for at least two consecutive assessments. 2) Any improvement in retinal function from pre-intervention that is greater than the baseline variation and measurable by electroretinography (ERG). 3) Quality of life measures including EQ-5D and IVI or equivalent as appropriate. | — |
Countries
United Kingdom, United States
Contacts
Public ContactAmy De Sa
MeiraGTx II UK Ltd
Outcome results
None listed