Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)

King Khaled Eye Specialist Hospital

Status and phase: Completed; Phase 1

Conditions: Retinitis Pigmentosa; Retinal Disease

Treatments: Biological: Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus

Study type: Interventional

Funder types: Other

Identifiers

NCT01482195

0916-P

Details and patient eligibility

About

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).

Full description

In this phase I open-label, dose-escalation trial, one eye of each patient (the worse-seeing eye in five subjects) will receive a submacular injection of the subretinal rAAV2-VMD2-hMERTK. Patients will be followed daily for 10 days and then at 30, 60, 90, 180, 270, 365, 540, and 730 days post-injection. Data will be collected on (1) full ophthalmologic examination including best-corrected VA, intraocular pressure, color fundus photographs, macular spectral-domain optical coherence tomography, and full-field stimulus threshold test (FST) in both the study and fellow eyes; (2) systemic safety data including CBC, liver, and kidney function tests, coagulation profiles, urine analysis, AAV antibody titers, peripheral blood PCR and ASR measurement; and (3) listing of ophthalmological or systemic adverse effects.

Enrollment: 6 patients

Sex: All

Ages: 14 to 70 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 2 patient groups

Subretinal Injection of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus

Experimental group

Description:

Single arm of 6 patients undergoing subretinal injection of Gene Therapy using rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus. Each patient received the injection in one eye.

Treatment:

Biological: Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus

fellow eye without intervention

No Intervention group

Description:

fellow eye without intervention