Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

NCTID	NCT04411654 (View at clinicaltrials.gov)
Description	J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2). For each patient, the study will be approximately 5 years in duration. During the first 12 months after dosing, patients will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will be followed up for an additional 4 years to monitor safety and changes on selected biomarkers and clinical outcomes. (Show More)
Indication	Gaucher Disease, Type 1
Compound Name	LY3884961 (PR001) AAV9-CMV-CBA-GBA1
Sponsor	Prevail Therapeutics
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	15

Target Gene/Variant	GBA1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracisterna magna
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type
Dose 1	Undisclosed low dose
Dose 2	Undisclosed high dose
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States,United Kingdom

Has US IND	True
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