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Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease — PAPAYA  

Pompe Disease Clinical Trial

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

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01410890
Study type Interventional
Source Sanofi
Contact
Status Completed
Phase Phase 4
Start date November 3, 2014
Completion date November 20, 2020
View Details
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