Status:
ACTIVE_NOT_RECRUITING
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
Lead Sponsor:
Beacon Therapeutics
Conditions:
X-Linked Retinitis Pigmentosa
Eligibility:
MALE
6-50 years
Phase:
PHASE1
PHASE2
Brief 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.
Detailed Description
This protocol includes a non-randomized, open-label, Phase 1/2 study (HORIZON). Approximately 30 participants will be enrolled into the dose escalation study (HORIZON). Each participant will receive t...
Eligibility Criteria
Inclusion
- Phase 1/2 Dose Escalation
- 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
- 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
Key Trial Info
Start Date :
April 16 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2025
Estimated Enrollment :
29 Patients enrolled
Trial Details
Trial ID
NCT03316560
Start Date
April 16 2018
End Date
March 1 2025
Last Update
May 20 2024
Active Locations (4)
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1
Duke University
Durham, North Carolina, United States, 27701
2
Cincinnati Eye Institute
Cincinnati, Ohio, United States, 45242
3
Casey Eye Institute
Portland, Oregon, United States, 97239
4
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231