A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
NCT03374657 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12
Last updated 2026-01-15
Summary
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.
Conditions
Interventions
- BIOLOGICAL
-
CPK850
In one of 4 dose levels administered via subretinal injection under anesthesia
Sponsors & Collaborators
- lead INDUSTRY
Principal Investigators
-
Novartis Pharmaceuticals · Novartis Pharmaceuticals
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- SINGLE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 18 Years
- Max Age
- 70 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2018-08-22
- Primary Completion
- 2026-05-11
- Completion
- 2026-05-11
Countries
- Sweden
Study Locations
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