Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused b… (NCT03316560) | Clinical Trial Compass
UnknownPhase 1/2
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
United States29 participantsStarted 2018-04-16
Plain-language summary
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
Who can participate
Age range
6 Years – 50 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Phase 1/2 Dose Escalation Inclusion Criteria:
* Male subjects with a documented RPGR mutation
* Clinical diagnosis of X-linked retinitis pigmentosa (XLRP)
* Best-corrected visual acuity not better than 78 ETDRS letters (20/32) in the study eye;
* Ability to perform tests of visual and retinal function and structure and ability to comply with other research procedures;
* Detectable baseline mean macular sensitivity, as measured by microperimetry.
* Have detectable Ellipsoid Zone (EZ) line during the pre-treatment period as assessed by OCT and confirmed by the CRC.
Phase 1/2 Dose Escalation Exclusion Criteria:
* Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or increase the risk of surgical complications (for example, glaucoma, corneal or lenticular opacities, diabetic retinopathy, retinal vasculitis);
* Use of anti-coagulant agents that may alter coagulation within 7 days prior to study agent administration;
* Use of systemic corticosteroids or other immunosuppressive medications within 3 months prior to enrollment;
* Any other condition that would prevent a subject from completing follow-up examinations during the course of the study;
* Any other condition or reason that, in the opinion of the investigator, makes the subject unsuitable for the study;
* Previous receipt of any AAV gene therapy product;
* Monocular or having BCVA less than 20/800 in the fellow eye
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number and proportion of Adverse Events
Timeframe: Day 0 - Month 36
2
Number and proportion of participants experiencing abnormal clinically relevant hematology or clinical chemistry parameters.