Status and phase
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About
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.
Full 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 the study agent by subretinal injection in one eye on a single occasion. Enrollment will begin with the lowest dose and will proceed to higher doses only after review of safety data by a Data and Safety Monitoring Committee (DSMC). There are a total of 15 visits over approximately 36 months, and long-term follow-up evaluations annually at years 4 and 5.
Enrollment
Sex
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Volunteers
Inclusion and exclusion criteria
Phase 1/2 Dose Escalation Inclusion Criteria:
Phase 1/2 Dose Escalation Exclusion Criteria:
Primary purpose
Allocation
Interventional model
Masking
29 participants in 6 patient groups
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Central trial contact
Jill Dolgin, PharmD; Serva Health
Data sourced from clinicaltrials.gov
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