Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

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.

Timeline

Start

2014-11-03

Primary completion

2020-11-20

Completion

2020-11-20

Publications

• Nicolas X, Hurbin F, Periquet M, Richards S, Sensinger C, Welch K, An Haack K. Pharmacokinetics of Alglucosidase Alfa Manufactured at the 4000-L Scale in Participants with Pompe Disease: A Phase 3/4 Open-Label Study. Clin Pharmacol Drug Dev. 2023 Dec;12(12):1185-1193. doi: 10.1002/cpdd.1314. Epub 2023 Sep 13.

Drugs

Indications