Triheptanoin

Summary

Triheptanoin (CHEMBL4297585) is an approved small molecule (ATC A16AX17); indicated across 12 conditions including huntington disease and glycogen storage disease v.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A16AX17
• Indications: 12 conditions
• Clinical trials: 34
• Chemistry: 428.6 Da · C24H44O6

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297585
Name	Triheptanoin
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	69286
ATC	A16AX17
Molecular formula	C24H44O6
Molecular weight	428.6
InChIKey	PJHKBYALYHRYSK-UHFFFAOYSA-N

SMILES: CCCCCCC(=O)OCC(COC(=O)CCCCCC)OC(=O)CCCCCC

IUPAC name: 2,3-di(heptanoyloxy)propyl heptanoate

Also known as: Dermofeel tc 7, Dermofeel tc-7, Dojolvi, Dub thg, Heptanoin, tri-, Lanol 37 t, Radiamuls 2375, Trienanthoin, Triheptanoic glyceride, Triheptanoin, Triheptanoina

Patent coverage: 553 distinct patent families (1,262 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

12 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
Huntington disease	2	MONDO:0007739	MONDO:0007739
glycogen storage disease V	2	MONDO:0009293	MONDO:0009293
glycogen storage disease VII	2	MONDO:0009295	MONDO:0009295
Rett syndrome	2	MONDO:0010726	MONDO:0010726
hemiplegia	2	MONDO:0001170	EFO:0009453
ataxia telangiectasia	2	MONDO:0008840	MONDO:0008840
amyotrophic lateral sclerosis	1	MONDO:0004976	MONDO:0004976
metabolic disease	1	MONDO:0005066	EFO:0000589
glycogen storage disease I	0	MONDO:0002413	Orphanet:364
migraine disorder	0	MONDO:0005277	MONDO:0005277

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 34.

Phase distribution

Phase	Trials
PHASE2	19
Not specified	5
PHASE1/PHASE2	3
PHASE1	3
PHASE3	2
EARLY_PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05933200	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
NCT02960217	PHASE3	TERMINATED	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)
NCT07097311	PHASE2	RECRUITING	Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
NCT00947960	PHASE2	COMPLETED	Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease
NCT01379625	PHASE2	COMPLETED	Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
NCT01882062	PHASE2	COMPLETED	Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease
NCT01886378	PHASE2	COMPLETED	A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
NCT01993186	PHASE2	COMPLETED	Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
NCT02000960	PHASE2	UNKNOWN	Pilot Study of Triheptanoin in Patients With Glucose Transporter 1 Deficiency Syndrome
NCT02021526	PHASE1/PHASE2	WITHDRAWN	Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D)
NCT02036853	PHASE2	COMPLETED	An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome
NCT02214160	PHASE2	COMPLETED	Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
NCT02408354	PHASE2	COMPLETED	Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood
NCT02432768	PHASE2	COMPLETED	The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)
NCT02599961	PHASE2	TERMINATED	Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
NCT02679235	PHASE1/PHASE2	COMPLETED	Brain Energy and Aging With Triheptanoin
NCT02696044	PHASE2	UNKNOWN	Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin
NCT02919631	PHASE2	COMPLETED	Triheptanoin in Mc Ardle
NCT03181399	PHASE2	COMPLETED	Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)
NCT03301532	PHASE2	COMPLETED	Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
NCT03506425	PHASE1/PHASE2	COMPLETED	A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
NCT03642860	PHASE2	COMPLETED	The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses
NCT04513002	PHASE2	COMPLETED	Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis
NCT06067802	PHASE2	SUSPENDED	Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
NCT06340685	PHASE1	RECRUITING	Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
NCT02018315	PHASE1	COMPLETED	Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)
NCT03041363	PHASE1	COMPLETED	Treatment Development of Triheptanoin (G1D)
NCT02784847	EARLY_PHASE1	UNKNOWN	A Pilot Trial of Triheptanoin for the Preventive Treatment of Migraine
NCT03665636	EARLY_PHASE1	COMPLETED	Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I
NCT03773770	Not specified	AVAILABLE	Expanded Access to Triheptanoin
NCT01461304	Not specified	NO_LONGER_AVAILABLE	Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
NCT02018302	Not specified	NO_LONGER_AVAILABLE	Post Study Continuation of C7 for G1D
NCT02500082	Not specified	NO_LONGER_AVAILABLE	Triheptanoin (UX007) to Treat Citrate Transporter Deficiency
NCT02968953	Not specified	NO_LONGER_AVAILABLE	Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).