NCT03374657CompletedPhase 1/2
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
Source: ClinicalTrials.gov · Last updated 11 Jun 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 12 (actual)
- First submitted
- 11 Dec 2017
- Start date
- 22 Aug 2018
- Primary completion date
- 12 May 2026
- Lead sponsor
- Novartis Pharmaceuticals
- Diseases
- Retinitis pigmentosa
- NCT number
- NCT03374657
- EU CT number
- 2023-508688-54-00
- EudraCT number
- 2016-002696-10
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 12
- From 22 Aug 2018 to the primary completion date, 12 May 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Any contraindication to the planned surgery or anesthesia as determined by the treating physician (surgeon, anesthesiologist, internist, or designee).
Other illness
- 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
Other
- 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
Who the study is looking for
- 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.
Age in the registry: 18 years to 70 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| St Erik Eye Hospital Coordinator of this site? Confirm its status | Solna | Sweden | Completed |
This page is information, not medical advice. Discuss any trial with your doctor.