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

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 5 cohorts with a minimum of 3 patients per cohort, with an optional cohort of up to 6 patients. This trial design uses 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.


Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 15 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description: This is a non-confirmatory, open-label single ascending dose gene replacement-therapy study to assess safety, tolerability and efficacy of CPK850 in patients with RLBP1 retinitis pigmentosa due to biallelic mutations in the RLBP1 gene. The trial design uses a staggered patient enrollment.
Masking: Single (Outcomes Assessor)
Masking Description: This is a partially masked study. The patients will not be masked. The treating physicians and personnel at the surgical location (surgeons, anesthesiologist, operating room personnel and others) will not be masked.

At the clinical sites, there will be an unmasked ophthalmologist. The remaining assessors at the clinical sites (ophthalmologist, study nurse, ophthalmic technician, etc) doing the ophthalmic examinations should be masked to the study (treated) eye.

The following unmasked sponsor roles are required for this study:
Sponsor clinical staff required to assist in the management and re-supply of investigational drug product.

The independent committee assessing unmasked interim results and the independent analysis team.

All other sponsor staff will stay masked to treatment assignments

Primary Purpose: Treatment
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
Actual Study Start Date: August 22, 2018
Estimated Primary Completion Date: July 28, 2026
Estimated Study Completion Date: July 29, 2026

Arm:
- Experimental: CPK Dose 1 (lowest dose)
- Experimental: CPK Dose 2 (next lowest dose)
- Experimental: CPK Dose 3 (third lowest dose)
- Experimental: CPK Dose 4 (next to highest dose)
- Experimental: CPK Dose 5 (highest dose)

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