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

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

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
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type
Dose 1	Dose escalation with 4 levels, unknown concentrations
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	Sweden

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