A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale

The objective of this study is to evaluate the efficacy and safety of treatment with 4000 L alglucosidase alfa (LumizymeŽ) in Pompe patients.

Stanford is now accepting new patients for this trial. Please contact Shirley Paulose at 650-724-3792 for more information.



  • drug : Alglucosidase alfa

Phase: Phase 4


Ages Eligible For Study:

1 Year - N/A

Inclusion Criteria

A patient must meet all of the following criteria to be eligible for this study. 1. The patient and/or their parent/legal guardian is willing and able to provide signed informed consent. 2. The patient must be at least 1 year of age at the time of informed consent. 3. The patient has a diagnosis of Pompe disease and must have received treatment with 160 L alglucosidase alfa prior to screening. 4. The patient, if female and of childbearing potential, must have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: all female patients of childbearing potential and sexually mature males must agree to use a medically accepted method of contraception throughout the study.

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Contact information

Primary Contact:

Shirley Paulose 650-724-3792

Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305

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