Triheptanoin

Phase 2Active

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Hype Score

Development Stage

✓

Pre-clinical

✓

Phase 1

Phase 2

Phase 3

Approved

Indication / Disease

Medium-chain Acyl-CoA Dehydrogenase Deficiency

Conditions

Medium-chain Acyl-CoA Dehydrogenase Deficiency

Trial Timeline

Feb 1, 2026 → Jul 1, 2028

NCT ID

NCT06067802

About Triheptanoin

Triheptanoin is a phase 2 stage product being developed by Ultragenyx Pharmaceutical for Medium-chain Acyl-CoA Dehydrogenase Deficiency. The current trial status is active. This product is registered under clinical trial identifier NCT06067802. Target conditions include Medium-chain Acyl-CoA Dehydrogenase 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

2 competing products in Medium-chain Acyl-CoA Dehydrogenase Deficiency

See all competitors

Product	Company	Stage	Hype Score
Triheptanoin	Ultragenyx Pharmaceutical	Phase 2	47
Sodium phenylbutyrate	Zevra Therapeutics	Phase 2	44

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