Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)
Condition: Retinal Disease; Retinitis Pigmentosa
MeSH terms: Retinitis; Retinitis Pigmentosa; Retinal Diseases
Intervention: Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus
Intervention description: The study is an open-label, dose-escalation, phase I clinical trial of subretinal administration of rAAV2-VMD2-hMERTK in patients with retinitis pigmentosa due to MERTK mutation.
Phase: Phase 1
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Intervention model description: Ocular Subretinal administration of rAAV2-VMD2-hMERTK .
Masking: None (Open Label)
Enrollment: 6.0
Sex: All
Minimum age: 14 Years
Maximum age: 70 Years
Healthy volunteers: No
Keywords: Retinal Disease; subretinal gene therapy; MERTK; retinitis pigmentosa

Summary:

      This study was to assess the safety of gene transfer via subretinal administration of
      rAAV2-VMD2-hMERTK in subjects with MERTK-associated retinitis pigmentosa (RP).
    

Eligibility 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.