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

Gaucher Disease, Type 2 Clinical Trial

Official title:
An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose LY3884961 in Infants With Type 2 Gaucher Disease

Status Active, not recruiting

Clinical Trial Summary

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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04411654
Study type	Interventional
Source	Prevail Therapeutics
Contact
Status	Active, not recruiting
Phase	Phase 1/Phase 2
Start date	June 29, 2021
Completion date	May 2028

View Details

See also
	Status	Clinical Trial	Phase
	Completed	`NCT04470713` - Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
	Recruiting	`NCT04532047` - In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases	Phase 1