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

Phase 1/2 Completed 12 enrolled Novartis Pharmaceuticals

Non-randomizedSingle-groupSingle-blindTreatment

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.

Timeline

Start: 2018-08-22
Primary completion: 2026-05-12
Completion: 2026-05-12

Outcome

Met primary endpoint

paper Dark adaptation kinetics, the primary efficacy endpoint, improved significantly in all dose-cohorts. PMID 39256350 ↗

Publications

Kvanta A, Rangaswamy N, Holopigian K, Watters C, Jennings N, Liew MSH, Bigelow C, Grosskreutz C, Burstedt M, Venkataraman A, Westman S, Geirsdottir A, Stasi K, Andre H. Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial. Nat Commun. 2024 Sep 10;15(1):7438. doi: 10.1038/s41467-024-51575-4.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	CPK850	Gene therapy (AAV / viral vector)	—	Other