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Results for “Enzyme Replacement Therapy in Pompe Disease”

19 trials

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Showing 19 of 19 results

Testing effectiveness (Phase 2)WithdrawnNCT04094948
What this trial is testing

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Duke University
Not applicableUnknownNCT04755751
What this trial is testing

Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.

Who this might be right for
Pompe Disease Infantile-Onset
Rambam Health Care Campus 4
Not applicableStudy completedNCT03147664
What this trial is testing

Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease

Who this might be right for
Enzyme Replacement Therapy in Pompe Disease
Rambam Health Care Campus 5
Not applicableUnknownNCT00713453
What this trial is testing

Muscle Response to Enzyme Replacement Therapy in Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 16
Large-scale testing (Phase 3)Study completedNCT02782741
What this trial is testing

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II;Pompe's Disease
Genzyme, a Sanofi Company 101
Testing effectiveness (Phase 2)Looking for participantsNCT07123155
What this trial is testing

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 45
Testing effectiveness (Phase 2)Study completedNCT01885936
What this trial is testing

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

Who this might be right for
Pompe Disease
Duke University 16
Testing effectiveness (Phase 2)Study completedNCT00051935
What this trial is testing

The Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 2
Testing effectiveness (Phase 2)Study completedNCT00765414
What this trial is testing

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

Who this might be right for
Pompe Disease Late-OnsetGlycogen Storage Disease Type II GSD II
Genzyme, a Sanofi Company 1
Not applicableUnknownNCT04942912
What this trial is testing

Effect of Enzyme Replacement Therapy in Patients With Juvenile-onset Pompe Disease

Who this might be right for
Pompe's Disease Juvenile Onset
Central Hospital, Nancy, France 10
Not applicableStudy completedNCT01451879
What this trial is testing

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

Who this might be right for
Pompe Disease
University of Florida 11
Testing effectiveness (Phase 2)Study completedNCT00763932
What this trial is testing

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

Who this might be right for
Pompe Disease Infantile-OnsetGlycogen Storage Disease Type II
Genzyme, a Sanofi Company 7
Not applicableEnded earlyNCT03347253
What this trial is testing

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

Who this might be right for
Late-onset Pompe Disease
Amicus Therapeutics 12
Not applicableUnknownNCT05448131
What this trial is testing

ERT in Pompe Disease: Elucidation of Molecular Structures Contributing to Enzyme Uptake and Immunoreactivity

Who this might be right for
Pompe-Disease
Centre for Analytical Biochemistry and Biomedical Mass Spectrometry 50
Early research (Phase 1)Ended earlyNCT02185651
What this trial is testing

A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction

Who this might be right for
Pompe DiseaseHypersensitivity Reaction
University of Florida 2
Testing effectiveness (Phase 2)Study completedNCT01942590
What this trial is testing

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

Who this might be right for
Pompe Disease
Dwight Koeberl, M.D., Ph.D. 17
Early research (Phase 1)Looking for participantsNCT07354724
What this trial is testing

Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Who this might be right for
Late-onset Pompe Disease
Denali Therapeutics Inc. 32
Early research (Phase 1)Study completedNCT01859624
What this trial is testing

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Duke University 8
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07750990
What this trial is testing

An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 60