Status:
COMPLETED
Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)
Lead Sponsor:
King Khaled Eye Specialist Hospital
Collaborating Sponsors:
King Faisal Specialist Hospital & Research Center
Conditions:
Retinal Disease
Retinitis Pigmentosa
Eligibility:
All Genders
14-70 years
Phase:
PHASE1
Brief 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).
Detailed 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 wil...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
August 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2019
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT01482195
Start Date
August 1 2011
End Date
August 1 2019
Last Update
January 26 2022
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
King Khaled Eye Specialist Hospital
Riyadh, Saudi Arabia, 11462