Trials / Completed
CompletedNCT02781480
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 15 (actual)
- Sponsor
- MeiraGTx UK II Ltd · Industry
- Sex
- All
- Age
- 3 Years
- Healthy volunteers
- Not accepted
Summary
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
Detailed description
A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | AAV RPE65 | Comparison of different dosages of AAV RPE65 |
Timeline
- Start date
- 2016-04-01
- Primary completion
- 2018-12-01
- Completion
- 2018-12-01
- First posted
- 2016-05-24
- Last updated
- 2021-07-12
- Results posted
- 2021-04-08
Locations
2 sites across 2 countries: United States, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT02781480. Inclusion in this directory is not an endorsement.