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

Novartis Pharmaceuticals12 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Retinitis Pigmentosa

Interventions

CPK850BIOLOGICAL

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

Eligibility

Sex: ALLMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

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

Locations (1)

Novartis Investigative Site

Stockholm, Sweden

Outcomes

Primary Outcomes

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

Safety events

Time frame: 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.

Time frame: Screening/baseline up to Year 1

Secondary Outcomes

Number of responders with recovery of the cone system

cone recovery during dark adaptation

Time frame: Screening/baseline up to Year 1

Change from screening/baseline in Visual field perimetry mean deviation

Assessed using automated static perimetry

Time frame: Screening/baseline up to Year 2

Change from screening/baseline in Total contrast sensitivity score

Contrast sensitivity (ie, the ability to detect relatively dim objects) will be assessed

Time frame: Screening/baseline up to Year 2

Change from screening/baseline in Light-adapted microperimetry sensitivity

Assessed using standard microperimetry equipment

Time frame: Screening/baseline up to Year 5

Change from screening/baseline in the local electrical activity of the retina

Assessed using a system designed to record multifocal electroretinogram (ERG) responses from a number of locations at one time

Data from ClinicalTrials.gov

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