Triheptanoin

Generic Name: Triheptanoin

Brand Names: Dojolvi

Drug Type: Small Molecule

Chemical Formula: C_₂₄H_₄₄O_₆

CAS Number: 620-67-7

Unique Ingredient Identifier: 2P6O7CFW5K

Background: Triheptanoin is a source of heptanoate fatty acids, which can be metabolized without the enzymes of long chain fatty acid oxidation. In clinical trials, patients with long chain fatty acid oxidation disorders (lc-FAODs) treated with triheptanoin are less likely to develop hypoglycemia, cardiomyopathy, rhabdomyolysis, and hepatomegaly. Complications in lc-FAOD patients are reduced from approximately 60% to approximately 10% with the addition of triheptanoin.

Triheptanoin was granted FDA approval on 30 June 2020.

Indication: Triheptanoin is a medium chain triglyceride indicated to provide calories and fatty acids to treat long chain fatty acid oxidation disorders (lc-FAODs).

Associated Conditions: Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Associated Therapies: -

Clinical Trials

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

Conditions: Glucose Transporter 1 Deficiency Syndrome

First Posted Date: 2016-11-21

Last Posted Date: 2019-02-08

Lead Sponsor: Jerry Vockley, MD, PhD

Registration Number: NCT02968953

Locations: 🇺🇸
Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States

Triheptanoin in Mc Ardle

Phase 2

Conditions: Glycogen Storage Disease Type V

Interventions: Drug: Placebo oil
Drug: Triheptanoin

First Posted Date: 2016-09-29

Last Posted Date: 2016-09-29

Lead Sponsor: Institut National de la Santé Et de la Recherche Médicale, France

Target Recruit Count: 12

Registration Number: NCT02919631

A Pilot Trial of Triheptanoin for the Preventive Treatment of Migraine

Early Phase 1

Conditions: Migraine

Interventions: Drug: Triheptanoin

First Posted Date: 2016-05-27

Last Posted Date: 2016-05-27

Lead Sponsor: University of Liege

Target Recruit Count: 10

Registration Number: NCT02784847

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

Phase 2

Conditions: Rett Syndrome

Interventions: Drug: triheptanoin

First Posted Date: 2016-03-02

Last Posted Date: 2020-07-17

Lead Sponsor: Center for Rare Neurological Diseases, Norcross, GA

Target Recruit Count: 12

Registration Number: NCT02696044

Locations: 🇺🇸
Center for Rare Neurological Diseases, Norcross, Georgia, United States

Triheptanoin (UX007) to Treat Citrate Transporter Deficiency

Conditions: Citrate Transporter Deficiency
SLC13A5 Gene Mutation

First Posted Date: 2015-07-16

Last Posted Date: 2016-01-28

Lead Sponsor: Irina A Anselm

Registration Number: NCT02500082

Locations: 🇺🇸
Department of Neurology, Boston Children's Hospital, Boston, Massachusetts, United States

The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)

Phase 2

Completed

Conditions: Glycogen Storage Disease Type V

Interventions: Drug: Triheptanoin
Other: Placebo oil

First Posted Date: 2015-05-04

Last Posted Date: 2019-10-16

Lead Sponsor: Rigshospitalet, Denmark

Target Recruit Count: 22

Registration Number: NCT02432768

Locations: 🇩🇰
Copenhagen Neuromuscular Center, 3342, Rigshospitalet, Copenhagen, Denmark

Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood

Phase 2

Completed

Conditions: Alternating Hemiplegia of Childhood

Interventions: Drug: Placebo
Drug: Triheptanoin

First Posted Date: 2015-04-03

Last Posted Date: 2020-02-12

Lead Sponsor: Institut National de la Santé Et de la Recherche Médicale, France

Target Recruit Count: 10

Registration Number: NCT02408354

Locations: 🇫🇷
Groupe hospitalier Pitié Salpêtrière, Paris, France

An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome

Phase 2

Completed

Conditions: Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)

Interventions: Drug: Triheptanoin

First Posted Date: 2014-01-15

Last Posted Date: 2021-01-28

Lead Sponsor: Adrian Lacy

Target Recruit Count: 20

Registration Number: NCT02036853

Locations: 🇺🇸
Cook Childrens Medical Center, Fort Worth, Texas, United States

Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D)

Phase 1

Withdrawn

Conditions: Glucose Transporter Type 1 Deficiency Syndrome
Glut1 Deficiency Syndrome

Interventions: Drug: Triheptanoin

First Posted Date: 2013-12-27

Last Posted Date: 2019-02-15

Lead Sponsor: Juan Pascual

Registration Number: NCT02021526

Locations: 🇺🇸
UT Southwestern Medical Center, Dallas, Texas, United States

Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)

Phase 1

Completed

Conditions: Glucose Transporter Type 1 Deficiency Syndrome
GLUT1 Deficiency Syndrome

Interventions: Drug: Triheptanoin

First Posted Date: 2013-12-23

Last Posted Date: 2019-10-31

Lead Sponsor: Juan Pascual

Target Recruit Count: 14

Registration Number: NCT02018315

Locations: 🇺🇸
UT Southwestern Medical Center, Dallas, Texas, United States

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