Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

Plain-language summary

This Phase 1/2 study evaluates the safety and efficacy of the recombinant adeno-associated viral vector rAAV2tYF-GRK1-RPGR in patients with X-linked retinitis pigmentosa (XLRP) caused by RPGR mutations. It is sponsored by Beacon Therapeutics. Eligibility for the dose-escalation portion requires male subjects with a documented RPGR mutation, a clinical diagnosis of XLRP, best-corrected visual acuity no better than 78 ETDRS letters (20/32) in the study eye, and the ability to perform tests of visual and retinal function.

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

Trial details

Phase

PHASE1/PHASE2

Status

Active, not recruiting

Sponsor

Beacon Therapeutics

Intervention

rAAV2tYF-GRK1-RPGR

Start date

4/16/2018

Estimated completion

3/1/2025

Countries

United States

Related genes

Locations

• Durham, United States
• Cincinnati, United States
• Portland, United States
• Dallas, United States

Eligibility criteria

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

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