Triheptanoin

Phase 1Recruiting

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Hype Score

Development Stage

✓

Pre-clinical

Phase 1

Phase 2

Phase 3

Approved

Indication / Disease

Pyruvate Dehydrogenase Complex Deficiency

Conditions

Pyruvate Dehydrogenase Complex Deficiency

Trial Timeline

Jul 11, 2024 → Jun 30, 2029

NCT ID

NCT06340685

About Triheptanoin

Triheptanoin is a phase 1 stage product being developed by Ultragenyx Pharmaceutical for Pyruvate Dehydrogenase Complex Deficiency. The current trial status is recruiting. This product is registered under clinical trial identifier NCT06340685. Target conditions include Pyruvate Dehydrogenase Complex 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

9 competing products in Pyruvate Dehydrogenase Complex Deficiency

See all competitors

Product	Company	Stage	Hype Score
Mitapivat + Mitapivat-matching placebo	Agios Pharmaceuticals	Phase 3	72
Mitapivat + Mitapivat-matching placebo	Agios Pharmaceuticals	Phase 3	72
Mitapivat	Agios Pharmaceuticals	Phase 3	72
Placebo + AG-348	Agios Pharmaceuticals	Phase 3	72
Mitapivat	Agios Pharmaceuticals	Approved	80
AG-348	Agios Pharmaceuticals	Phase 3	72
AG-348	Agios Pharmaceuticals	Phase 2	47
RP-L301	Rocket Pharmaceuticals	Phase 1	25
RP-L301	Rocket Pharmaceuticals	Phase 2	44

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