Ophthalmological

Retinitis Pigmentosa

Also known as RP, rod-cone dystrophy, pigmentary retinopathy

Retinitis Pigmentosa is a clinically and genetically heterogeneous group of inherited retinal dystrophies characterised by progressive degeneration of rod photoreceptors followed by cone involvement, leading to night blindness, constricting

ORPHA:791 ↗Gene RPGRGene RP1Gene RHOGene USH2A (multiple)Prevalence 1 per 3,500–4,000Onset Childhood to early adulthood (variable by subtype)Autosomal recessive, autosomal dominant, X-linked

48

studies recruiting now

as of 7 Sept 2026

319

studies registered in total

as of 7 Sept 2026

14

countries with a recruiting site

as of 7 Sept 2026

23 Jan 2025

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 48 recruiting studies

Where recruiting studies are running

Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.

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About Retinitis Pigmentosa

Retinitis Pigmentosa is a clinically and genetically heterogeneous group of inherited retinal dystrophies characterised by progressive degeneration of rod photoreceptors followed by cone involvement, leading to night blindness, constricting visual fields, and eventual central vision loss. Over 90 causative genes have been identified, making RP the most genetically diverse of the inherited retinal diseases. It can occur as an isolated ocular condition or as part of syndromic conditions such as Usher syndrome and Bardet-Biedl syndrome.

Common clinical features

Night blindness (nyctalopia), often the first symptomProgressive peripheral visual field constriction (tunnel vision)Difficulty with dark adaptationBone-spicule pigmentation on fundus examinationAttenuated retinal blood vesselsWaxy pallor of the optic discReduced or extinguished rod ERG responsesLate-stage central vision loss

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

19 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Phase 3Lutein
Phase 3Retinol (Arovit)
Phase 3Unoprostone Isopropyl (Rescula)
Phase 3Acetylcysteine (A-cys)
Phase 3Vitamin E (Aquasol e)
Phase 3Voretigene Neparvovec (Luxturna)
Phase 2/3Ciliary Neurotrophic Factor
Phase 2Levodopa (Bendopa)

+ 11 more in development

Before you apply

Things trial teams commonly ask about for Retinitis Pigmentosa. Not eligibility rules; those are set by each study.

  • Genetic subtype is critical; trials are often gene-specific (e.g., RPGR-targeted for X-linked RP), so a confirmed genetic diagnosis via a retinal gene panel is essential before screening.
  • Visual field area and ERG amplitude are standard eligibility benchmarks; retain copies of recent Goldmann or automated perimetry results.
  • Syndromic RP (e.g., Usher syndrome) may have separate dedicated trials; confirm whether your diagnosis is isolated or syndromic before applying.

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).