Trials / Completed
CompletedNCT01482195
Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)
Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-VMD2-hMERTK) Gene Vector to Patients With Retinal Disease Due to MERTK Mutations
- Status
- Completed
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 6 (actual)
- Sponsor
- King Khaled Eye Specialist Hospital · Other Government
- Sex
- All
- Age
- 14 Years – 70 Years
- Healthy volunteers
- Not accepted
Summary
This study was to assess the safety of gene transfer via subretinal administration of rAAV2-VMD2-hMERTK in subjects with MERTK-associated retinitis pigmentosa (RP).
Detailed description
In this phase I open-label, dose-escalation trial, one eye of each patient (the worse-seeing eye in five subjects) will receive a submacular injection of the subretinal rAAV2-VMD2-hMERTK. Patients will be followed daily for 10 days and then at 30, 60, 90, 180, 270, 365, 540, and 730 days post-injection. Data will be collected on (1) full ophthalmologic examination including best-corrected VA, intraocular pressure, color fundus photographs, macular spectral-domain optical coherence tomography, and full-field stimulus threshold test (FST) in both the study and fellow eyes; (2) systemic safety data including CBC, liver, and kidney function tests, coagulation profiles, urine analysis, AAV antibody titers, peripheral blood PCR and ASR measurement; and (3) listing of ophthalmological or systemic adverse effects.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus | The study is an open-label, dose-escalation, phase I clinical trial of subretinal administration of rAAV2-VMD2-hMERTK in patients with retinitis pigmentosa due to MERTK mutation. |
Timeline
- Start date
- 2011-08-01
- Primary completion
- 2013-08-01
- Completion
- 2019-08-01
- First posted
- 2011-11-30
- Last updated
- 2022-01-26
- Results posted
- 2021-07-15
Locations
1 site across 1 country: Saudi Arabia
Source: ClinicalTrials.gov record NCT01482195. Inclusion in this directory is not an endorsement.