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Results for “Glycogen Storage Disease”

233 trials

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Showing 20 of 233 results

Testing effectiveness (Phase 2)Study completedNCT02675465
What this trial is testing

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Who this might be right for
Pompe Disease
Amicus Therapeutics 29
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 applicableEnrolling By InvitationNCT06605612
What this trial is testing

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

Who this might be right for
Inclusion Body MyositisMyotonic DystrophyLimb-girdle and Facioscapulohumeral Muscular Dystrophies+9 more
LMU Klinikum 108
Not applicableLooking for participantsNCT06396546
What this trial is testing

'Glycogen Storage Diseases (GSDs) in Indian Children- Establishing an Indian GSD (I-GSD) Registry'

Who this might be right for
Glycogen Storage Diseases
Institute of Liver and Biliary Sciences, India 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
Not applicableLooking for participantsNCT06636383
What this trial is testing

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Who this might be right for
Glycogen Storage Disease Type Ia
Ultragenyx Pharmaceutical Inc 140
Testing effectiveness (Phase 2)Active Not RecruitingNCT04174105
What this trial is testing

Gene Transfer Study in Patients With Late Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Astellas Gene Therapies 11
Not applicableStudy completedNCT01773954
What this trial is testing

Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration

Who this might be right for
Age-Related Macular DegenerationMacular Degeneration
MidAtlantic Retina 40
Post-approval studies (Phase 4)UnknownNCT02405624
What this trial is testing

CPAP for Infantile Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 3
Not applicableUnknownNCT05951790
What this trial is testing

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease (Late-onset)
Fondazione Don Carlo Gnocchi ETS 20
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 applicableLooking for participantsNCT06557369
What this trial is testing

A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD

Who this might be right for
Intermediate AMD
Oculox Technologies SA 30
Testing effectiveness (Phase 2)Study completedNCT00947960
What this trial is testing

Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

Who this might be right for
Adult Polyglucosan Body DiseaseGlycogen Brancher Enzyme DeficiencyGlycogen Storage Disease Type IV
Baylor Research Institute 23
Testing effectiveness (Phase 2)WithdrawnNCT04094948
What this trial is testing

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Duke University
Not applicableLooking for participantsNCT06833489
What this trial is testing

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

Who this might be right for
Rare Genetic Muscle DiseasesMuscular Dystrophy, DuchenneMuscular Dystrophy, Becker+2 more
Assistance Publique Hopitaux De Marseille 50
Not applicableStudy completedNCT00001342
What this trial is testing

Study of Glycogen Storage Disease and Associated Disorders

Who this might be right for
Glycogen Storage Disease
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 20
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 applicableLooking for participantsNCT07645898
What this trial is testing

Use of Continuous Glucose Monitoring to Evaluate Postprandial Response to Raw Cornstarch Supplementation in Adult Glycogen Storage Disease Type I

Who this might be right for
Glycogen Storage Disease Type I
Federico II University 8
Not applicableLooking for participantsNCT07336394
What this trial is testing

Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques

Who this might be right for
Danon DiseaseFabry DiseaseCardiac Amyloidosis+4 more
Chinese Academy of Medical Sciences, Fuwai Hospital 1,000
Not applicableStudy completedNCT04708015
What this trial is testing

Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa

Who this might be right for
Glycogen Storage Disease Type IA
Ultragenyx Pharmaceutical Inc 15
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