A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Glycogenosis 2 Clinical Trial

Official title:
A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Status Terminated

Clinical Trial Summary

A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.

Clinical Trial Description

n/a

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT01597596
Study type	Interventional
Source	Sanofi
Contact
Status	Terminated
Phase	Phase 4
Start date	August 2012
Completion date	December 2014

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT00158600` - A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease	Phase 3
Completed	`NCT00074932` - Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease	N/A
Completed	`NCT00025896` - Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease	Phase 2
Completed	`NCT00051935` - A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II	Phase 2
Completed	`NCT00250939` - A Study of rhGAA in Patients With Late-Onset Pompe Disease	Phase 2
Completed	`NCT00053573` - rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)	Phase 1/Phase 2
Completed	`NCT00268944` - Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support	Phase 3
Approved for marketing	`NCT00074919` - Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease	N/A
Approved for marketing	`NCT00520143` - Alglucosidase Alfa Temporary Access Program	N/A