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NCT03316560 · ClinicalTrials.gov registry record · Phase 1
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
A Phase 1 study of X-Linked Retinitis Pigmentosa, sponsored by Beacon Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 29
- Enrollment target
- 4
- Study locations
NCT03316560: Active Phase 1 study of X-Linked Retinitis Pigmentosa, sponsored by Beacon Therapeutics.
NCT03316560 is a Phase 1 study of X-Linked Retinitis Pigmentosa that is active but no longer recruiting, run by Beacon Therapeutics. The registered enrollment target is 29 participants, below the 55-participant average among 8 other X-Linked Retinitis Pigmentosa trials with a reported enrollment target (47% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03316560, a Phase 1 study of X-Linked Retinitis Pigmentosa, is active but no longer recruiting, sponsored by Beacon Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 29 participants
- Enrollment target
- 4
- Study locations
Study 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.
Primary Outcome
Number and proportion of participants experiencing Grade 3 or higher local (ocular) or systemic treatment-emergent adverse events that occur during the 36 months after study agent administration; number and proportion of participants experiencing treatment-emergent AEs, including treatment-emergent serious AEs;
Conditions Studied
Interventions
- BIOLOGICAL rAAV2tYF-GRK1-RPGR
Study Locations (4)
North Carolina
- Duke University - Durham
Ohio
- Cincinnati Eye Institute - Cincinnati
Oregon
- Casey Eye Institute - Portland
Texas
- Retina Foundation of the Southwest - Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 29 participants |
| Start Date | 2018-04-16 |
| Est. Completion | 2025-03 |
| Phase | Phase 1 |
What the registry record for NCT03316560 still lists
NCT03316560 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 29 participants, a relatively small participant target, below the 55-participant average among 8 other X-Linked Retinitis Pigmentosa trials with a reported enrollment target (47% lower).
The record links to 1 condition, with X-Linked Retinitis Pigmentosa appearing as the primary indexed condition, and to 1 intervention - of which rAAV2tYF-GRK1-RPGR is the first listed.
NCT03316560 reports a single indexed study location in North Carolina, Ohio, Oregon.
Frequently Asked Questions
What is clinical trial NCT03316560 about?
NCT03316560 is a clinical study titled "Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations". 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.
What is the current status of trial NCT03316560?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 29 participants. The study started on 2018-04-16. Estimated completion is 2025-03.
What conditions does trial NCT03316560 study?
This clinical trial studies the following conditions: X-Linked Retinitis Pigmentosa.
What interventions are being tested in trial NCT03316560?
The interventions under investigation include: rAAV2tYF-GRK1-RPGR (BIOLOGICAL).
Who is sponsoring clinical trial NCT03316560?
This trial is sponsored by Beacon Therapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03316560 being conducted?
This trial has 4 study locations across North Carolina, Ohio, Oregon, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for X-Linked Retinitis Pigmentosa
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03316560's enrollment target sits among peer trials
29 6th of 8 higher than 3 of 8 other X-Linked Retinitis Pigmentosa trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other X-Linked Retinitis Pigmentosa trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
ACTIVE NOT RECRUITING · Phase 1
-
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
COMPLETED · Phase 1
-
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
ACTIVE NOT RECRUITING · Phase 2
-
A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
ACTIVE NOT RECRUITING · Phase 2
-
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
ACTIVE NOT RECRUITING · Phase 2
-
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
ACTIVE NOT RECRUITING · Phase 3
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