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

• Register: ClinicalTrials.gov
• Last refreshed on: 8 January 2024
• Main ID: NCT03374657
• Date of registration: 11/12/2017
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
• Scientific title: 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
• Date of first enrolment: August 22, 2018
• Target sample size: 12
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03374657
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: Single (Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

Sweden

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Retinitis Pigmentosa

Intervention(s)

Biological: CPK850

Primary Outcome(s)

Number of responders in dark adaptation [Time Frame: Screening/baseline up to year 1]

Number of participants with adverse events (AEs), serious adverse events (SAEs) and deaths [Time Frame: Up to year 5]

Secondary Outcome(s)

Change from screening/baseline in Light-adapted microperimetry sensitivity [Time Frame: Screening/baseline up to year 1]

Change from screening/baseline in the National Eye Institute - Visual function questionnaire 25 (NEI-VFQ 25) composite score [Time Frame: Screening/baseline up to year 1]

Change from screening/baseline in Change from baseline in mobility test scores [Time Frame: Screening/baseline up to year 1]

Change from screening/baseline in Total contrast sensitivity score [Time Frame: Screening/baseline up to year 1]

Change from screening/baseline in the electrical activity of the retina [Time Frame: Screening/baseline up to year 1]

Number of patients with improvement in rod function in the treated eye vs the untreated eye [Time Frame: Screening/baseline up to year 1]

Number of patients with recovery of the cone system [Time Frame: Screening/baseline up to year 1]

Change from screening/baseline in eye dominance [Time Frame: Screening/baseline up to year 1]

Change from screening/baseline in Reading speed [Time Frame: Screening/baseline up to year 1]

Change from screening/baseline in the local electrical activity of the retina [Time Frame: Screening/baseline up to year 1]

Change from screening/baseline in the low luminance questionnaire (LLQ) responses [Time Frame: Screening/baseline up to year 1]

Change from screening/baseline in Visual field perimetry mean deviation [Time Frame: Screening/baseline up to year 1]

Secondary ID(s)

2016-002696-10

CCPK850X2202

Source(s) of Monetary Support

Please refer to primary and secondary sponsors