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Results for “Glycogen Storage Disorder Type 1”

41 trials

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Showing 20 of 41 results

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 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 applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
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
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 applicableUnknownNCT02635269
What this trial is testing

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Who this might be right for
Metabolism, Inborn ErrorsLipid Metabolism, Inborn ErrorsCarbohydrate Metabolism, Inborn Errors+20 more
Rigshospitalet, Denmark 60
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
Testing effectiveness (Phase 2)Study completedNCT01885936
What this trial is testing

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

Who this might be right for
Pompe Disease
Duke University 16
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
Testing effectiveness (Phase 2)Looking for participantsNCT06735755
What this trial is testing

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Who this might be right for
Glycogen Storage Disease Type Ia
Beam Therapeutics Inc. 36
Early research (Phase 1)Study completedNCT03041363
What this trial is testing

Treatment Development of Triheptanoin (G1D)

Who this might be right for
EpilepsyGLUT1DS1Glut1 Deficiency Syndrome 1, Autosomal Recessive+4 more
Juan Pascual 12
Testing effectiveness (Phase 2)Active Not RecruitingNCT05095727
What this trial is testing

MRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)

Who this might be right for
Glycogen Storage Disease
ModernaTX, Inc. 15
Testing effectiveness (Phase 2)UnknownNCT04930627
What this trial is testing

Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia

Who this might be right for
Glucose 6 Phosphatase Deficiency
Children's Memorial Health Institute, Poland 20
Not applicableLooking for participantsNCT06852612
What this trial is testing

Dietary Treatment Strategies and Metabolic Control in Glycogen Storage Disease Type I

Who this might be right for
Glycogen Storage Disease Type I
Insel Gruppe AG, University Hospital Bern 20
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)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 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
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 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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