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
A recombinant adeno-associated virus serotype 2 (rAAV2) vector has been altered to carry the human MERTK (hMERTK) gene. This vector has been shown to restore vision in animal models that resemble human MERTK-associated Retinitis Pigmentosa (RP), an incurable retinal degeneration that causes severe vision loss. The proposed study is an open label, Phase I clinical trial of subretinal rAAV2-VMD2-hMERTK administration to individuals with MERTK-associated retinal disease. This trial will lead to a greater understanding of the safety and thereby potential value of gene transfer in MERTK-associated retinal disease and will have implications for other forms of retinal degenerative disease amenable to this type of intervention.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||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|
- Ocular and Systemic safety of uniocular subretinal administration of rAAV2-VMD2-hMERTK in individuals with MERTK-associated retinal disease [ Time Frame: 12 yrs ] [ Designated as safety issue: Yes ]
- Vitreous hemorrhage.
- Organs systemic toxicity.
- Antibody titers to AAV capsid components and Antigen-specific Reactivity (ASR) Measurement.
- Peripheral Blood PCR.
- Visual Outcome [ Time Frame: 12 yrs ] [ Designated as safety issue: No ]
- ETDRS visual acuity measurement.
- Full-field Stimulation Threshold (FST).
|Study Start Date:||August 2011|
|Estimated Study Completion Date:||August 2023|
|Estimated Primary Completion Date:||August 2014 (Final data collection date for primary outcome measure)|
|Experimental: Recombinant Adeno-Associated Virus||
Genetic: Recombinant Adeno-Associated Virus
Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus
Please refer to this study by its ClinicalTrials.gov identifier: NCT01482195
|Contact: Fowzan S Alkuraya, MD||+966 1 442 email@example.com|
|King Khaled Eye Specialist Hospital||Recruiting|
|Riyadh, Saudi Arabia, 11462|
|Contact: Deepak Edward, MD 96614821234 ext 1362 firstname.lastname@example.org|
|Principal Investigator: Emad Abboud, MD|
|Principal Investigator:||Fowzan S Alkuraya, MD||King Faisal Specialist Hospital & Research Center|