4 completed, 2 recruiting, 2 terminated, 2 unknown. 3 Not applicable, 2 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 3 | Unknown 2, Completed 1 |
| Phase 2 | 2 | Completed 1, Terminated 1 |
| Phase 1 | 1 | Terminated 1 |
Follow Glycogen Storage Disease Type III: new trials by email Literature Review of Glycogen Storage Disease Type III trials Research Report on Glycogen Storage Disease Type III
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Non-invasive Assessment of Liver Fibrosis in a French Cohort of Pediatric Patients With Type III Glycogen Storage Disease: Current State and Perspectives Liver Fibrosis | Recruiting | University Hospital, Strasbourg, France | 30 | 2024-10-18 | 2025-12-24 | |
| Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III No Intervention - Glycogen Storage Disease Type III | Terminated | Ultragenyx Pharmaceutical Inc | 14 | 2022-05-20 | 2023-09-28 | |
| Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) UX053, Placebo, Antipyretic - Glycogen Storage Disease Type III | Phase 1 / Phase 2 | Terminated | Ultragenyx Pharmaceutical Inc | 9 | 2021-10-18 | 2024-04-16 |
| Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III) No Intervention - Glycogen Storage Disease Type III | Completed | Ultragenyx Pharmaceutical Inc | 18 | 2020-11-23 | 2022-09-13 | |
| Biomarker for Glycogen Storage Diseases (BioGlycogen) Fructose Metabolism, Inborn Errors, Glycogen Storage Disease | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 | ||
| The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses Triheptanoin, Placebo Oil - Tarui Disease, Debrancher Deficiency | Phase 2 | Completed | Copenhagen University Hospital, Rigshospitalet | 3 | 2018-08-15 | 2024-02-22 |
| Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy Sugar - Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors | Not applicable | Unknown | Copenhagen University Hospital, Rigshospitalet | 60 | 2016-01-01 | 2019-10-16 |
| Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies FAXE Kondi, Faxe Kondi Free - Glycogen Storage Disease Type III | Not applicable | Completed | Copenhagen University Hospital, Rigshospitalet | 6 | 2015-01-01 | 2024-02-26 |
| Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch Glycosade - Glycogen Storage Disease Type IA, Glycogen Storage Disease Type IB | Completed | John Mitchell | 11 | 2013-11-01 | 2015-09-25 | |
| French Observatory for Patients with Type 3 Glycogenosis Glycogen Storage Disease Type III | Recruiting | Institut de Myologie, France | 150 | 2013-09-01 | 2024-09-27 | |
| Prospective Follow-up of Patients With Glycogen Storage Disease Type III Different motor function tests, Manual dexterity assessment - Neuromuscular Disorders | Not applicable | Unknown | Institut de Myologie, France | 30 | 2011-02-01 | 2012-12-20 |
Glycogen Storage Disease Type VII (3) · Glycogen Storage Disease (2) · Glycogen Storage Disease Type II (2) · Glycogen Storage Disease Type IV (2) · Glycogen Storage Disease Type V (2) · Carbohydrate Metabolism, Inborn Errors (1) · Carnitine palmitoyl transferase 2 deficiency (1) · Chanarin-Dorfman Syndrome (1) · Dimauro disease (1) · Fructose Metabolism, Inborn Errors (1) · Glycogen Storage Disease IB (1) · Glycogen Storage Disease Type I (1) · Glycogen Storage Disease Type Ix (1) · Glycogen Storage Disease Type VI (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Genetic Diseases, Inborn (11) · Nutritional and Metabolic Diseases (11) · Carbohydrate Metabolism, Inborn Errors (10) · Metabolic Diseases (10) · Metabolism, Inborn Errors (10) · Glycogen Storage Disease (9) · Nervous System Diseases (4) · Muscular Diseases (3) · Muscular Disorders, Atrophic (3) · Muscular Dystrophies (3) · Musculoskeletal Diseases (3) · Neuromuscular Diseases (3) · Brain Diseases (2) · Brain Diseases, Metabolic (2)
Acetaminophen (1) · Antipyretics (1) · Cetirizine (1) · Famotidine (1) · Histamine Antagonists (1) · Histamine H2 Antagonists (1) · Ibuprofen (1) · Sugars (1) · tricaprylin (1) · triheptanoin (1)
Copenhagen University Hospital, Rigshospitalet (3) · Ultragenyx Pharmaceutical Inc (3) · Institut de Myologie, France (2) · CENTOGENE GmbH Rostock (1) · John Mitchell (1) · University Hospital, Strasbourg, France (1)
Denmark (3) · France (3) · United States (3) · Canada (1) · Germany (1) · India (1) · Italy (1) · Netherlands (1) · Spain (1) · Sri Lanka (1)
Astrid E Buch, BSc Medicine (1) · John J Mitchell, MD (1) · Karen L Madsen, MD (1) · Peter Bauer, Prof. (1)
PMID 12122118, PMID 12210323, PMID 1281472, PMID 15293280, PMID 15781190, PMID 1580445, PMID 16602102, PMID 16763895, PMID 16763896, PMID 17584774, PMID 1824792, PMID 18401027, PMID 18541798, PMID 18645163, PMID 18929906, PMID 19237700, PMID 20357282, PMID 20393176, PMID 20631546, PMID 20655781, PMID 2199596, PMID 2295969, PMID 23031366, PMID 23507172, PMID 24453079, PMID 25272951, PMID 25832663, PMID 26255073, PMID 26652229, PMID 28932990, PMID 30990523, PMID 31492716, PMID 3447177, PMID 42082675, PMID 5235982, PMID 7634753, PMID 8755644
2017 (1)