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Results for “GAA Deficiency”

59 trials

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Showing 20 of 59 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
Not applicableEnded earlyNCT00277979
What this trial is testing

3D Echocardiography Managing Infantile Pompe's Disease

Who this might be right for
Congenital Disorders
Children's Healthcare of Atlanta 2
Not applicableStudy completedNCT06371651
What this trial is testing

Creatine and GAA for Brain Oxygenation

Who this might be right for
Oxygen Deficiency
University of Novi Sad, Faculty of Sport and Physical Education 19
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 applicableStudy completedNCT02903654
What this trial is testing

Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana

Who this might be right for
Pompe's Disease
Centre Hospitalier de Cayenne 925
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
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 completedNCT02950298
What this trial is testing

Pompe Telemedicine Developmental Study

Who this might be right for
Pompe DiseaseGlycogen Storage Disease II
Duke University 22
Not applicableUnknownNCT02635269
What this trial is testing

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Who this might be right for
Metabolism, Inborn ErrorsLipid Metabolism, Inborn ErrorsCarbohydrate Metabolism, Inborn Errors+20 more
Rigshospitalet, Denmark 60
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
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
Not applicableUnknownNCT02527239
What this trial is testing

Prevalence of Pompe's Disease in Respiratory Clinics

Who this might be right for
Pompe's Disease
Royal Brompton & Harefield NHS Foundation Trust 400
Post-approval studies (Phase 4)Looking for participantsNCT06666413
What this trial is testing

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

Who this might be right for
Glycogen Storage Disease Type IIPompe's Disease
Genzyme, a Sanofi Company 13
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
Post-approval studies (Phase 4)Study completedNCT01410890
What this trial is testing

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD II)
Genzyme, a Sanofi Company 21
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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