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:

    1. Antibody titers to AAV capsid components and Antigen-specific Reactivity (ASR) Measurement.
    2. Peripheral Blood PCR.


Original Primary Outcome: Same as current

Current Secondary Outcome: Visual Outcome [ Time Frame: 12 yrs ]

Visual function:

  1. ETDRS visual acuity measurement.
  2. 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