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Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

NCT01410890

CompletedPhase 4

Sponsor: Genzyme, a Sanofi Company

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

Interventions: alglucosidase alfa

Countries: United States, Bulgaria, India, Russia, Ukraine, United Kingdom

* 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.

Eligibility overview

Sex: ALL

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
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)
  • Valhalla, New York, United States
  • Cincinnati, Ohio, United States
  • Salt Lake City, Utah, United States
  • Fairfax, Virginia, United States
  • Sofia, Bulgaria
  • New Delhi, India
  • Vellore, India
  • Moscow, Russia
  • Moscow, Russia
  • Kiev, Ukraine
  • Birmingham, United Kingdom
  • Salford, United Kingdom