Nightstar Therapeutics will begin a phase 2/3 expansion study of its gene therapy candidate for X-linked retinitis pigmentosa, according to a press release.
Designed to evaluate the safety and efficacy of NSR-RPGR in patients with X-linked retinitis pigmentosa due to RPGR mutations, the study will evaluate changes in retinal sensitivity following treatment, the release said.
It will include approximately 45 patients in six U.S. and U.K. surgical centers who have functional impairment as measured by microperimetry and the presence of viable photoreceptors.
Three randomized study arms will be
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