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ACTIVE NOT RECRUITING
NCT03374657
PHASE1/PHASE2

A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa

Sponsor: Novartis Pharmaceuticals

View on ClinicalTrials.gov

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

Interventions

BIOLOGICAL

CPK850

In one of 4 dose levels administered via subretinal injection under anesthesia

Locations (1)

Novartis Investigative Site

Stockholm, Sweden