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. (see Contacts and Locations)
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

September 28, 2011
November 29, 2011
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 ] [ 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.
Same as current
Complete list of historical versions of study NCT01482195 on ClinicalTrials.gov Archive Site
Visual Outcome [ Time Frame: 12 yrs ] [ Designated as safety issue: No ]

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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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
Not Provided

*   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.
Both
14 Years to 70 Years
No
Contact: Fowzan S Alkuraya, MD +966 1 442 7875 falkuraya@kfshrc.edu.sa
Saudi Arabia
 
NCT01482195
0916-P
Yes
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