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Results for “Glycogen Storage Disease II”

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Showing 20 of 0 results

Post-approval studies (Phase 4)Ended earlyNCT01526785
What this trial is testing

Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

Who this might be right for
Pompe Disease
Genzyme, a Sanofi Company 113
Post-approval studies (Phase 4)Study completedNCT00486889
What this trial is testing

Growth and Development Study of Alglucosidase Alfa

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease
Genzyme, a Sanofi Company 12
Early research (Phase 1)Study completedNCT02240407
What this trial is testing

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 2
Testing effectiveness (Phase 2)Study completedNCT01380743
What this trial is testing

Drug-drug Interaction Study

Who this might be right for
Pompe Disease
Amicus Therapeutics 25
Post-approval studies (Phase 4)Study completedNCT01288027
What this trial is testing

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

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 16
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)Study completedNCT00976352
What this trial is testing

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 9
Not applicableAvailableNCT04327973
What this trial is testing

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Who this might be right for
Pompe Disease Infantile-Onset
Amicus Therapeutics
Post-approval studies (Phase 4)UnknownNCT02525172
What this trial is testing

Immune Modulation Therapy for Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 8
Not applicableStudy completedNCT01556516
What this trial is testing

Pregnancy and Birth Outcome in Women With Pompe Disease

Who this might be right for
Pompe Disease
O & O Alpan LLC 20
Large-scale testing (Phase 3)Study completedNCT03729362
What this trial is testing

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 125
Not applicableStudy completedNCT02363153
What this trial is testing

Diet and Exercise in Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type IILysosomal Storage Diseases
University of Florida 2
Testing effectiveness (Phase 2)Study completedNCT00053573
What this trial is testing

RhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 20
Not applicableStudy completedNCT01758354
What this trial is testing

Newborn Screening Assay of Pompe's Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 236,536
Not applicableStudy completedNCT02824068
What this trial is testing

Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)

Who this might be right for
Pompe Disease
LMU Klinikum 112
Not applicableStudy completedNCT05431127
What this trial is testing

High Dose Inspiratory Muscle Training in LOPD

Who this might be right for
Late-Onset Pompe DiseaseLysosomal Disease
Duke University 34
Large-scale testing (Phase 3)Study completedNCT00125879
What this trial is testing

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 16
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT04093349
What this trial is testing

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+4 more
Spark Therapeutics, Inc. 4
Not applicableUnknownNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
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