Status:
TERMINATED
A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Pompe Disease
Eligibility:
All Genders
1+ years
Phase:
PHASE4
Brief Summary
The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.
Eligibility Criteria
Inclusion
- A participant might meet all of the following criteria to be eligible for this study.
- The participant and/or their parent/legal guardian was willing and able to provide signed informed consent.
- The participant might be at least 1 year of age at the time of informed consent.
- The participant had a diagnosis of Pompe disease and might have received treatment with 160 L alglucosidase alfa prior to screening.
- The participant, if female and of childbearing potential, might have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: all female participants of childbearing potential and sexually mature males might agree to use a medically accepted method of contraception throughout the study.
Exclusion
- A participant who met any of the following criteria were to be excluded from this study.
- The participant had within the past 3 months received or was currently receiving any investigational product other than 160 L alglucosidase alfa or was currently participating in another clinical treatment study.
- The participant, in the opinion of the Investigator, was clinically unstable and would not be expected to survive to completion of the 52-week treatment period.
- The participant and/or their parent/legal guardian, in the opinion of the Investigator, was unable to adhere to the requirements of the study.
Key Trial Info
Start Date :
March 1 2012
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2014
Estimated Enrollment :
113 Patients enrolled
Trial Details
Trial ID
NCT01526785
Start Date
March 1 2012
End Date
December 1 2014
Last Update
December 8 2015
Active Locations (51)
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1
Birmingham, Alabama, United States
2
Phoenix, Arizona, United States
3
Little Rock, Arkansas, United States
4
Oakland, California, United States