NCT04848779View on ClinicalTrials.gov

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Active, not recruitingOBSERVATIONAL
Enrollment

16

Participants

Timeline

Start Date

June 10, 2021

Primary Completion Date

October 28, 2026

Study Completion Date

October 28, 2026

Conditions
Glycogen Storage Disease Type II
Interventions
DRUG

Alglucosidase alfa GZ419829

"Pharmaceutical form: Lyophilized powder for solution~Route of administration: intravenous"

Trial Locations (15)

100

Investigational Site Number : 1580001, Taipei

3000

Investigational Site Number : 0560001, Leuven

10532

Advanced Medical Genetics- Site Number : 8400002, Hawthorne

20052

Investigational Site Number : 3800002, Monza

27710

Duke University Medical Center- Site Number : 8400004, Durham

35392

Investigational Site Number : 2760001, Giessen

37000

Investigational Site Number : 2500001, Tours

38103

Le Bonheur Children's Hospital- Site Number : 8400005, Memphis

45229

Cincinnati Children's Hospital Medical Center- Site Number : 8400001, Cincinnati

50139

Investigational Site Number : 3800001, Florence

98105

Seattle Children's Hospital- Site Number : 8400003, Seattle

3015 CE

Investigational Site Number : 5280001, Rotterdam

08950

Investigational Site Number : 7240001, Esplugues de Llobregat

WC1N 3JH

Investigational Site Number : 8260001, London

M13 9WL

Investigational Site Number : 8260002, Manchester

Sponsors

Lead Sponsor

Sanofi
All Listed Sponsors
lead

Sanofi

INDUSTRY

NCT04848779 - A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD) | Biotech Hunter | Biotech Hunter