Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease (PAPAYA)
Pompe Disease, Glycogen Storage Disease Type II (GSD II)
About this trial
This is an interventional other trial for Pompe Disease
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.
Sites / Locations
- Investigational Site Number 840008
- Investigational Site Number 840007
- Investigational Site Number 840005
- Investigational Site Number 840003
- Investigational Site Number 1028
- Investigational Site Number 356001
- Investigational Site Number 356002
- Investigational Site Number 643001
- Investigational Site Number 643002
- Investigational Site Number 804001
- Investigational Site Number 826003
- Investigational Site Number 826002
Arms of the Study
Arm 1
Experimental
Alglucosidase alfa
Participants received intravenous (IV) infusion of Alglucosidase alfa 20 milligrams per kilogram (mg/kg) body weight on Day 1. Infusion was administered at an initial rate of approximately 1 milligram per kilogram per hour (mg/kg/hr) with allowed rate increased of 2 mg/kg/hr every 30 minutes, if there were no signs of infusion-associated reactions (IARs), until a maximum rate of approximately 7 mg/kg/hr was reached.