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Results for “Glycogen Storage Disease Type II GSD II”

162 trials

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Showing 20 of 162 results

Not applicableWithdrawnNCT02385162
What this trial is testing

Biomarker for Glycogen Storage Diseases (BioGlycogen)

Who this might be right for
Fructose Metabolism, Inborn ErrorsGlycogen Storage DiseaseGlycogen Storage Disease Type I+7 more
CENTOGENE GmbH Rostock
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
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
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
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 applicableUnknownNCT05567627
What this trial is testing

Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease

Who this might be right for
Infantile-onset Pompe Disease
Seventh Medical Center of PLA General Hospital 6
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
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 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
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 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 applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Not applicableUnknownNCT01409486
What this trial is testing

Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry

Who this might be right for
Pompe Disease
Rambam Health Care Campus 10,000
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 applicableUnknownNCT00713453
What this trial is testing

Muscle Response to Enzyme Replacement Therapy in Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 16
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
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
Testing effectiveness (Phase 2)Looking for participantsNCT07123155
What this trial is testing

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 45
Not applicableUnknownNCT04981210
What this trial is testing

High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient Clinics

Who this might be right for
Pompe Disease
National Taiwan University Hospital 1,500
Not applicableEnded earlyNCT02191917
What this trial is testing

Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

Who this might be right for
Late-onset Pompe Disease
BioMarin Pharmaceutical 8
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