This Phase 2/3 study is a randomized, controlled, masked, multi-center study evaluating and comparing 2 doses of AGTC-501 to an untreated control group. A single subretinal injection of AGTC-501 Dose 1(low dose) or Dose 2(high dose) will be administered in subjects in 2 treatment groups (N=~42), while subjects in the untreated control group (N=~21) will be followed and evaluated up to Month 12, after which they will be eligible to receive treatment with AGTC-501 Dose 2.~Approximately 63 eligible male subjects between 8 and 50 years of age (inclusive) will be randomized in a 1:1:1 ratio to 1 of 3 groups (low dose, high dose, untreated control).
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501) to an untreated control group in male patients with X-linked retinitis pigmentosa caused by RPGR mutations.