12 completed, 2 recruiting, 2 terminated, 2 withdrawn. 8 Phase 2, 5 Not applicable, 3 Phase 1, 1 Early Phase 1, 1 Phase 3.
Follow Glut1 Deficiency Syndrome: new trials by email Literature Review of Glut1 Deficiency Syndrome trials Research Report on Glut1 Deficiency Syndrome
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 |
|---|---|---|---|---|---|---|
| Red Blood Cell Exchange Transfusion as a Novel Treatment for GLUT1 Deficiency Syndrome Red Blood Cell Transfusion - Glucose Transporter Type 1 Deficiency Syndrome, GLUT1DS1 | Not applicable | Suspended | Weill Medical College of Cornell University | 12 | 2027-09-01 | 2025-12-11 |
| A Phase II Study With Exploratory Outcomes of Fucose Supplementation in GLUT1 Deficiency Syndrome L-fucose, Placebo - Glut1 Deficiency, GLUT1DS1 | Phase 2 | Recruiting | Oregon Health and Science University | 16 | 2026-05-05 | 2026-05-13 |
| 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 | |
| Sodium Lactate Infusion in GLUT1DS Patients sodiumlactate - GLUT1 Deficiency Syndrome, GLUT1DS1 | Early Phase 1 | Completed | Radboud University Medical Center | 2 | 2022-05-20 | 2022-11-23 |
| Brain Metabolism Observed at 3 Tesla or 7 Tesla in Health and Metabolic Disease Magnetic resonance imaging - Glut1 Deficiency Syndrome 1, Glucose Metabolism Disorders | Recruiting | Weill Medical College of Cornell University | 20 | 2022-05-03 | 2026-06-01 | |
| Evaluation of Ketoflo Ketoflo - Epilepsy Intractable, Glucose Transporter Type 1 Deficiency Syndrome | Not applicable | Completed | Vitaflo International, Ltd | 25 | 2020-02-24 | 2024-02-15 |
| 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 | |
| Early Diagnosis of the GLUT1 Deficiency Syndrome With a Blood Based Test METAglut1 - Glut1 Deficiency Syndrome, De Vivo Disease | Not applicable | Completed | METAFORA biosystems | 636 | 2018-09-24 | 2022-12-01 |
| Evaluation of Krio in Children and Adults With Epilepsy Krio - Ketogenic Dieting, Epilepsy | Not applicable | Withdrawn | Vitaflo International, Ltd | 2018-05-11 | 2024-02-16 | |
| Diet Treatment Glucose Transporter Type 1 Deficiency (G1D) Triheptanoin - GLUT1DS1, Epilepsy | Phase 2 | Completed | University of Texas Southwestern Medical Center | 45 | 2018-04-18 | 2025-08-07 |
| Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) UX007, Placebo - Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | Phase 3 | Terminated | Ultragenyx Pharmaceutical Inc | 44 | 2017-04-19 | 2020-06-16 |
| Treatment Development of Triheptanoin (G1D) Triheptanoin - Epilepsy, GLUT1DS1 | Phase 1 | Completed | Juan Pascual | 12 | 2017-03-29 | 2022-12-22 |
| Evaluation of Keyo in Children With Epilepsy Keyo - Intractable Epilepsy, Glucose Transporter Type 1 Deficiency Syndrome | Not applicable | Completed | Vitaflo International, Ltd | 17 | 2016-04-01 | 2024-02-16 |
| Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D) Triheptanoin - Glucose Transporter Type 1 Deficiency Syndrome, Glut1 Deficiency Syndrome | Phase 1 / Phase 2 | Withdrawn | Juan Pascual | 2015-12-01 | 2019-02-15 | |
| Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS) UX007 - Glucose Transporter Type 1 Deficiency Syndrome | Phase 2 | Terminated | Ultragenyx Pharmaceutical Inc | 15 | 2015-09-10 | 2020-06-11 |
| Pilot Study of Triheptanoin in Patients With Glucose Transporter 1 Deficiency Syndrome Triheptanoin - Glucose Transporter Type 1 Deficiency Syndrome | Phase 2 | Unknown | University of British Columbia | 3 | 2014-04-01 | 2016-06-01 |
| Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) UX007, Placebo - Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | Phase 2 | Completed | Ultragenyx Pharmaceutical Inc | 36 | 2014-02-28 | 2020-06-19 |
| An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome Triheptanoin - Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS) | Phase 2 | Completed | Adrian Lacy | 20 | 2014-02-20 | 2021-01-28 |
| Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DS GLUT1 DS - Glut1 Deficiency Syndrome | Phase 2 | Completed | Institut National de la Santé Et de la Recherche Médicale, France | 20 | 2013-12-04 | 2025-10-03 |
| The Glucose Transporter Type I Deficiency (G1D) Registry No intervention - GLUT1 Deficiency Syndrome, Glucose Transporter Type 1 Deficiency Syndrome | Completed | University of Texas Southwestern Medical Center | 471 | 2013-12-01 | 2025-03-07 |
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Epilepsy (8) · Glucose Metabolism Disorders (3) · Glycogen Storage Disease Id (3) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (2) · Acidemia, isovaleric (2) · Anemia, Sickle Cell (2) · Argininosuccinic Aciduria (2) · Ataxia with vitamin E deficiency (2) · Basal ganglia disease, biotin-responsive (2) · Beta ketothiolase deficiency (2) · Biotinidase Deficiency (2) · Brown-Vialetto-Van Laere syndrome (2) · Carbamoyl-Phosphate Synthase I Deficiency Disease (2) · Carnitine palmitoyl transferase 1A deficiency (2)
Central Nervous System Diseases (11) · Nervous System Diseases (11) · Brain Diseases (10) · Metabolic Diseases (6) · Nutritional and Metabolic Diseases (6) · Pathological Conditions, Signs and Symptoms (5) · Neurologic Manifestations (4) · Neurobehavioral Manifestations (3) · Signs and Symptoms (3) · Abnormalities, Multiple (2) · Albinism (2) · Amino Acid Metabolism, Inborn Errors (2) · Anemia (2) · Anemia, Hemolytic (2) · Anemia, Hemolytic, Congenital (2)
triheptanoin (9) · Erythrocyte Transfusion (1) · Fucose (1) · Magnetic Resonance Imaging (1)
Juan Pascual (4) · Ultragenyx Pharmaceutical Inc (3) · Vitaflo International, Ltd (3) · University of Texas Southwestern Medical Center (2) · Weill Medical College of Cornell University (2) · Adrian Lacy (1) · Centre Hospitalier Universitaire de Liege (1) · Institut National de la Santé Et de la Recherche Médicale, France (1) · METAFORA biosystems (1) · Oregon Health and Science University (1) · RTI International (1) · Radboud University Medical Center (1) · University of British Columbia (1)
United States (14) · United Kingdom (5) · France (4) · Spain (3) · Australia (2) · Italy (2) · Belgium (1) · Canada (1) · Denmark (1) · Germany (1) · Israel (1) · Netherlands (1)
Fanny Mochel, MD, PhD (2) · Juan M. Pascual, MD, PhD (2) · Juan Pascual, MD, PhD (2) · Adrian Lacy, MD (1) · Curt Scharfe, MD, PhD (1) · Helen McCullagh, BSc, MBChB (1) · Juan M Pascual, MD, PhD (1) · Juan Pascual (1) · Juan Pascual, MD (1) · Kathryn Lightfoot (1) · Laurent Servais (1) · Rachel Meskell (1) · Rodrigo T. Starosta, MD, PhD (1) · Sylvia Stockler (1)
PMID 26536893, PMID 30948626, PMID 31315600, PMID 31694879, PMID 35142618, PMID 35441706, PMID 36523131, PMID 36859467
R01NS094257 (2), RM1NS133593 (2), 2015 (1)