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

This study is currently recruiting participants.
Verified November 2011 by King Khaled Eye Specialist Hospital
Sponsor:
Collaborators:
King Khaled Eye Specialist Hospital
King Faisal Specialist Hospital & Research Center
Information provided by (Responsible Party):
Fowzan Alkuraya, King Khaled Eye Specialist Hospital
ClinicalTrials.gov Identifier:
NCT01482195
First received: September 28, 2011
Last updated: November 29, 2011
Last verified: November 2011
  Purpose

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.


Condition Intervention Phase
Retinal Disease
Genetic: Recombinant Adeno-Associated Virus
Phase 1

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by King Khaled Eye Specialist Hospital:

Primary Outcome Measures:
  • 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 ]

    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.


Secondary Outcome Measures:
  • Visual Outcome [ Time Frame: 12 yrs ] [ Designated as safety issue: No ]

    Visual function:

    1. ETDRS visual acuity measurement.
    2. Full-field Stimulation Threshold (FST).


Estimated Enrollment: 6
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)
Arms Assigned Interventions
Experimental: Recombinant Adeno-Associated Virus Genetic: Recombinant Adeno-Associated Virus
Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus

  Eligibility

Ages Eligible for Study:   14 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • MERTK-associated retinal disease;
  • VA: 20/100 or less in worse eye
  • Ability to perform tests of visual and retinal function;
  • Good general health based on a complete physical examination and hematology and chemistry studies performed at a pre-treatment evaluation;
  • Ability to comply with research procedures;

Exclusion Criteria:

  • Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or surgical complications (for example, glaucoma, corneal or lenticular opacities);
  • Complicating systemic diseases (such as medical conditions causing immunosuppression) that would preclude the gene transfer, ocular surgery or known sensitivity or allergy to medications planned for use in the peri-operative period;
  • Use of anti-platelet agents that may alter coagulation within 7 days prior to study agent administration;
  • Use of immunosuppressive medications;
  • Pregnancy or breastfeeding;
  • Individuals (males and females) of childbearing potential who are unwilling to use effective contraception for 1 year following agent administration and barrier contraception for 3 months following agent administration;
  • Any other condition that would prevent a subject from completing follow-up examinations during the course of the study and that, in the opinion of the investigator, makes the subject unsuitable for the study.
  • Current, or recent (within the past 30 days, or 10 half lives of the drug) participation, in any other research protocol involving investigational agents or therapies.
  • Recent (within past 6 months) receipt of an investigational biologic therapeutic agent.Subjects will not be excluded based on their gender, race or ethnicity.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01482195

Contacts
Contact: Fowzan S Alkuraya, MD +966 1 442 7875 falkuraya@kfshrc.edu.sa

Locations
Saudi Arabia
King Khaled Eye Specialist Hospital Recruiting
Riyadh, Saudi Arabia, 11462
Contact: Deepak Edward, MD    96614821234 ext 1362    dedward@kkesh.med.sa   
Principal Investigator: Emad Abboud, MD         
Sponsors and Collaborators
Fowzan Alkuraya
King Khaled Eye Specialist Hospital
King Faisal Specialist Hospital & Research Center
Investigators
Principal Investigator: Fowzan S Alkuraya, MD King Faisal Specialist Hospital & Research Center
  More Information

No publications provided

Responsible Party: Fowzan Alkuraya, Head, Developmental Genetics Unit, King Khaled Eye Specialist Hospital
ClinicalTrials.gov Identifier: NCT01482195     History of Changes
Other Study ID Numbers: 0916-P
Study First Received: September 28, 2011
Last Updated: November 29, 2011
Health Authority: Saudi Arabia: Food and Drug Administation

Keywords provided by King Khaled Eye Specialist Hospital:
Retinal Disease

Additional relevant MeSH terms:
Retinal Diseases
Eye Diseases

ClinicalTrials.gov processed this record on April 14, 2014