Status:
COMPLETED
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Pompe Disease
Glycogen Storage Disease Type II (GSD II)
Eligibility:
All Genders
Phase:
PHASE4
Brief 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 diseas...
Detailed Description
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...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
November 3 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 20 2020
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT01410890
Start Date
November 3 2014
End Date
November 20 2020
Last Update
March 28 2022
Active Locations (12)
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1
Investigational Site Number 840008
Valhalla, New York, United States, 10595
2
Investigational Site Number 840007
Cincinnati, Ohio, United States, 45219
3
Investigational Site Number 840005
Salt Lake City, Utah, United States, 84108
4
Investigational Site Number 840003
Fairfax, Virginia, United States, 22030