A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing The Efficacy And Safety Of Repeated Biweekly Infusions Of Neogaa (Gz402666) And Alglucosidase Alfa In Treatment-Naïve Patients With Late Onset Pompe Disease
Posted Date: Oct 6, 2017
- Investigator: Hani Kushlaf
- Specialties: Neurology, Neuromuscular Disorders
- Type of Study: Drug
The purpose of this study is to evaluate the possible risks and efficacy (improvement of disease), as well as pharmacokinetics (breakdown, use and removal from the body of the study drug (neoGAA) of the study drug (neoGAA) compared to Myozyme®/Lumizyme® (alglucosidase alfa) in the treatment of patients with late onset Pompe disease.
To Be Considered For This Study, You Must Have Been Diagnosed With Late Onset Pompe Disease (Acid A Glucosidase (Gaa) Deficiency) And Have Never Received The Study Drug (Neogaa), Myozyme®/Lumizyme® Or Other Investigational Drug In A Clinical Study For Your Disease. You Cannot Participate In Another Clinical Study Using Investigational Treatment During The Same Time You Participate In Efc14028. If You Have Previously Received Or Currently Use Immune Tolerance Induction Therapy, You Cannot Participate.
Abnormal Emg, Myopathy, Pompe, Elevated Liver Enzymes, Enzyme Replacement Therapy
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