A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
NCT ID: NCT03374657
Last Updated: 2026-01-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
12 participants
INTERVENTIONAL
2018-08-22
2026-05-11
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
SINGLE
At the clinical sites, there will be an unmasked ophthalmologist. The remaining assessors at the clinical sites (ophthalmologist, study nurse, ophthalmic technician, etc) doing the ophthalmic examinations should be masked to the study (treated) eye.
The following unmasked sponsor roles are required for this study:
Sponsor clinical staff required to assist in the management and re-supply of investigational drug product.
The independent committee assessing unmasked interim results and the independent analysis team.
All other sponsor staff will stay masked to treatment assignments
Study Groups
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CPK Dose 1 (lowest dose)
CPK850, one subretinal injection to the study eye
CPK850
In one of 4 dose levels administered via subretinal injection under anesthesia
CPK Dose 2 (next lowest dose)
CPK850, one subretinal injection to the study eye
CPK850
In one of 4 dose levels administered via subretinal injection under anesthesia
CPK Dose 3 (third lowest dose)
CPK850, one subretinal injection to the study eye
CPK850
In one of 4 dose levels administered via subretinal injection under anesthesia
CPK Dose 4 (highest dose)
CPK850, one subretinal injection to the study eye
CPK850
In one of 4 dose levels administered via subretinal injection under anesthesia
Interventions
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CPK850
In one of 4 dose levels administered via subretinal injection under anesthesia
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
18 Years
70 Years
ALL
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Novartis Pharmaceuticals
Role: STUDY_DIRECTOR
Novartis Pharmaceuticals
Locations
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Novartis Investigative Site
Stockholm, , Sweden
Countries
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References
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Kvanta A, Rangaswamy N, Holopigian K, Watters C, Jennings N, Liew MSH, Bigelow C, Grosskreutz C, Burstedt M, Venkataraman A, Westman S, Geirsdottir A, Stasi K, Andre H. Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial. Nat Commun. 2024 Sep 10;15(1):7438. doi: 10.1038/s41467-024-51575-4.
Other Identifiers
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2016-002696-10
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CCPK850X2202
Identifier Type: -
Identifier Source: org_study_id
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