Clinical Trial: 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
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
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 Mutation
Brief Summary: 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.
Detailed Summary:
Sponsor: Fowzan Alkuraya
Current Primary Outcome: Ocular and Systemic safety of uniocular subretinal administration of rAAV2-VMD2-hMERTK in individuals with MERTK-associated retinal disease [ Time Frame: 12 yrs ]
1.Ocular safety:
- Keratitis.
- Glaucoma.
- Cataract.
- Uveitis.
- Vitreous hemorrhage.
Retinal detachment.
2.Systemic Safety:
- Organs systemic toxicity.
Viral Signs:
- Antibody titers to AAV capsid components and Antigen-specific Reactivity (ASR) Measurement.
- Peripheral Blood PCR.
Original Primary Outcome: Same as current
Current Secondary Outcome: Visual Outcome [ Time Frame: 12 yrs ]
Visual function:
- ETDRS visual acuity measurement.
- Full-field Stimulation Threshold (FST).
Original Secondary Outcome: Same as current
Information By: King Khaled Eye Specialist Hospital
Dates:
Date Received: September 28, 2011
Date Started: August 2011
Date Completion: August 2023
Last Updated: November 29, 2011
Last Verified: November 2011