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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

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ClinicalTrials.gov Identifier: NCT01482195
Recruitment Status : Recruiting
First Posted : November 30, 2011
Last Update Posted : November 30, 2011
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

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.

Condition or disease Intervention/treatment Phase
Retinal Disease Genetic: Recombinant Adeno-Associated Virus Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : August 2011
Estimated Primary Completion Date : August 2014
Estimated Study Completion Date : August 2023


Arm Intervention/treatment
Experimental: Recombinant Adeno-Associated Virus Genetic: Recombinant Adeno-Associated Virus
Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus



Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Visual Outcome [ Time Frame: 12 yrs ]

    Visual function:

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



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Ages Eligible for Study:   14 Years to 70 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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
First Posted: November 30, 2011    Key Record Dates
Last Update Posted: November 30, 2011
Last Verified: November 2011

Keywords provided by Fowzan Alkuraya, King Khaled Eye Specialist Hospital:
Retinal Disease

Additional relevant MeSH terms:
Retinal Diseases
Eye Diseases