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A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
Sponsor: Novartis Pharmaceuticals
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.
Official title: An Open-label First-in-human Single Ascending Dose Study to Explore Safety, Tolerability and Efficacy of Subretinal Administration of CPK850 Gene Therapy in Patients With Retinitis Pigmentosa Due to Mutations in the Retinaldehyde Binding Protein 1 (RLBP1) Gene
Key Details
Gender
All
Age Range
18 Years - 70 Years
Study Type
INTERVENTIONAL
Enrollment
12
Start Date
2018-08-22
Completion Date
2026-05-11
Last Updated
2026-01-15
Healthy Volunteers
No
Conditions
Interventions
CPK850
In one of 4 dose levels administered via subretinal injection under anesthesia
Locations (1)
Novartis Investigative Site
Stockholm, Sweden