A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

Stopped early · Phase 4

Conditions studied: Pompe Disease

In brief

The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.

Key facts

Study ID

NCT01526785

Run by

Genzyme, a Sanofi Company

People needed

113

Starts

2012-03-01

Expected to finish

2014-12-01

Last updated by the study team

2015-12-08

Who can join

Age: 1 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Study site — Phoenix, Arizona, United States
• Study site — Little Rock, Arkansas, United States
• Study site — Oakland, California, United States
• Study site — Orange, California, United States
• Study site — San Diego, California, United States
• Study site — San Jose, California, United States
• Study site — Stanford, California, United States
• Study site — Aurora, Colorado, United States
• Study site — Washington D.C., District of Columbia, United States
• Study site — Gainesville, Florida, United States
• Study site — Miami, Florida, United States
• Study site — Decatur, Georgia, United States
• Study site — Chicago, Illinois, United States
• Study site — Indianapolis, Indiana, United States
• Study site — Kansas City, Kansas, United States
• Study site — Louisville, Kentucky, United States
• Study site — Baton Rouge, Louisiana, United States
• Study site — Shreveport, Louisiana, United States
• Study site — Boston, Massachusetts, United States
• Study site — Detroit, Michigan, United States
• Study site — Grand Rapids, Michigan, United States
• Study site — Minneapolis, Minnesota, United States
• Study site — Jackson, Mississippi, United States
• Study site — Columbia, Missouri, United States
• Study site — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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