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Results for “Glycogen Storage Disease Type II Late Onset”

71 trials

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Showing 20 of 71 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)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
Not applicableUnknownNCT05951790
What this trial is testing

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease (Late-onset)
Fondazione Don Carlo Gnocchi ETS 20
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
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
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
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableStudy completedNCT03893240
What this trial is testing

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+3 more
Spark Therapeutics, Inc. 61
Large-scale testing (Phase 3)Active Not RecruitingNCT03911505
What this trial is testing

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 21
Not applicableLooking for participantsNCT05989620
What this trial is testing

Long-Term Development of Muscular Dystrophy Outcome Assessments

Who this might be right for
LGMD1BLGMD1CLGMD1D+26 more
Virginia Commonwealth University 1,000
Not applicableStudy completedNCT07685314
What this trial is testing

LATE-ONSET POMPE DISEASE AND CEREBROVASCULAR MANIFESTATIONS

Who this might be right for
Late-Onset Pompe Disease
Hospitales Universitarios Virgen del Rocío 477
Large-scale testing (Phase 3)Study completedNCT00268944
What this trial is testing

Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support

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 completedNCT02654886
What this trial is testing

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

Who this might be right for
Pompe DiseaseMuscle Weakness
University of California, Irvine 10
Not applicableApproved For MarketingNCT00520143
What this trial is testing

Alglucosidase Alfa Temporary Access Program

Who this might be right for
Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-Onset)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company
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
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 applicableLooking for participantsNCT00231400
What this trial is testing

Pompe Disease Registry Protocol

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease
Genzyme, a Sanofi Company 2,000
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
Not applicableStudy completedNCT00566878
What this trial is testing

Pompe Lactation Sub-Registry

Who this might be right for
Glycogen Storage DiseasePompe Disease
Genzyme, a Sanofi Company 2
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
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