Summary

Eligibility
for people ages 0 months to 24 months (full criteria)
Location
at Oakland, California and other locations
Dates
study started
completion around

Description

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.

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

Keywords

Gaucher Disease, Type 2, Gaucher Disease, GD, Gaucher, Type 2 Gaucher, Neuronopathic Gaucher, nGD, AAV9, GBA, Gene Therapy, Glucocerebrosidase, GBA1 mutation, Infants, Sirolimus, Prednisone, Methylprednisolone, Methylprednisolone Acetate, Methylprednisolone Hemisuccinate, Prednisolone, Prednisolone acetate, Prednisolone hemisuccinate, Prednisolone phosphate

Eligibility

Locations

  • UCSF Benioff Children's Hospital, 5700 Martin Luther King Jr Way
    Oakland California 94609 United States
  • University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue
    Minneapolis Minnesota 55454 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Prevail Therapeutics
ID
NCT04411654
Phase
Phase 1/2 Gaucher Disease Research Study
Study Type
Interventional
Participants
Expecting 15 study participants
Last Updated