Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

43

Participants

Timeline

Start Date

April 18, 2022

Primary Completion Date

October 2, 2025

Study Completion Date

October 30, 2026

Conditions
Gaucher's Disease Type III
Interventions
DRUG

Venglustat

tablet; oral

DRUG

imiglucerase

sterile lyophilized product; intravenous

Trial Locations (22)

1426

Hospital de Ninos - Investigational Site Number: 320001, Buenos Aires

10041

National Taiwan University Hospital-Investigational Site Number: 1580001, Taipei

20122

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Investigational Site Number : 3800003, Milan

22030

Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001, Fairfax

34093

Istanbul University Medical Faculty Hospital-Investigational Site Number : 7920004, Istanbul

52242

University of Iowa - Investigational Site Number: 8400002, Iowa City

65239

SphinCS GmbH - Investigational Site Number: 2760001, Hochheim am Main

75013

47-87, boulevard de l'hôpital - Investigational Site Number: 2500003, Paris

75015

Hopital Necker - Investigational Site Number: 2500001, Paris

75230

Texas Oncology - Medical City Dallas Site Number : 8400008, Dallas

80131

"Azienda Ospedaliera Universitaria (AOU) Federico II - Investigational Site Number: 3800002", Napoli

100005

Peking Union Medical College Hospital - Investigational Site Number: 1560001, Beijing

200092

Xinhua Hospital - Investigational Site Number: 1560004, Shanghai

510080

The First Affiliated Hospital - Investigational Site Number: 1560002, Guangzhou

06511

Yale University School of Medicine - Investigational Site Number: 8400003, New Haven

R3E 3P4

Children's Hospital Research Institute of Manitoba - Investigational Site Number: 1240001, Winnipeg

H-4032

Debreceni Egyetem, Klinikai Központ, Reumatológiai Klinika - Investigational Site Number: 3480001, Debrecen

250-8558

Odawara Municipal Hospital-Investigational Site Number : 3920002, Odawara

980-8574

Tohoku University School of Medicine - Investigational Site Number: 3920001, Sendai

01790

Cukurova University Medical School Hospital-Investigational Site Number : 7920001, Adana

06500

Gazi University Medical Hospital-Investigational Site Number : 7920002, Ankara

NW3-2PF

Investigational Site Number : 8260001, London

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Sanofi

INDUSTRY