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

NCT03374657 | Active Not Recruiting Phase 1 DMC

• 2 other identifiers: CCPK850X22022016-002696-10
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Retinitis Pigmentosa

Keywords

Clinical trials, dark adaptation, gene therapy, RLBP1 mutation, retinitis pigmentosa.

Outcome Measures

Primary Outcomes (2)

• Number of participants with adverse events (AEs), serious adverse events (SAEs) and deaths

  Safety events

  Up to year 5

• Number of responders in dark adaptation

  A patient is considered a responder if sensitivity recovery values at 1 hour post-bleach are observed to be outside of the patient's prediction interval at ≥2 consecutive post-treatment visits within one year after treatment.

  Screening/baseline up to year 1

Secondary Outcomes (12)

• Number of patients with recovery of the cone system

  Screening/baseline up to year 1

• Number of patients with improvement in rod function in the treated eye vs the untreated eye

  Screening/baseline up to year 1

• Change from screening/baseline in Visual field perimetry mean deviation

  Screening/baseline up to year 1

• Change from screening/baseline in Total contrast sensitivity score

  Screening/baseline up to year 1

• Change from screening/baseline in Light-adapted microperimetry sensitivity

  Screening/baseline up to year 1

Study Arms (4)

CPK Dose 1 (lowest dose)

EXPERIMENTAL

CPK850, one subretinal injection to the study eye

Biological: CPK850

CPK Dose 2 (next lowest dose)

EXPERIMENTAL

CPK850, one subretinal injection to the study eye

Biological: CPK850

CPK Dose 3 (third lowest dose)

EXPERIMENTAL

CPK850, one subretinal injection to the study eye

Biological: CPK850

CPK Dose 4 (highest dose)

EXPERIMENTAL

CPK850, one subretinal injection to the study eye

Biological: CPK850

Interventions

CPK850 BIOLOGICAL

In one of 4 dose levels administered via subretinal injection under anesthesia

CPK Dose 1 (lowest dose); CPK Dose 2 (next lowest dose); CPK Dose 3 (third lowest dose); CPK Dose 4 (highest dose)

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis Investigative Site

Stockholm, SE-112 82, Sweden

Location

Related Publications (1)

• 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.

  PMID: 39256350 DERIVED

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Masking Details: This is a partially masked study. The patients will not be masked. The treating physicians and personnel at the surgical location (surgeons, anesthesiologist, operating room personnel and others) will not be masked. 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
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a non-confirmatory, open-label single ascending dose gene replacement-therapy study to assess safety, tolerability and efficacy of CPK850 in patients with RLBP1 retinitis pigmentosa due to biallelic mutations in the RLBP1 gene. The trial design uses a staggered patient enrollment.

Study Record Dates

• First Submitted: December 11, 2017
• First Posted: December 15, 2017
• Study Start: August 22, 2018
• Primary Completion (Estimated): May 11, 2026
• Study Completion (Estimated): May 11, 2026
• Last Updated: January 15, 2026

Record last verified: 2026-01

Locations

SE (1)