NCT01410890

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

* The primary objective of this study was to characterize the pharmacokinetics (PK) of alglucosidase alfa manufactured at the 4000 L scale in participants who had a confirmed diagnosis of Pompe disease. * A secondary objective of this study was to evaluate and explore the relationship between anti-recombinant human acid alpha-glucosidase antibody titers and the PK of alglucosidase alfa.

Detailed description

The total study duration per participant was 4 to 8 weeks that consisted of a screening period (from 2 days to 4 weeks), treatment visit (1 day), and a follow up call (greater than or equal to 30 days). Two participants enrolled prior to protocol amendment 2 (dated 17 December 2015), which changed the study to single-dose, and were treated for 26 weeks, with a 4-week follow up.

Conditions

• Pompe Disease
• Glycogen Storage Disease Type II (GSD II)

Interventions

Type	Name	Description
BIOLOGICAL	alglucosidase alfa	Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)

Timeline

• Start date: 2014-11-03
• Primary completion: 2020-11-20
• Completion: 2020-11-20
• First posted: 2011-08-05
• Last updated: 2022-03-28
• Results posted: 2021-06-11

Locations

12 sites across 6 countries: United States, Bulgaria, India, Russia, Ukraine, United Kingdom