Trials / Completed
CompletedNCT02946879
Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children With Retinal Dystrophy Owing to Defects in RPE65 (LCA2)
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 15 (actual)
- Sponsor
- MeiraGTx UK II Ltd · Industry
- Sex
- All
- Age
- 3 Years – 100 Years
- Healthy volunteers
- Not accepted
Summary
This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.
Detailed description
The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | AAV OPTIRPE65 | comparison of different doses of AAV RPE65 |
Timeline
- Start date
- 2016-11-01
- Primary completion
- 2023-07-01
- Completion
- 2023-07-01
- First posted
- 2016-10-27
- Last updated
- 2024-04-11
Locations
2 sites across 2 countries: United States, United Kingdom
Source: ClinicalTrials.gov record NCT02946879. Inclusion in this directory is not an endorsement.