Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

Completed · Phase 4

Conditions studied: Pompe Disease, Glycogen Storage Disease Type II (GSD II)

In brief

* 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.

Key facts

Study ID

NCT01410890

Run by

Genzyme, a Sanofi Company

People needed

21

Starts

2014-11-03

Expected to finish

2020-11-20

Last updated by the study team

2022-03-28

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• A participant was to meet all of the following criteria to be eligible for this study:
• The participant and/or the participant's parent/legal guardian was willing and able to provide signed informed consent.
• The participant had a confirmed acid alpha-glucosidase (GAA) enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations.
• Infant and toddler Pompe disease participants could be included in the study only under condition (minimal body weight) that the trial-related blood loss (including any losses in the maneuver) would not exceed 3 percent (%) of the total blood volume during a period of 4 weeks and would not exceed 1 % at any single time.
• The participant, if female and of childbearing potential, must have had a negative pregnancy test (urine beta-human chorionic gonadotropin) at screening. Note: All female participants of childbearing potential and sexually mature males must have agreed to use a medically accepted method of contraception throughout the study.
• For participants previously treated with alglucosidase alfa the participant had received alglucosidase alfa for at least 6 months.

You may not qualify if…

• A participant who met any of the following criteria was excluded from this study:
• The participant was participating in another clinical study using an investigational product.
• The participant, in the opinion of the Investigator, was unable to adhere to the requirements of the study.
• The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Where it is running

• Investigational Site Number 840008 — Valhalla, New York, United States
• Investigational Site Number 840007 — Cincinnati, Ohio, United States
• Investigational Site Number 840005 — Salt Lake City, Utah, United States
• Investigational Site Number 840003 — Fairfax, Virginia, United States
• Investigational Site Number 1028 — Sofia, Bulgaria
• Investigational Site Number 356001 — New Delhi, India
• Investigational Site Number 356002 — Vellore, India
• Investigational Site Number 643001 — Moscow, Russia
• Investigational Site Number 643002 — Moscow, Russia
• Investigational Site Number 804001 — Kiev, Ukraine
• Investigational Site Number 826003 — Birmingham, United Kingdom
• Investigational Site Number 826002 — Salford, United Kingdom

Full record on ClinicalTrials.gov

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