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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

This study is currently recruiting participants.
Verified November 2011 by Fowzan Alkuraya, King Khaled Eye Specialist Hospital
Sponsor:
ClinicalTrials.gov Identifier:
NCT01482195
First Posted: November 30, 2011
Last Update Posted: October 12, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborators:
King Khaled Eye Specialist Hospital
King Faisal Specialist Hospital & Research Center
Information provided by (Responsible Party):
Fowzan Alkuraya, King Khaled Eye Specialist Hospital
September 28, 2011
November 30, 2011
October 12, 2017
August 2011
August 2014   (Final data collection date for primary outcome measure)
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.
Same as current
No Changes Posted
Visual Outcome [ Time Frame: 12 yrs ]

Visual function:

  1. ETDRS visual acuity measurement.
  2. Full-field Stimulation Threshold (FST).
Same as current
Not Provided
Not Provided
 
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
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
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.
Not Provided
Interventional
Phase 1
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Retinal Disease
Genetic: Recombinant Adeno-Associated Virus
Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus
Experimental: Recombinant Adeno-Associated Virus
Intervention: Genetic: Recombinant Adeno-Associated Virus
Parinot C, Nandrot EF. A Comprehensive Review of Mutations in the MERTK Proto-Oncogene. Adv Exp Med Biol. 2016;854:259-65. doi: 10.1007/978-3-319-17121-0_35. Review.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
6
August 2023
August 2014   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
14 Years to 70 Years   (Child, Adult, Senior)
No
Contact: Fowzan S Alkuraya, MD +966 1 442 7875 falkuraya@kfshrc.edu.sa
Saudi Arabia
 
 
NCT01482195
0916-P
Yes
Not Provided
Not Provided
Fowzan Alkuraya, King Khaled Eye Specialist Hospital
Fowzan Alkuraya
  • King Khaled Eye Specialist Hospital
  • King Faisal Specialist Hospital & Research Center
Principal Investigator: Fowzan S Alkuraya, MD King Faisal Specialist Hospital & Research Center
King Khaled Eye Specialist Hospital
November 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP