2 unknown, 1 completed, 1 not yet recruiting. 2 Phase 2, 1 Not applicable, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Not yet recruiting 1, Unknown 1 |
| Not applicable | 1 | Unknown 1 |
| Phase 1 | 1 | Unknown 1 |
Follow Glycogen Storage Disease XIV: new trials by email Literature Review of Glycogen Storage Disease XIV trials Research Report on Glycogen Storage Disease XIV
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 |
|---|---|---|---|---|---|---|
| Evaluating the Efficacy and Safety of D-galactose in PGM1-CDG (AVTX-801) AVTX-801, Placebo - PGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of Glycosylation | Phase 2 | Not yet recruiting | Eva Morava-Kozicz | 8 | 2026-10-01 | 2026-03-16 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14 ORL-1G - D-galactose - Glycogen Storage Disease, Type 14 | Phase 1 / Phase 2 | Unknown | Orpha Labs | 5 | 2017-10-31 | 2019-01-25 |
| 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 |
Carnitine palmitoyl transferase 2 deficiency (2) · Glycogen Storage Disease (2) · Glycogen Storage Disease Type II (2) · Medium chain acyl CoA dehydrogenase deficiency (2) · Systemic carnitine deficiency (2) · Trifunctional Protein Deficiency With Myopathy And Neuropathy (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency (1) · Acidemia, isovaleric (1) · Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of (1) · Adrenal Hyperplasia, Congenital (1) · Adrenoleukodystrophy (1) · Andersen Syndrome (1) · Anemia, Diamond-Blackfan (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Metabolic Diseases (3) · Nutritional and Metabolic Diseases (3) · Amino Acid Metabolism, Inborn Errors (2) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Carbohydrate Metabolism, Inborn Errors (2) · Central Nervous System Diseases (2) · Lipid Metabolism Disorders (2) · Lysosomal Storage Diseases (2) · Lysosomal Storage Diseases, Nervous System (2) · Metabolism, Inborn Errors (2) · Mitochondrial Diseases (2)
Centre Hospitalier Universitaire de Liege (1) · Copenhagen University Hospital, Rigshospitalet (1) · Eva Morava-Kozicz (1) · Orpha Labs (1)
Belgium (1) · Denmark (1) · Turkey (Türkiye) (1) · United States (1)
Eva Morava-Kozicz, MD, PhD (1) · Karen L Madsen, MD (1) · Laurent Servais (1)