NCT04411654 - Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

June 29, 2021

Primary Completion Date

May 31, 2028

Study Completion Date

May 31, 2028

Conditions

Gaucher Disease, Type 2

Interventions

GENETIC

LY3884961

Participants will receive a single dose of LY3884961 administered intracisternally.

DRUG

Methylprednisolone

Single IV pulse administered as concomitant medication.

DRUG

Sirolimus

Loading dose, followed by maintenance doses, followed by dose tapering; administered as concomitant medication.

DRUG

Prednisone

Administered orally as concomitant medication, followed by dose tapering.

Trial Locations (5)

Children's Hospital of Pittsburgh, 4401 Penn Avenue, Pittsburgh

Lysosomal Rare Disorders Research and Treatment Center, Fairfax

University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue, Minneapolis

UCSF Benioff Children's Hospital, 5700 Martin Luther King Jr Way, Oakland

Manchester Centre for Genomic Medicine, 6th Floor, St Mary's Hospital, Oxford Road, Manchester