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Results for “GAA Deficiency”

57 trials

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Enrolling by Invitation

Showing 20 of 57 results

Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Post-approval studies (Phase 4)Study completedNCT00701129
What this trial is testing

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II
Genzyme, a Sanofi Company 4
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 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
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
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
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
Testing effectiveness (Phase 2)Study completedNCT00025896
What this trial is testing

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type IIAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 8
Not applicableStudy completedNCT02904395
What this trial is testing

Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth

Who this might be right for
Pompe's Disease
Centre Hospitalier de Cayenne 3,413
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
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
Post-approval studies (Phase 4)Study completedNCT00483379
What this trial is testing

High Dose or High Dose Frequency Study of Alglucosidase Alfa

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 13
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
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
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 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
Testing effectiveness (Phase 2)WithdrawnNCT01656590
What this trial is testing

High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Columbia University
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
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
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
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