Retinitis pigmentosa 3
MONDO:0010227Any retinitis pigmentosa in which the cause of the disease is a mutation in the RPGR gene.
Also known as: RP3, RPGR retinitis pigmentosa, retinitis pigmentosa 3, retinitis pigmentosa caused by mutation in RPGR, retinitis pigmentosa type 3, Choroidoretinal Degeneration with retinal reflex in heterozygous Women, cone-rod Degeneration, X-linked, retinitis pigmentosa 15
32 clinical trials for this condition and its sub-types.
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Gene therapy targets second eye in rare blindness disorder
Disease control OngoingThis study tests a gene therapy called botaretigene sparoparvovec in the second eye of people with X-linked retinitis pigmentosa, a genetic condition that causes vision loss. The 24 participants previously received the therapy in one eye and are now being followed for safety and …
Phase: PHASE2 • Sponsor: Janssen Research & Development, LLC • Aim: Disease control
Last updated Jul 08, 2026 00:00 UTC
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Gene therapy shows promise for rare blindness disease
Disease control OngoingThis study is a follow-up to a Phase 3 trial testing a gene therapy called AAV5-hRKp.RPGR for people with X-linked retinitis pigmentosa, a genetic eye disease that causes vision loss. The treatment aims to slow or stop vision decline by delivering a working copy of the RPGR gene …
Phase: PHASE3 • Sponsor: Janssen Research & Development, LLC • Aim: Disease control
Last updated Jul 08, 2026 00:00 UTC
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Experimental gene therapy aims to halt vision loss in rare eye disease
Disease control OngoingThis Phase 3 study tests a gene therapy called AAV5-hRKp.RPGR in 4 Japanese people with X-linked retinitis pigmentosa, a genetic condition that causes progressive vision loss. The therapy is injected into the eye to deliver a working copy of the RPGR gene. The main goal is to che…
Phase: PHASE3 • Sponsor: Janssen Pharmaceutical K.K. • Aim: Disease control
Last updated Jul 08, 2026 00:00 UTC
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Gene therapy shot into both eyes aims to save sight in rare blindness
Disease control OngoingThis study tests a gene therapy called laruparetigene zovaparvovec (AGTC-501) in 10 males aged 12-50 with X-linked retinitis pigmentosa, a genetic condition that leads to vision loss. The therapy is injected into both eyes to deliver a working copy of the RPGR gene. The main goal…
Phase: PHASE2 • Sponsor: Beacon Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC