Trial Search Results

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

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

Stanford is currently accepting patients for this trial.

Lead Sponsor:

Genzyme, a Sanofi Company

Stanford Investigator(s):


  • Drug: Alglucosidase alfa


Phase 4


Inclusion Criteria:

A participant might meet all of the following criteria to be eligible for this study.

   1. The participant and/or their parent/legal guardian was willing and able to provide
   signed informed consent.

   2. The participant might be at least 1 year of age at the time of informed consent.

   3. The participant had a diagnosis of Pompe disease and might have received treatment
   with 160 L alglucosidase alfa prior to screening.

   4. 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 Criteria:

A participant who met any of the following criteria were to be excluded from this study.

   1. 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.

   2. 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.

   3. The participant and/or their parent/legal guardian, in the opinion of the
   Investigator, was unable to adhere to the requirements of the study.

Ages Eligible for Study

1 Year - N/A

Genders Eligible for Study


Now accepting new patients

Contact Information

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