Gene Therapy Trial for patients with Achromatopsia due to a gene defect (CNGA3)
- Conditions
- Achromatopsia caused by mutations in the CNGA3 geneMedDRA version: 20.0 Level: LLT Classification code 10000454 Term: Achromatopsia System Organ Class: 100000004850Therapeutic area: Diseases [C] - Eye Diseases [C11]
- Registration Number
- EUCTR2018-003431-29-GB
- Lead Sponsor
- MeiraGTx UK II Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Not specified
- Target Recruitment
- 36
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
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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method