A Phase 1/2 interventional study of CPK850 in Retinitis Pigmentosa, sponsored by Novartis Pharmaceuticals. Completed at 1 site in Sweden. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-06-11.
Sponsored by Novartis Pharmaceuticals · Phase 1/2, Interventional, and Treatment
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.
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.
Exclusion Criteria:
CPK850, one subretinal injection to the study eye
Biological: CPK850
CPK850, one subretinal injection to the study eye
Biological: CPK850
CPK850, one subretinal injection to the study eye
Biological: CPK850
CPK850, one subretinal injection to the study eye
Biological: CPK850
In one of 4 dose levels administered via subretinal injection under anesthesia
Number of participants with adverse events (AEs), serious adverse events (SAEs) and deaths
Safety events
Time frame: Up to Year 5
Number of responders in dark adaptation
A patient is considered a responder if sensitivity recovery values at 1 hour post-bleach are observed to be outside of the patient's prediction interval at ≥2 consecutive post-treatment visits within one year after treatment.
Time frame: Screening/baseline up to Year 1
Number of responders with recovery of the cone system
cone recovery during dark adaptation
Time frame: Screening/baseline up to Year 1
Change from screening/baseline in Visual field perimetry mean deviation
Assessed using automated static perimetry
Time frame: Screening/baseline up to Year 2
Change from screening/baseline in Total contrast sensitivity score
Contrast sensitivity (ie, the ability to detect relatively dim objects) will be assessed
Time frame: Screening/baseline up to Year 2
Change from screening/baseline in Light-adapted microperimetry sensitivity
Assessed using standard microperimetry equipment
Time frame: Screening/baseline up to Year 5
Change from screening/baseline in the local electrical activity of the retina
Assessed using a system designed to record multifocal electroretinogram (ERG) responses from a number of locations at one time
Time frame: Screening/baseline up to Year 2
Change from screening/baseline in the electrical activity of the retina
Assessed using a system designed to record full-field electroretinogram (ERG) responses with Ganzfeld stimulation.
Time frame: Screening/baseline up to Year 5
Change from screening/baseline in Reading speed
Assessed using standard reading speed charts
Time frame: Screening/baseline up to Year 2
Change from screening/baseline in eye dominance
Dominant eye for viewing targets at distance
Time frame: Screening/baseline up to Year 5
Change from screening/baseline in Change from baseline in mobility test scores
Assessed using a system designed to measure the ability to navigate obstacles in a maze-like environment under varying light conditions
Time frame: Screening/baseline up to Year 2
Change from screening/baseline in the National Eye Institute - Visual function questionnaire 25 (NEI-VFQ 25) composite score
Questionnaire completed by the participant to measure the influence of visual impairment on quality of life
Time frame: Screening/baseline up to Year 5
Change from screening/baseline in the low luminance questionnaire (LLQ) responses
Questionnaire completed by the participant to assess visual problems under low luminance conditions, including nighttime
Time frame: Screening/baseline up to Year 5
Plan to share: Undecided — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
This study is completed, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Novartis Pharmaceuticals