A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Retinitis Pigmentosa
- Type
- Interventional
- Phase
- Phase 1Phase 2
- Design
- Allocation: Non-RandomizedIntervention Model: Single Group AssignmentIntervention 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 assignmentsPrimary Purpose: Treatment
Participation Requirements
- Age
- Between 18 years and 70 years
- Gender
- Both males and females
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 ...
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.
Tracking Information
- NCT #
- NCT03374657
- Collaborators
- Not Provided
- Investigators
- Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
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