6 completed, 4 recruiting, 2 active not recruiting, 2 not yet recruiting. 4 Phase 2, 3 Not applicable, 2 Phase 1, 1 Early Phase 1, 1 Phase 3.
Follow Glycogen Storage Disease Type I: new trials by email Literature Review of Glycogen Storage Disease Type I trials Research Report on Glycogen Storage Disease Type I
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 |
|---|---|---|---|---|---|---|
| Effects of the Carbohydrate Counting Method on Blood Glucose Regulation, Quality of Life, Urinary Glc4 and Other Biomarkers in Children With GSD1 Glycogen Storage Disease Type I, Glycogen Storage Disease Type IB | Not yet recruiting | Şükrü Arman AKSOY | 8 | 2026-10-30 | 2026-10-15 | |
| Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease Lactate Meter, Accu Chek - Glycogen Storage Disease Type IA, Glycogen Storage Disease Type I | Recruiting | Connecticut Children's Medical Center | 10 | 2026-09-18 | 2026-10-29 | |
| Diazoxide in the Treatment of Type 1 Glycogenosis Glycogen Storage Disease Type I | Not yet recruiting | Central Hospital, Nancy, France | 15 | 2026-08-30 | 2026-07-31 | |
| Use of Continuous Glucose Monitoring to Evaluate Postprandial Response to Raw Cornstarch Supplementation in Adult Glycogen Storage Disease Type I Glycogen Storage Disease Type I | Recruiting | Federico II University | 8 | 2026-02-05 | 2026-06-12 | |
| Dietary Treatment Strategies and Metabolic Control in Glycogen Storage Disease Type I Fructose, Fructose and galactose - Glycogen Storage Disease Type I | Not applicable | Recruiting | Insel Gruppe AG, University Hospital Bern | 20 | 2025-04-24 | 2026-05-12 |
| Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program No Intervention - Glycogen Storage Disease Type Ia | Recruiting | Ultragenyx Pharmaceutical Inc | 140 | 2024-11-04 | 2026-10-14 | |
| A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a) mRNA-3745 - Glycogen Storage Disease | Phase 1 / Phase 2 | Active not recruiting | ModernaTX, Inc. | 15 | 2022-06-01 | 2025-12-15 |
| A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) DTX401, Placebo, Oral prednisolone - Glycogen Storage Disease Type IA | Phase 3 | Completed | Ultragenyx Pharmaceutical Inc | 52 | 2021-11-08 | 2026-10-14 |
| Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia Empagliflozin - Glucose 6 Phosphatase Deficiency | Phase 2 | Unknown | Children's Memorial Health Institute, Poland | 20 | 2021-07-01 | 2021-06-23 |
| Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I Triheptanoin - Glycogen Storage Disease Type I | Early Phase 1 | Completed | Areeg El-Gharbawy | 4 | 2020-10-16 | 2021-12-15 |
| Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia No intervention - Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia) | Completed | Ultragenyx Pharmaceutical Inc | 12 | 2019-07-15 | 2026-03-18 | |
| Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency Empagliflozin - Glycogen Storage Disease Type I, Glucose 6 Phosphatase Deficiency | Phase 2 | Unknown | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | 5 | 2019-06-20 | 2019-10-28 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Biomarker for Glycogen Storage Diseases (BioGlycogen) Fructose Metabolism, Inborn Errors, Glycogen Storage Disease | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 | ||
| Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa) DTX401, steroid regimen - GSD1 | Phase 1 / Phase 2 | Completed | Ultragenyx Pharmaceutical Inc | 12 | 2018-05-18 | 2022-11-18 |
| The Use of Uncooked Sweet Manioc Starch to Treat Hepatic Glycogen Storage Diseases Sweet manioc starch, Cornstarch - Glycogen Storage Disease Type I | Not applicable | Completed | Hospital de Clinicas de Porto Alegre | 11 | 2017-04-29 | 2023-09-26 |
| Overnight Feeding Study in Glycogen Storage Disease Type 1 overnight nutrition regime - Glycogen Storage Disease Type 1 (GSD 1) | Not applicable | Completed | University of Zurich | 5 | 2013-11-01 | 2015-03-23 |
Glucose Metabolism Disorders (5) · Hepatorenal form of glycogen storage disease (5) · Glycogen Storage Disease (3) · Glycogen Storage Disease IB (3) · Genetic Diseases, Inborn (2) · Glycogen Storage Disease Type II (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Acidemia, isovaleric (1) · Acrodermatitis enteropathica (1) · Adenine phosphoribosyltransferase deficiency (1) · Adrenal hyperplasia, congenital, type 5 (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (17) · Metabolism, Inborn Errors (17) · Nutritional and Metabolic Diseases (17) · Carbohydrate Metabolism, Inborn Errors (16) · Metabolic Diseases (16) · Genetic Diseases, Inborn (15) · Glycogen Storage Disease (14) · Bone Diseases (2) · Bone Diseases, Metabolic (2) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Central Nervous System Diseases (2) · Female Urogenital Diseases (2) · Female Urogenital Diseases and Pregnancy Complications (2)
empagliflozin (2) · Fructose (1) · Galactose (1) · Prednisolone (1) · Starch (1) · triheptanoin (1)
Ultragenyx Pharmaceutical Inc (4) · Areeg El-Gharbawy (1) · CENTOGENE GmbH Rostock (1) · Central Hospital, Nancy, France (1) · Children's Memorial Health Institute, Poland (1) · Cliniques universitaires Saint-Luc- Université Catholique de Louvain (1) · Connecticut Children's Medical Center (1) · Federico II University (1) · Hospital de Clinicas de Porto Alegre (1) · Insel Gruppe AG, University Hospital Bern (1) · ModernaTX, Inc. (1) · RTI International (1) · University of Zurich (1) · Şükrü Arman AKSOY (1)
United States (8) · Canada (5) · Netherlands (5) · Spain (5) · Brazil (3) · Germany (3) · Italy (3) · Denmark (2) · Japan (2) · Poland (2) · Switzerland (2) · Belgium (1) · France (1) · India (1) · Sri Lanka (1)
Areeg El-Gharbawy, MD (1) · Curt Scharfe, MD, PhD (1) · Giatgen Spinas, Prof MD (1) · Ida D Schwartz, Dr (1) · Malaya Mount, MS, RD, CDN (1) · Michel Hochuli, MD, PhD (1) · Peter Bauer, Prof. (1) · Rebecca Riba-Wolman, MD (1) · Xavier Stephenne, MD, PhD (1)
PMID 30714174, PMID 31315600, PMID 32430177, PMID 34082801, PMID 35142618, PMID 37476587, PMID 40064185
2023 (3), 2016 (1), 2018 (1)