Trials / NCT03374657
A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
Completed Phase 1 / Phase 2 Gene therapy
- Sponsor
- Novartis Pharmaceuticals
- Treatment
- CPK850
- Gene
- RLBP1
- Conditions
- Retinitis Pigmentosa
- Ages
- 18 Years to 70 Years
- Enrollment
- 12 participants
- Started
- 2018-08-22
- Main results expected
- 2026-05-12
- Results posted
- Not yet
- Record last updated
- 2026-06-11
What the study is testing
The purpose of this first-in-human study is to explore the maximum tolerated dose (MTD) of CPK850 as determined by the single ascending dose ranging portion of the study. This study will also evaluate the safety and potential efficacy of CPK850 on improving visual function in patients with decreased visual function from RLBP1 retinitis pigmentosa due to biallelic mutations in the RLBP1 gene.
Where it runs
Sweden (1)
- Novartis Investigative Site, Stockholm