16 completed, 4 withdrawn, 3 unknown, 1 terminated. 24 Phase 2, 3 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 24 | Completed 16, Withdrawn 4, Unknown 3, Terminated 1 |
| Phase 1 | 3 | Withdrawn 2, Completed 1 |
Follow triheptanoin: new trials by email Literature Review of triheptanoin trials Research Report on triheptanoin
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 |
|---|---|---|---|---|---|---|
| Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) Triheptanoin - Medium-chain Acyl-CoA Dehydrogenase Deficiency | Phase 2 | Withdrawn | Jerry Vockley, MD, PhD | 2026-08-01 | 2026-07-02 | |
| Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) Triheptanoin - Medium-chain Acyl-CoA Dehydrogenase Deficiency | Phase 2 | Withdrawn | Jerry Vockley, MD, PhD | 2026-07-01 | 2026-07-02 | |
| Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis Triheptanoin - Ataxia Telangiectasia | Phase 2 | Completed | The University of Queensland | 30 | 2022-03-15 | 2023-07-20 |
| 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 |
| A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS) Triheptanoin - ALS | Phase 1 / Phase 2 | Completed | Richard Bedlack, M.D., Ph.D. | 15 | 2018-06-21 | 2019-12-24 |
| Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D Triheptanoin - GLUT1DS1 | Phase 2 | Completed | University of Texas Southwestern Medical Center | 10 | 2018-06-05 | 2024-01-17 |
| 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 |
| Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. Tridecanoic Acid - Rett Syndrome | Phase 2 | Unknown | Sheba Medical Center | 10 | 2017-04-01 | 2017-02-23 |
| Triheptanoin in Mc Ardle Triheptanoin, Placebo oil - Glycogen Storage Disease Type V | Phase 2 | Completed | Institut National de la Santé Et de la Recherche Médicale, France | 12 | 2016-11-16 | 2025-09-04 |
| Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin triheptanoin - Rett Syndrome | Phase 2 | Unknown | Center for Rare Neurological Diseases, Norcross, GA | 12 | 2016-06-01 | 2020-07-17 |
| 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 |
| A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease Triheptanoin oil, Placebo - Huntington Disease | Phase 2 | Completed | Institut National de la Santé Et de la Recherche Médicale, France | 100 | 2015-06-29 | 2026-06-03 |
| The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V) Triheptanoin, Placebo oil - Glycogen Storage Disease Type V | Phase 2 | Completed | Copenhagen University Hospital, Rigshospitalet | 22 | 2015-04-01 | 2019-10-16 |
| Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood Triheptanoin, Placebo - Alternating Hemiplegia of Childhood | Phase 2 | Completed | Institut National de la Santé Et de la Recherche Médicale, France | 10 | 2015-03-25 | 2025-12-10 |
| Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies UX007 - Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency | Phase 2 | Completed | Ultragenyx Pharmaceutical Inc | 94 | 2014-12-09 | 2023-08-01 |
| 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 |
| A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) UX007 - Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency | Phase 2 | Completed | Ultragenyx Pharmaceutical Inc | 29 | 2014-02-06 | 2021-02-11 |
Showing 20 of 24. Sign in to see all of them.
Glut1 Deficiency Syndrome (6) · VLCAD deficiency (3) · Epilepsy (2) · Glycogen Storage Disease Type V (2) · Huntington Disease (2) · Medium chain acyl CoA dehydrogenase deficiency (2) · Rett Syndrome (2) · Alternating hemiplegia of childhood (1) · Ataxia Telangiectasia (1) · Gastrointestinal Diseases (1) · Glucose Metabolism Disorders (1) · Glycogen Storage Disease (1) · Glycogen Storage Disease Id (1) · Glycogen Storage Disease Type III (1) · Glycogen Storage Disease Type IV (1)
Institut National de la Santé Et de la Recherche Médicale, France (4) · Ultragenyx Pharmaceutical Inc (4) · University of Texas Southwestern Medical Center (3) · Copenhagen University Hospital, Rigshospitalet (2) · Jerry Vockley, MD, PhD (2) · Adrian Lacy (1) · Baylor Research Institute (1) · Center for Rare Neurological Diseases, Norcross, GA (1) · Juan Pascual (1) · Oregon Health and Science University (1) · Richard Bedlack, M.D., Ph.D. (1) · Sheba Medical Center (1) · The University of Queensland (1) · University of British Columbia (1)
United States (14) · France (6) · United Kingdom (4) · Australia (3) · Denmark (3) · Spain (2) · Canada (1) · Israel (1) · Italy (1) · Netherlands (1)
Adrian Lacy, MD (1) · Aslan T Turer, MD, MHS (1) · Bruria Ben-Zeev, MD (1) · Daniel Tarquinio, DO (1) · David Coman, MBBS FRACP (1) · Emmanuel Flamand-Roze, MD, PhD (1) · Gerard Vockley (1) · Gerard Vockley, MD, PhD (1) · Juan Pascual, M.D. (1) · Juan Pascual, MD (1) · Juan Pascual, MD, PhD (1) · Karen L Madsen, MD (1) · Melanie B Gillingham, PhD (1) · Raphael Schiffmann, M.D, M.H.Sc (1)
PMID 16763896, PMID 28871440, PMID 28969699, PMID 29110179, PMID 12122118, PMID 12210323, PMID 1281472, PMID 15293280, PMID 15781190, PMID 1580445, PMID 16602102, PMID 16763895, PMID 16934683, 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 22144182, PMID 22875082, PMID 22927201, PMID 2295969, PMID 23031366, PMID 23507172, PMID 24236946, PMID 24453079, PMID 25272951, PMID 25568297, PMID 25656498, PMID 25832663, PMID 26255073, PMID 26433381, PMID 26547562, PMID 26652229, PMID 26851119, PMID 28189603, PMID 28932990, PMID 30586396, PMID 30740733, PMID 30796268, PMID 30948626, PMID 31381524, PMID 31520525
2014 (2), R01NS094257 (2), 2015 (1), 2016 (1), 2017 (1)