Drugs / Triheptanoin
last change Aug 2026 re-read 2 minutes ago

Triheptanoin

Other / unclassified

Developed for
GLUT1 deficiency syndrome · long chain 3-hydroxyacyl-CoA dehydrogenase deficiency · mitochondrial trifunctional protein deficiency · very long chain acyl-CoA dehydrogenase deficiency · carnitine palmitoyl transferase deficiency · carnitine palmitoyltransferase II deficiency · glucose metabolism disease · glucose transport disorder
+19 more · medium chain acyl-CoA dehydrogenase deficiency · acyl-CoA dehydrogenase 9 deficiency · adult polyglucosan body disease · amyotrophic lateral sclerosis · Barth syndrome · carnitine-acylcarnitine translocase deficiency · disorder of fatty acid oxidation and ketogenesis · disorder of glycogen metabolism · disorder of metabolite absorption and transport · glycogen storage disease I · GYG1-related disorder of glycogen metabolism · Huntington disease · long chain fatty acids, defect in transport of · migraine disorder · pyruvate carboxylase deficiency disease · pyruvate dehydrogenase deficiency · Rett syndrome · alternating hemiplegia of childhood · glycogen storage disease V
Investigated by
Ultragenyx Pharmaceutical Inc · Jerry Vockley, MD, PhD · Juan Pascual · National Institute of Neurological Disorders and Stroke (NINDS) · French National Institute of Health and Medical Research · University of Texas Southwestern Medical Center
+17 more · Adrian Lacy · Areeg El-Gharbawy · Baylor Research Institute · Center for Rare Neurological Diseases, Norcross, GA · Fonds de la Recherche en Santé du Québec · Fundación Ramón Domínguez · Irina A Anselm · Jirair Krikor Bedoyan · Maria Luz Couce Pico · Oregon Health and Science University · Rett Syndrome Research Trust · Richard Bedlack, M.D., Ph.D. · Rigshospitalet, Denmark · University of British Columbia · University of Liege · University of Pittsburgh · University of Sherbrooke

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved US (FDA) DOJOLVI 2020-06-30 fda.gov

Trials 31 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 02 trials
Phase 0 NCT03665636 Oct 2020 → Oct 2021 glycogen storage disease I Areeg El-Gharbawy Completed No outcome recorded
Phase 0 NCT02784847 Jun → Nov 2016 migraine disorder University of Liege Unknown No outcome recorded
Phase 13 trials
Phase 1 NCT06340685 Jul 2024 → Dec 2028 expected pyruvate dehydrogenase deficiency Jirair Krikor Bedoyan Recruiting No outcome recorded
Phase 1 NCT03041363 Mar → Dec 2017 GLUT1 deficiency syndrome, glucose metabolism disease, glucose transport disorder Juan Pascual Completed No outcome recorded
Phase 1 NCT02018315 Jan 2012 → Jan 2013 GLUT1 deficiency syndrome Juan Pascual Completed No outcome recorded
Phase 1/23 trials
Phase 1/2 NCT03506425 Jun 2018 → Mar 2019 amyotrophic lateral sclerosis Richard Bedlack, M.D., Ph.D. Completed No outcome recorded
Phase 1/2 NCT02679235 Apr 2016 → Aug 2018 University of Sherbrooke Completed No outcome recorded
Phase 1/2 NCT02021526 Dec 2015 → Jun 2020 GLUT1 deficiency syndrome Juan Pascual Withdrawn No outcome recorded
Phase 216 trials
Phase 2 NCT06067802 Aug 2026 → Aug 2028 medium chain acyl-CoA dehydrogenase deficiency Jerry Vockley, MD, PhD Withdrawn No outcome recorded
Phase 2 NCT07097311 Jul 2026 → Dec 2027 medium chain acyl-CoA dehydrogenase deficiency Jerry Vockley, MD, PhD Withdrawn No outcome recorded
Phase 2 NCT03642860 Aug 2018 → Aug 2019 GYG1-related disorder of glycogen metabolism Rigshospitalet, Denmark Completed No outcome recorded
Phase 2 NCT03301532 Jun 2018 → Jun 2021 GLUT1 deficiency syndrome University of Texas Southwestern Medical Center Completed No outcome recorded
Phase 2 NCT03181399 Apr 2018 → Sep 2023 GLUT1 deficiency syndrome, glucose metabolism disease, glucose transport disorder University of Texas Southwestern Medical Center Completed No outcome recorded
Phase 2 NCT02696044 Jun 2016 → Feb 2021 Rett syndrome Center for Rare Neurological Diseases, Norcross, GA Unknown No outcome recorded
Phase 2 NCT02599961 Sep 2015 → Oct 2019 GLUT1 deficiency syndrome Ultragenyx Pharmaceutical Inc Terminated No outcome recorded
Phase 2 NCT02214160 Dec 2014 → Dec 2020 carnitine palmitoyl transferase deficiency, carnitine-acylcarnitine translocase deficiency, long chain 3-hydroxyacyl-CoA dehydrogenase deficiency, mitochondrial trifunctional protein deficiency +1 Ultragenyx Pharmaceutical Inc Completed No outcome recorded
Phase 2 NCT02000960 Apr 2014 → Feb 2016 GLUT1 deficiency syndrome University of British Columbia Unknown No outcome recorded
Phase 2 NCT01993186 Feb 2014 → Sep 2017 GLUT1 deficiency syndrome Ultragenyx Pharmaceutical Inc Completed No outcome recorded
Phase 33 trials
Phase 3 NCT05933200 Feb 2023 → Aug 2027 expected disorder of fatty acid oxidation and ketogenesis Ultragenyx Pharmaceutical Inc Active not recruiting No outcome recorded
Phase 3 NCT02960217 Apr 2017 → Oct 2019 GLUT1 deficiency syndrome Ultragenyx Pharmaceutical Inc Terminated No outcome recorded
Phase 3 NCT02201368 Nov 2009 → Mar 2010 long chain fatty acids, defect in transport of Maria Luz Couce Pico Withdrawn No outcome recorded
Phase not stated4 trials
NCT02500082 disorder of metabolite absorption and transport Irina A Anselm No longer available No outcome recorded
NCT02018302 GLUT1 deficiency syndrome Juan Pascual No longer available No outcome recorded
NCT02968953 GLUT1 deficiency syndrome Jerry Vockley, MD, PhD No longer available No outcome recorded
NCT01461304 Barth syndrome, acyl-CoA dehydrogenase 9 deficiency, carnitine palmitoyl transferase deficiency, disorder of glycogen metabolism +4 Jerry Vockley, MD, PhD No longer available No outcome recorded
Also used as a comparator or background therapy in 5 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 24 trials
Phase 2 NCT02919631 Comparator Nov 2016 → Jun 2017 glycogen storage disease V French National Institute of Health and Medical Research Completed No outcome recorded
Phase 2 NCT02453061 Comparator Jun 2015 → Dec 2019 Huntington disease French National Institute of Health and Medical Research Completed No outcome recorded
Phase 2 NCT02432768 Comparator Apr 2015 → Apr 2018 glycogen storage disease V Rigshospitalet, Denmark Completed No outcome recorded
Phase 2 NCT02408354 Comparator Mar 2015 → Apr 2017 alternating hemiplegia of childhood French National Institute of Health and Medical Research Completed No outcome recorded
Phase not stated1 trial
NCT03773770 Background disorder of fatty acid oxidation and ketogenesis Ultragenyx Pharmaceutical Inc Available No outcome recorded

Evidence & citations 18 cited values

Every value below carries the sentence it was read from. 37 sources stand behind the page.

FieldValueCited text
Known as Triheptanoin “The purpose of this project is to study the efficacy of triheptanoin oil in patients with GLUT1 deficiency syndrome.” NCT02014883
30

NCT02919631

NCT02500082

NCT05933200

NCT03665636

NCT02018315

NCT03506425

NCT02968953

NCT06067802

NCT00947960

NCT02679235

NCT02432768

NCT01379625

NCT06340685

NCT03041363

NCT07097311

NCT02696044

NCT02408354

NCT03181399

NCT02018302

NCT02021526

NCT02201368

NCT03301532

NCT02000960

NCT01461304

NCT02784847

NCT02453061

NCT02036853

NCT03773770

NCT03642860

NCT01882062

Known as C7 oil “Triheptanoin (C7 oil) is a triglyceride of the anaplerotic odd-chain fatty acid heptonate.” NCT02000960
Known as Dermofeel tc 7 ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as Dojolvi ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as Dub thg ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as Heptanoin, tri- ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as Lanol 37 t ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as Radiamuls 2375 ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as SpezialölÒ 107 “Triheptanoin (SpezialölÒ 107®)” NCT02201368
Known as Trienanthoin ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as Triheptanoic glyceride ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as Triheptanoina ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as Triheptanoine ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as Trioenanthoin ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585
Known as UX007 “This treatment plan is intended for the use of UX007 (triheptanoin) in the treatment of a single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome.” NCT02968953
6

“Triheptanoin (UX007) is a medium chain triglyceride of three seven-carbon fatty acids (C7), on a glycerol backbone, with a molecular formula of C24H44O6.” NCT02500082

NCT02599961

NCT02214160

NCT01993186

NCT02960217

NCT01886378

Action Restore “The hypothesis is that triheptanoin will restore deficient energy metabolism in these patients” NCT02500082
Modality Other / unclassified “triheptanoin, a 7 carbon triglyceride” NCT00947960
Route Oral “The trial involves 18 subjects ingesting a diet supplemented with triheptanoin” NCT00947960