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

Plain-language summary

This study assessed the safety of gene transfer via subretinal administration of rAAV2-VMD2-hMERTK in people with MERTK-associated retinitis pigmentosa (RP). The Phase 1 trial enrolled participants with MERTK-associated retinal disease, visual acuity of 20/100 or worse in the worse eye, the ability to perform tests of visual and retinal function, and good general health.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 1

Status

Completed

Sponsor

King Khaled Eye Specialist Hospital

Intervention

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

Start date

8/1/2011

Estimated completion

8/1/2019

Countries

Saudi Arabia

Related genes

Locations

• Riyadh, Saudi Arabia

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.