triheptanoin

Pre-clinicalCompleted

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Hype Score

Development Stage

Pre-clinical

Phase 1

Phase 2

Phase 3

Approved

Indication / Disease

Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency

Conditions

Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome

Trial Timeline

— → —

NCT ID

NCT01461304

About triheptanoin

triheptanoin is a pre-clinical stage product being developed by Ultragenyx Pharmaceutical for Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency. The current trial status is completed. This product is registered under clinical trial identifier NCT01461304. Target conditions include Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency.

Hype Score Breakdown

Clinical

Activity

Company

Novelty

Community

Clinical Trials (14)

NCT ID	Phase	Status	Start	Completion	Indication
NCT01461304	Pre-clinical	Completed	—	—	Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency
NCT02968953	Pre-clinical	Completed	—	—	Glucose Transporter 1 Deficiency Syndrome
NCT03773770	Pre-clinical	Active	—	—	Long Chain Fatty Acid Oxidation Disorders
NCT07097311	Phase 2	Recruiting	Jul 1, 2026	Apr 1, 2028	Medium-chain Acyl-CoA Dehydrogenase Deficiency
NCT06067802	Phase 2	Active	Feb 1, 2026	Jul 1, 2028	Medium-chain Acyl-CoA Dehydrogenase Deficiency
NCT06340685	Phase 1	Recruiting	Jul 11, 2024	Jun 30, 2029	Pyruvate Dehydrogenase Complex Deficiency
NCT05933200	Phase 3	Active	Feb 28, 2023	Aug 1, 2027	Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
NCT03665636	Phase 1	Completed	Oct 16, 2020	Oct 21, 2021	Glycogen Storage Disease Type I
NCT03506425	Phase 1/2	Completed	Jun 21, 2018	Mar 28, 2019	ALS
NCT02696044	Phase 2	UNKNOWN	Jun 1, 2016	Feb 1, 2022	Rett Syndrome
NCT02432768	Phase 2	Completed	Apr 1, 2015	Feb 1, 2019	Glycogen Storage Disease Type V
NCT02000960	Phase 2	UNKNOWN	Apr 1, 2014	Oct 1, 2016	Glucose Transporter Type 1 Deficiency Syndrome
NCT02036853	Phase 2	Completed	Feb 20, 2014	Jun 30, 2019	Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
NCT00947960	Phase 2	Completed	Jun 1, 2009	Jul 1, 2015	Adult Polyglucosan Body Disease

Competing Products

10 competing products in Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency

See all competitors

Product	Company	Stage	Hype Score
Roxadustat + Placebo	Astellas Pharma	Phase 3	77
Benralizumab Arm A + Benralizumab Arm B + Placebo	AstraZeneca	Phase 3	77
Benralizumab Arm A + Benralizumab Arm B + Benralizumab Arm C + Placebo	AstraZeneca	Phase 3	77
Ruxolitinib	Novartis	Pre-clinical	23
Etavopivat	Novo Nordisk	Phase 2	51
Ipilimumab	Bristol Myers Squibb	Phase 1/2	40
BAY207543 (Bepanthol, Bepantol® Derma Spray)	Bayer	Approved	82
BAY207543 (Bepanthol, Bepantol® Derma Spray)	Bayer	Approved	82
Chondrogen + Chondrogen + Placebo	Mesoblast	Phase 1/2	36
Mesenchymal Stem Cells + Hyaluronan	Mesoblast	Phase 1/2	36

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