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Gene Therapy Trial Report

Summary

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


NCTID NCT03374657 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Retinitis Pigmentosa
Disease Ontology Term DOID:0050683; DOID:0061127
Compound Name CPK850
Compound Description scAAV8.pRLBP1.hRLBP1
Sponsor Novartis Pharmaceuticals
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 12 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant RLBP1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV8
Dose 1 5E9 vg/eye (n=3)
Dose 2 1E10 vg/eye (n=3)
Dose 3 3E10 vg/eye (n=3)
Dose 4 1E11 vg/eye (n=3)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2017-12-11
Completion Date 2026-05-11
Last Update 2026-01-15

Participation Criteria


Eligible Age 18 Years - 70 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations Sweden

Regulatory Information


Has US IND False
FDA Designations
Recent Updates Novartis announced their decision to partner in July 2022

Resources/Links