A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa

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

ClinicalTrials.gov Identifier: NCT03374657

Novartis Reference Number: CCPK850X2202

Last Update: May 17, 2022

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

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.

Retinitis Pigmentosa
Phase 1
Phase 2
Overall status 
Start date 
Aug 22, 2018
Completion date 
May 11, 2026
18 Years - 70 Years (Adult, Older Adult)


In one of 5 dose levels administered via subretinal injection under anesthesia

Eligibility 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:

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

Novartis Investigative Site
Stockholm, SE-112 82


Novartis Pharmaceuticals

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