19 completed, 5 withdrawn, 4 no longer available, 4 unknown. 24 Phase 2, 6 Phase 1, 2 Early Phase 1, 2 Phase 3.
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Acyl-CoA Dehydrogenase Deficiency (MCADD) Triheptanoin - Medium-chain Acyl-CoA Dehydrogenase Deficiency | Phase 2 | Withdrawn | Jerry Vockley, MD, PhD | 2026-08-01 | 2026-07-02 | |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Acyl-CoA Dehydrogenase Deficiency (MCADD) Triheptanoin - Medium-chain Acyl-CoA Dehydrogenase Deficiency | Phase 2 | Withdrawn | Jerry Vockley, MD, PhD | 2026-07-01 | 2026-07-02 | |
| Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency Triheptanoin - Pyruvate Dehydrogenase Complex Deficiency | Phase 1 | Recruiting | Jirair Krikor Bedoyan | 6 | 2024-07-11 | 2026-02-17 |
| A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD Triheptanoin, MCT Oil - Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Phase 3 | Active not recruiting | Ultragenyx Pharmaceutical Inc | 69 | 2023-02-28 | 2026-09-17 |
| 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 |
| 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 |
| 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 |
| Treatment Development of Triheptanoin (G1D) Triheptanoin - Epilepsy, GLUT1DS1 | Phase 1 | Completed | Juan Pascual | 12 | 2017-03-29 | 2022-12-22 |
| 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 |
| A Pilot Trial of Triheptanoin for the Preventive Treatment of Migraine Triheptanoin - Migraine | Early Phase 1 | Unknown | University of Liege | 10 | 2016-06-01 | 2016-05-27 |
| 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 |
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Glut1 Deficiency Syndrome (9) · VLCAD deficiency (6) · Epilepsy (3) · Glucose Metabolism Disorders (2) · Glycogen Storage Disease Id (2) · Glycogen Storage Disease Type V (2) · Huntington Disease (2) · Medium chain acyl CoA dehydrogenase deficiency (2) · Rett Syndrome (2) · Trifunctional Protein Deficiency With Myopathy And Neuropathy (2) · Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of (1) · Alternating hemiplegia of childhood (1) · Ataxia Telangiectasia (1) · Barth Syndrome (1) · Carnitine-Acylcarnitine Translocase Deficiency (1)
Ultragenyx Pharmaceutical Inc (6) · Institut National de la Santé Et de la Recherche Médicale, France (4) · Jerry Vockley, MD, PhD (4) · Juan Pascual (4) · University of Texas Southwestern Medical Center (3) · Copenhagen University Hospital, Rigshospitalet (2) · Adrian Lacy (1) · Areeg El-Gharbawy (1) · Baylor Research Institute (1) · Center for Rare Neurological Diseases, Norcross, GA (1) · Irina A Anselm (1) · Jirair Krikor Bedoyan (1) · Maria Luz Couce Pico (1) · Oregon Health and Science University (1) · Richard Bedlack, M.D., Ph.D. (1)
United States (22) · France (6) · Spain (4) · United Kingdom (4) · Australia (3) · Denmark (3) · Canada (1) · Czechia (1) · Germany (1) · Israel (1) · Italy (1) · Japan (1) · Netherlands (1) · Poland (1) · Saudi Arabia (1)
Gerard Vockley, MD, PhD (2) · Adrian Lacy, MD (1) · Areeg El-Gharbawy, 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) · Irina A Anselm, MD (1) · Jaume Campistol Plana (1) · Jean Schoenen, MD,PhD (1) · Jerry Vockley, MD, PhD (1)
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R01NS094257 (3), 2014 (2), 2015 (1), 2016 (1), 2017 (1), 2023 (1)