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adeno-associated virus vector AAV- CNGA3 · 1 trial · 1 indication
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)
| Arm | Type | Description |
|---|---|---|
| Low dose adeno-associated virus (AAV) CNGA3 | EXPERIMENTAL | Subretinal administration of a single low dose AAV CNGA3 |
| Intermediate dose adeno-associated virus (AAV) CNGA3 | EXPERIMENTAL | Subretinal administration of a single intermediate dose AAV CNGA3 |
| High dose adeno-associated virus (AAV) CNGA3 | EXPERIMENTAL | Subretinal administration of a single high dose AAV CNGA3 |
| Name | Type | Description |
|---|---|---|
| adeno-associated virus vector AAV- CNGA3 | BIOLOGICAL | Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene |
Inclusion Criteria: * Are aged years or over * Have achromatopsia confirmed by a retinal specialist investigator Exclusion Criteria: * Are females who are pregnant or breastfeeding * Have participated in another research study involving an investigational medicinal therapy for ocular disease with...
Adeno-associated virus vector AAV-CNGA3 is an investigational gene therapy being developed for achromatopsia, a rare inherited retinal condition. It is designed to deliver a functional copy of the CNGA3 gene to patients affected by this disorder. The therapy is administered as a single treatment and remains in clinical development.
Adeno-associated virus vector AAV-CNGA3 is being developed by MeiraGTx Holdings plc, a clinical-stage gene therapy company. MeiraGTx trades on the Nasdaq under the ticker symbol MGTX. The company is responsible for the research, development, and clinical evaluation of this gene therapy candidate for achromatopsia.
Adeno-associated virus vector AAV-CNGA3 is in Phase 1 clinical development. The Phase 1 trial has been completed. As an investigational gene therapy, it has not been approved by the FDA or any other regulatory agency for commercial use. Further clinical studies would be required to support potential marketing approval.
Adeno-associated virus vector AAV-CNGA3 has been evaluated in one clinical trial, NCT03758404, titled Gene Therapy for Achromatopsia (CNGA3). This Phase 1 trial enrolled 11 participants across the United States and the United Kingdom, including patients aged 3 years and older. The study is now completed.