A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Stopped early · Phase 4

Conditions studied: Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency

In brief

A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.

Key facts

Study ID

NCT01597596

Run by

Genzyme, a Sanofi Company

People needed

4

Starts

2012-08-01

Expected to finish

2014-12-01

Last updated by the study team

2016-01-18

Who can join

Age: any, up to 1. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• The participant's parent/legal guardian was willing and able to provide signed informed consent.
• The participant might be less than or equal to 12 months of age.
• The participant might have documented GAA enzyme deficiency from blood, skin, or muscle tissue.
• The participant might be naïve to treatment with alglucosidase alfa.

You may not qualify if…

• The participant was cross-reactive immunologic material negative.
• The participant required invasive ventilator support at the time of enrollment.
• The participant had decompensated clinical heart failure.
• The participant had a major congenital abnormality, excluding cardiac hypertrophy.
• The participant had a clinically significant organ disease (excluding the signs and symptoms of Pompe disease).
• The participant was currently receiving any investigational product.
• The participant was participating in another clinical study.
• The participant and/or the patient's parent/legal guardian was unable to adhere to the requirements of the study.

Where it is running

• Study site — Little Rock, Arkansas, United States
• Study site — Oakland, California, United States
• Study site — Gainsville, Florida, United States
• Study site — Decatur, Georgia, United States
• Study site — Chicago, Illinois, United States
• Study site — Cambridge, Massachusetts, United States
• Study site — Detroit, Michigan, United States
• Study site — Las Vegas, Nevada, United States
• Study site — New Brunswick, New Jersey, United States
• Study site — New York, New York, United States
• Study site — Durham, North Carolina, United States
• Study site — Cincinnati, Ohio, United States
• Study site — Fort Worth, Texas, United States
• Study site — Seattle, Washington, United States
• Study site — Geiben, Germany
• Study site — Mainz, Germany
• Study site — Taipei, Taiwan

Full record on ClinicalTrials.gov

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