Trials / NCT05573984
Natural History of PRPF31 Mutation-Associated Retinal Dystrophy
Underway, not enrolling Observational Natural history / observational
- Sponsor
- PYC Therapeutics
- Treatment
- Not listed
- Gene
- PRPF31
- Conditions
- Retinitis Pigmentosa, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Dystrophy Rod, Retinal Dystrophy Rod Progressive
- Ages
- 10 Years to no upper limit
- Enrollment
- 50 participants
- Started
- 2022-07-07
- Main results expected
- 2026-09-09
- Results posted
- Not yet
- Record last updated
- 2026-04-13
What the study is testing
The purpose of this study is to characterize the natural history through temporal systemic evaluation of subjects identified with PRPF31 mutation-associated retinal dystrophy, also called retinitis pigmentosa type 11, or RP11.
Assessments will be completed to measure and evaluate structural and functional visual changes including those impacting patient quality of life associated with this inherited retinal condition and observing how these changes evolve over time.
Where it runs
Australia (2)
- Lions Eye Institute, Nedlands, Western Australia
- Centre For Eye Research Australia (CERA) - Retinal Gene Therapy Unit, East Melbourne
United States (5)
- University of California San Francisco, San Francisco, California
- University of Florida Health, Jacksonville, Florida
- University of Michigan Kellogg Eye Center, Ann Arbor, Michigan
- Oregon Health and Science University - Casey Eye Institute, Portland, Oregon
- Retina Foundation of the Southwest, Dallas, Texas