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Results for “GAA Deficiency”

57 trials

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Showing 20 of 57 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 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
Not applicableApproved For MarketingNCT00074919
What this trial is testing

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

Who this might be right for
Glycogen Storage Disease Type IIGlycogenosis 2
Genzyme, a Sanofi Company
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
Post-approval studies (Phase 4)Ended earlyNCT00701701
What this trial is testing

Immune Tolerance Induction Study

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 4
Not applicableLooking for participantsNCT01665326
What this trial is testing

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Who this might be right for
Pompe Disease
Duke University 400
Not applicableUnknownNCT02708784
What this trial is testing

An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-

Who this might be right for
Glycogen Storage Disease Type 2Dystrophia Myotonica
University of Aarhus 60
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
Not applicableUnknownNCT01914536
What this trial is testing

A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI

Who this might be right for
Pompe Disease
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau 30
Not applicableStudy completedNCT00830583
What this trial is testing

Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis

Who this might be right for
Pompe's Disease
Centre Hospitalier Universitaire de Nice 400
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
Post-approval studies (Phase 4)Ended earlyNCT01597596
What this trial is testing

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Who this might be right for
Pompe Disease (Infantile-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenosis 2+1 more
Genzyme, a Sanofi Company 4
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
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT03019406
What this trial is testing

Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

Who this might be right for
Glycogen Storage Disease Type II-Pompe's Disease
Genzyme, a Sanofi Company 22
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
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
Large-scale testing (Phase 3)Study completedNCT00059280
What this trial is testing

The Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 16
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