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

Launched by NOVARTIS PHARMACEUTICALS · Dec 11, 2017

Keywords

ClinConnect Summary

This clinical trial is testing a new treatment called CPK850 for patients with a type of vision loss known as RLBP1 retinitis pigmentosa. This condition affects the retina, the part of the eye that helps us see, and is caused by specific gene mutations. The study aims to find out the highest dose of CPK850 that can be safely given to patients and to see if it can help improve their vision.

To participate in this trial, individuals need to be between 18 and 70 years old and have certain visual impairments related to their condition. They must also have a confirmed diagnosis of RLBP1 retinitis pigmentosa through genetic testing. Participants will receive the treatment and their vision will be closely monitored to assess safety and effectiveness. It's important to note that individuals with certain eye conditions or who are pregnant or breastfeeding will not be eligible for this trial.

Gender

ALL

Eligibility 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