Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

Genzyme, a Sanofi Company21 enrolled

Overview

* 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.

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 Type

INTERVENTIONAL

Allocation

Purpose

OTHER

Masking

NONE

Enrollment

Conditions

Pompe Disease Glycogen Storage Disease Type II (GSD II)

Interventions

alglucosidase alfaBIOLOGICAL

Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: 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. Exclusion Criteria: 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.

Locations (12)

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