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Results for “Glycogen Storage Disorder Type 1”

40 trials

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Showing 20 of 40 results

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 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
Not applicableEnrolling By InvitationNCT07072676
What this trial is testing

The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.

Who this might be right for
Inclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more
LMU Klinikum 30
Not applicableLooking for participantsNCT06795152
What this trial is testing

Rare Glycogen Storage Diseases Natural History Study

Who this might be right for
Glycogen Storage DiseaseGSD Type 0AGSD Type 0B+12 more
Duke University 200
Not applicableStudy completedNCT04907162
What this trial is testing

Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

Who this might be right for
Pompe Disease (Late-onset)Myotonic Dystrophy Type 1 (DM1)Myotonic Dystrophy Type 2+4 more
LMU Klinikum 82
Early research (Phase 1)Study completedNCT03533673
What this trial is testing

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
AskBio Inc 7
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
Not applicableLooking for participantsNCT07136844
What this trial is testing

Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology

Who this might be right for
Neuromuscular DiseasesObesity (Disorder)Myotonic Dystrophy 1+9 more
Centre Hospitalier Universitaire de Liege 300
Not applicableLooking for participantsNCT05085704
What this trial is testing

Brain Metabolism Observed at 3 Tesla or 7 Tesla in Health and Metabolic Disease

Who this might be right for
Glut1 Deficiency Syndrome 1Glucose Metabolism DisordersEpilepsy+4 more
Weill Medical College of Cornell University 20
Not applicableStudy completedNCT02176096
What this trial is testing

Comparison of the Effect of a Novel Starch (Glycosade) Versus Gastrostomy Tube-Dextrose Infusion on Overnight Euglycaemia Control in Children With Glycogen Storage Disease Type I: Open Label Demonstration Trial

Who this might be right for
Glycogen Storage Disorder Type 1HypoglycemiaCornstarch+1 more
University of Manitoba 4
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
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 completedNCT03970278
What this trial is testing

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

Who this might be right for
Glycogen Storage Disease Type IAVon Gierke's Disease (GSD Type Ia)
Ultragenyx Pharmaceutical Inc 12
Testing effectiveness (Phase 2)UnknownNCT04138251
What this trial is testing

Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency

Who this might be right for
Glycogen Storage Disease Type IGlucose 6 Phosphatase Deficiency
Cliniques universitaires Saint-Luc- Université Catholique de Louvain 5
Not applicableLooking for participantsNCT07459582
What this trial is testing

Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease

Who this might be right for
Glycogen Storage Disease Type IAGlycogen Storage Disease Type IGlycogen Storage Disease Type IB+1 more
Connecticut Children's Medical Center 10
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)UnknownNCT03404856
What this trial is testing

Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14

Who this might be right for
Glycogen Storage Disease, Type 14
Orpha Labs 5
Testing effectiveness (Phase 2)Study completedNCT03517085
What this trial is testing

Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

Who this might be right for
GSD1
Ultragenyx Pharmaceutical Inc 12
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 applicableEnded earlyNCT05196165
What this trial is testing

Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III

Who this might be right for
Glycogen Storage Disease Type III
Ultragenyx Pharmaceutical Inc 14
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