Found 52 matching trials
A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD) N/A Primary Objective:
To describe the effect of routine practice with algluco...
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Active Not Recruiting
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Advanced Medical Genetics- Site Number : 8400002
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Pompe Pregnancy Sub-Registry N/A This Sub-registry is a multicenter, international, longitudinal, observatio...
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Barrow Neurol Group- Site Number : 840087
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rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) PHASE1 Glycogen Storage Disease Type II ("GSD-II"; also known as Pompe disease) is...
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University of Florida College of Medicine
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Treatment Frequency Reduction in Pompe Disease PHASE4 The aim of this study is to assess if dosing frequency reduction of algluco...
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Multiple Locations
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Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 PHASE2 Pompe disease (also known as glycogen storage disease type II) is caused by...
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University of Alabama
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BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) PHASE3 Study 701-301 is a single-arm, open-label, switchover study in patients wit...
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Neuromuscular Research Centre
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A Global Prospective Observational Registry of Patients With Pompe Disease N/A This is a global, multicenter, prospective, observational registry of patie...
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University of Arkansas Medical Science
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Immune Tolerance Induction Study PHASE4 An exploratory, open-labeled study of participants with Pompe disease, who ...
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Multiple Locations
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A Study of rhGAA in Patients With Late-Onset Pompe Disease PHASE2 Pompe disease (also known as glycogen storage disease Type II) is caused by...
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Sophia Kinderziekenhuis, Erasmus MC
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Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients. PHASE1 Primary Objective:
To evaluate the safety and tolerability of neoGAA in tr...
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Investigational Site Number 840006
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