Trials / Completed
CompletedNCT03758404
Gene Therapy for Achromatopsia (CNGA3)
An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Children and Adults With Achromatopsia Owing to Defects in CNGA3
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 11 (actual)
- Sponsor
- MeiraGTx UK II Ltd · Industry
- Sex
- All
- Age
- 3 Years
- Healthy volunteers
- Not accepted
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
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | adeno-associated virus vector AAV- CNGA3 | Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene |
Timeline
- Start date
- 2019-08-12
- Primary completion
- 2021-06-10
- Completion
- 2021-06-10
- First posted
- 2018-11-29
- Last updated
- 2022-12-01
- Results posted
- 2022-02-11
Locations
2 sites across 2 countries: United States, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03758404. Inclusion in this directory is not an endorsement.