A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
About the study
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.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Inclusion Criteria:
- Male and female patients aged 18 to 70 years inclusive.
- The visual acuity in the study eye at the screening 1 visit should be no better than 60 ETDRS letters.
- Clinical diagnosis of Bothnia dystrophy, Newfoundland rod-cone dystrophy or other progressive retinitis pigmentosa phenotype with mutations in the RLBP1 gene verified by genetic testing.
- Visible photoreceptor (outer nuclear) and Retinal Pigment Epithelium (RPE) layers on standard OCT scan in the study eye at the screening 1 visit.
EXCLUSION CRITERIA
Exclusion Criteria:
- History of hypersensitivity to the study drug or to drugs of similar classes or to any of the medications required in the perioperative period.
- Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints
- Any contraindication to the planned surgery or anesthesia as determined by the treating physician (surgeon, anesthesiologist, internist, or designee).
- Women who are pregnant, or lactating or women of childbearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for two months after treatment
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Retinitis Pigmentosa
Age (in years)
18 - 70
Phase
Phase 1/Phase 2
Participants needed
12
Est. Completion Date
May 11, 2026
Treatment type
Interventional
Sponsor
Novartis
ClinicalTrials.gov identifier
NCT03374657
Study number
CCPK850X2202
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