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

Novartis

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: Retinitis Pigmentosa

Treatments: Biological: CPK850

Study type: Interventional

Funder types: Industry

Identifiers

NCT03374657

2016-002696-10 (EudraCT Number)

CCPK850X2202

Details and patient eligibility

About

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.

Full description

This study will potentially include 4 cohorts with a minimum of 3 patients per cohort. This trial design used a staggered patient enrollment with continuous data reviews to limit as much unforeseen risk as possible prior to enrolling each patient in each cohort or initiating another cohort. Only one eye (designated as the study or treated eye) will be dosed per patient. Each patient will be followed for 5 years after the subretinal injection of CPK850.

Enrollment: 12 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: Single Blind

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12 participants in 4 patient groups

CPK Dose 1 (lowest dose)

Experimental group

Description:

CPK850, one subretinal injection to the study eye

Treatment:

Biological: CPK850

CPK Dose 2 (next lowest dose)

Experimental group

Description:

CPK850, one subretinal injection to the study eye

Treatment:

Biological: CPK850

CPK Dose 3 (third lowest dose)

Experimental group

Description:

CPK850, one subretinal injection to the study eye

Treatment:

Biological: CPK850

CPK Dose 4 (highest dose)

Experimental group

Description:

CPK850, one subretinal injection to the study eye

Treatment:

Biological: CPK850