For Healthcare Professionals

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:


  1. Male and female patients aged 18 to 70 years inclusive.
  2. The visual acuity in the study eye at the screening 1 visit should be no better than 60 ETDRS letters.
  3. 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.
  4. Visible photoreceptor (outer nuclear) and Retinal Pigment Epithelium (RPE) layers on standard OCT scan in the study eye at the screening 1 visit.


  1. History of hypersensitivity to the study drug or to drugs of similar classes or to any of the medications required in the perioperative period.
  2. Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints
  3. Any contraindication to the planned surgery or anesthesia as determined by the treating physician (surgeon, anesthesiologist, internist, or designee).
  4. 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
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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.
Phone iconCall +41613241111Email iconEmail Study Center

Study’s details


Retinitis Pigmentosa




Phase 1/Phase 2

Participants needed


Est. Completion Date

May 2026

Treatment type



Novartis identifier


Study number


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