Status:
ACTIVE_NOT_RECRUITING
A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Retinitis Pigmentosa
Eligibility:
All Genders
18-70 years
Phase:
PHASE1
PHASE2
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...
Detailed 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 ri...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
August 22 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 11 2026
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT03374657
Start Date
August 22 2018
End Date
May 11 2026
Last Update
January 10 2025
Active Locations (1)
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1
Novartis Investigative Site
Stockholm, Sweden, SE-112 82