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Results for “Glycogen Storage Diseases”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT04138277
What this trial is testing

Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 119
Not applicableStudy completedNCT02838368
What this trial is testing

Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices

Who this might be right for
Pompe Disease
University of California, Irvine 921
Not applicableLooking for participantsNCT06843330
What this trial is testing

Accuracy of Lactate Meter in GSDIa

Who this might be right for
Glycogen Storage Disease Type Ia
Connecticut Children's Medical Center 10
Post-approval studies (Phase 4)Active Not RecruitingNCT05164055
What this trial is testing

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 17
Not applicableLooking for participantsNCT07303140
What this trial is testing

Non-invasive Assessment of Liver Fibrosis in a French Cohort of Pediatric Patients With Type III Glycogen Storage Disease: Current State and Perspectives

Who this might be right for
Liver Fibrosis
University Hospital, Strasbourg, France 30
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
Testing effectiveness (Phase 2)Study completedNCT00467935
What this trial is testing

Scotoma Reduction in AMD Patients Treated With Ranibizumab

Who this might be right for
Macular Degeneration
Retina Research Foundation 20
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
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 applicableLooking for participantsNCT02683512
What this trial is testing

GBE Deficiency (GSD IV and APBD) Natural History Study

Who this might be right for
Glycogen Storage Disease Type IVAdult Polyglucosan Body DiseaseGSD4+2 more
Duke University 200
Not applicableStudy completedNCT04399694
What this trial is testing

Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders

Who this might be right for
Genetic DiseaseInborn Errors of MetabolismGlycogen Storage Disease+2 more
Duke University 56
Not applicableStudy completedNCT02054832
What this trial is testing

Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch

Who this might be right for
Glycogen Storage Disease Type IAGlycogen Storage Disease Type IBGlycogen Storage Disease Type III+1 more
John Mitchell 11
Not applicableStudy completedNCT05073783
What this trial is testing

Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting

Who this might be right for
Pompe DiseaseMucopolysaccharidosis Type I (MPS I)
Sanofi 57
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 applicableNot Yet RecruitingNCT07739394
What this trial is testing

Diazoxide in the Treatment of Type 1 Glycogenosis

Who this might be right for
Glycogen Storage Disease Type I
Central Hospital, Nancy, France 15
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
Testing effectiveness (Phase 2)Study completedNCT03181399
What this trial is testing

Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)

Who this might be right for
GLUT1DS1EpilepsyGlut1 Deficiency Syndrome 1, Autosomal Recessive+4 more
University of Texas Southwestern Medical Center 45
Not applicableStudy completedNCT05083806
What this trial is testing

MSOT in Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Pompe's Disease Juvenile Onset+1 more
University of Erlangen-Nürnberg Medical School 20
Not applicableStudy completedNCT02354651
What this trial is testing

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 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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