Avalglucosidase Alfa Extension Study

Completed · Phase 2

Conditions studied: Glycogen Storage Disease Type II Pompe Disease

In brief

Primary Objective: Long-term safety and pharmacokinetics (PK) of avalglucosidase alfa Secondary Objective: Long-term effect of avalglucosidase alfa on pharmacodynamic variables

Key facts

Study ID

NCT02032524

Run by

Genzyme, a Sanofi Company

People needed

19

Starts

2014-02-27

Expected to finish

2022-12-12

Last updated by the study team

2024-03-01

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants with Pompe disease who previously completed an avalglucosidase study.
• The participant and/or their parent/legal guardian is willing and able to provide signed informed consent, and the participant, if <18 years of age, was willing to provide assent if deemed able to do so.
• The participant (and participant's legal guardian if participant is <18 years of age) must have had the ability to comply with the clinical protocol.
• The participant, if female and of childbearing potential, had to have a negative pregnancy test [urine beta-human chorionic gonadotropin] at baseline.

You may not qualify if…

• The participant was concurrently participating in another clinical study using investigational treatment.
• The participant, in the opinion of the Investigator, was unable to adhere to the requirements of the study.
• The participant had clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precluded participation in the study or potentially decreases survival.
• The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Where it is running

• Investigational Site Number 840006 — Phoenix, Arizona, United States
• Investigational Site Number 840010 — Jacksonville, Florida, United States
• Investigational Site Number 840001 — Kansas City, Kansas, United States
• Investigational Site Number 840008 — St Louis, Missouri, United States
• Investigational Site Number 840002 — Durham, North Carolina, United States
• Investigational Site Number 840011 — Cincinnati, Ohio, United States
• Investigational Site Number 840009 — Dallas, Texas, United States
• Investigational Site Number 840003 — Fairfax, Virginia, United States
• Investigational Site Number 056001 — Leuven, Belgium
• Investigational Site Number 208001 — København Ø, Denmark
• Investigational Site Number 250003 — Nice, France
• Investigational Site Number 250002 — Paris, France
• Investigational Site Number 276003 — Mainz, Germany
• Investigational Site Number 276001 — München, Germany
• Investigational Site Number 276002 — Münster, Germany
• Investigational Site Number 528001 — Rotterdam, Netherlands
• Investigational Site Number 826003 — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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