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Results for “Pompe Disease (Late-onset)”

70 trials

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Showing 20 of 70 results

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)Looking for participantsNCT07282847
What this trial is testing

Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

Who this might be right for
Pompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD
AskBio Inc 12
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 applicableStudy completedNCT02765828
What this trial is testing

Identification of Tongue Involvement in Late-Onset Pompe Disease

Who this might be right for
MyopathyNeuropathyGlycogen Storage Disease Type II (Late-onset Pompe Disease)
Duke University 73
Post-approval studies (Phase 4)Study completedNCT04676373
What this trial is testing

Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen

Who this might be right for
Pompe's Disease
Genzyme, a Sanofi Company 41
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
Not applicableUnknownNCT03045042
What this trial is testing

Search for Serum/Plasma Biomarkers in Pompe's Disease

Who this might be right for
Late Onset Pompe DiseasePompe Disease
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau 35
Not applicableStudy completedNCT00731081
What this trial is testing

Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 8
Not applicableStudy completedNCT00077662
What this trial is testing

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 61
Not applicableActive Not RecruitingNCT03694561
What this trial is testing

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)GAA Deficiency
Duke University 20
Not applicableActive Not RecruitingNCT04929002
What this trial is testing

Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases

Who this might be right for
Glycogen Storage DiseaseMcArdle DiseasePompe Disease (Late-onset)
Rigshospitalet, Denmark 50
Large-scale testing (Phase 3)Study completedNCT00125879
What this trial is testing

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 16
Large-scale testing (Phase 3)Study completedNCT03729362
What this trial is testing

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 125
Not applicableUnknownNCT00713245
What this trial is testing

A Long Term Follow up Study in Late-onset Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 70
Testing effectiveness (Phase 2)Study completedNCT00250939
What this trial is testing

RhGAA in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 5
Very early researchNot Yet RecruitingNCT06178432
What this trial is testing

Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease (Late-onset)
Huashan Hospital 6
Post-approval studies (Phase 4)Study completedNCT01288027
What this trial is testing

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 16
Not applicableStudy completedNCT03285126
What this trial is testing

Pompe Gene Therapy- Screening for Eligibility

Who this might be right for
Pompe Disease (Late-onset)
Duke University 19
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
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