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 was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (LumizymeŽ) in Pompe participants.
Stanford is now accepting new patients for this trial.
- drug : Alglucosidase alfa
Phase: Phase 4
Ages Eligible For Study:
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.