A first-in-human, proof of concept study of CPK850 in patients with RLBP1 retinitis pigmentosa
A first-in-human, proof of concept study of CPK850 in patients with RLBP1 retinitis pigmentosa
| Trial Fact Box | |
|---|---|
| NCT Code | NCT03374657 |
| Sponsor | Novartis Pharmaceuticals |
| Study type(s) | Interventional |
| Phase | Phase 1 / Phase 2 |
| Enrollment (Actual) | 12 |
| Study Start (Actual) | 22 August 2018 |
| Primary Completion (Estimated) | 12 May 2026 |
| Study Completion (Estimated) | 12 May 2026 |
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
Brief 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.
Detailed Description:
This study will potentially include 4 cohorts with a minimum of 3 patients per cohort. This trial design used a staggered patient enrollment with continuous data reviews to limit as much unforeseen risk as possible prior to enrolling each patient in each cohort or initiating another cohort. Only one eye (designated as the study or treated eye) will be dosed per patient. Each patient will be followed for 5 years after the subretinal injection of CPK850.
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