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Results for “Pompe Disease (Late-Onset)”

66 trials

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Showing 20 of 66 results

Large-scale testing (Phase 3)Study completedNCT00158600
What this trial is testing

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) 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 90
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
Not applicableStudy completedNCT02354664
What this trial is testing

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 14
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
Not applicableStudy completedNCT00074932
What this trial is testing

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIGlycogenosis 2
Genzyme, a Sanofi Company 9
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
Not applicableUnknownNCT05272969
What this trial is testing

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more
LMU Klinikum 95
Large-scale testing (Phase 3)Ended earlyNCT01924845
What this trial is testing

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Who this might be right for
Late-onset Pompe Disease
BioMarin Pharmaceutical 24
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)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
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 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
Testing effectiveness (Phase 2)Active Not RecruitingNCT04174105
What this trial is testing

Gene Transfer Study in Patients With Late Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Astellas Gene Therapies 11
Early research (Phase 1)Not Yet RecruitingNCT07354724
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
Testing effectiveness (Phase 2)UnknownNCT06130228
What this trial is testing

Nutritional Therapy in Late-onset Pompe Disease

Who this might be right for
Pompe DiseaseMuscle LossObesity+5 more
McMaster University 28
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
Testing effectiveness (Phase 2)Ended earlyNCT02898753
What this trial is testing

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
Valerion Therapeutics, LLC 12
Not applicableActive Not RecruitingNCT06150820
What this trial is testing

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Astellas Gene Therapies 119
Post-approval studies (Phase 4)Not Yet RecruitingNCT06575829
What this trial is testing

Treatment Frequency Reduction in Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)GAA DeficiencyGlycogen Storage Disease Type II+1 more
Erasmus Medical Center 10
Testing effectiveness (Phase 2)Study completedNCT01230801
What this trial is testing

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
BioMarin Pharmaceutical 22
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