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

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Phase 1

Recruiting

Conditions: Pyruvate Dehydrogenase Complex Deficiency

Interventions: Drug: Triheptanoin

First Posted Date: 2024-04-01

Last Posted Date: 2024-06-05

Lead Sponsor: Jirair Krikor Bedoyan

Target Recruit Count: 6

Registration Number: NCT06340685

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

Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Phase 2

Recruiting

Conditions: Medium-chain Acyl-CoA Dehydrogenase Deficiency

Interventions: Drug: Triheptanoin

First Posted Date: 2023-10-05

Last Posted Date: 2024-05-03

Lead Sponsor: Jerry Vockley, MD, PhD

Target Recruit Count: 8

Registration Number: NCT06067802

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

A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD

Phase 3

Active, not recruiting

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

Interventions: Dietary Supplement: MCT Oil
Drug: Triheptanoin

First Posted Date: 2023-07-06

Last Posted Date: 2024-09-30

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 60

Registration Number: NCT05933200

Locations: 🇯🇵
Juntendo University Hospital, Bunkyo City, Tokyo, Japan
🇯🇵
The Jikei University Hospital, Minato City, Tokyo, Japan
🇨🇿
General University Hospital in Prague-GUH (Všeobecná fakultní nemocnice v Praze- VFN), Prague, Czechia

and more 13 locations

Expanded Access to Triheptanoin

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

First Posted Date: 2018-12-12

Last Posted Date: 2024-09-05

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Registration Number: NCT03773770

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

Early Phase 1

Completed

Conditions: Glycogen Storage Disease Type I

Interventions: Drug: Triheptanoin

First Posted Date: 2018-09-11

Last Posted Date: 2021-12-15

Lead Sponsor: Areeg El-Gharbawy

Target Recruit Count: 4

Registration Number: NCT03665636

Locations: 🇺🇸
Duke University Medical Center, Durham, North Carolina, United States

The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses

Phase 2

Completed

Conditions: GYG1 DEFICIENCY
Debrancher Deficiency
Tarui Disease

Interventions: Drug: Placebo Oil
Drug: Triheptanoin

First Posted Date: 2018-08-22

Last Posted Date: 2024-02-22

Lead Sponsor: Rigshospitalet, Denmark

Target Recruit Count: 3

Registration Number: NCT03642860

Locations: 🇩🇰
Copenhagen Neuromuscular Center, Copenhagen, Denmark

A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)

Phase 1

Completed

Conditions: ALS

Interventions: Drug: Triheptanoin

First Posted Date: 2018-04-24

Last Posted Date: 2019-12-24

Lead Sponsor: Richard Bedlack, M.D., Ph.D.

Target Recruit Count: 15

Registration Number: NCT03506425

Locations: 🇺🇸
Duke University, Durham, North Carolina, United States

Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D

Phase 2

Completed

Conditions: GLUT1DS1

Interventions: Drug: Triheptanoin

First Posted Date: 2017-10-04

Last Posted Date: 2024-01-17

Lead Sponsor: University of Texas Southwestern Medical Center

Target Recruit Count: 10

Registration Number: NCT03301532

Locations: 🇺🇸
UTexasSouthwestern, Dallas, Texas, United States

Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)

Phase 2

Active, not recruiting

Conditions: GLUT1DS1
Glucose Transport Defect
Glucose Transporter Type 1 Deficiency Syndrome
Glucose Transporter Protein Type 1 Deficiency Syndrome
Epilepsy
Glucose Metabolism Disorders
Glut1 Deficiency Syndrome 1, Autosomal Recessive

Interventions: Drug: Triheptanoin

First Posted Date: 2017-06-08

Last Posted Date: 2024-01-26

Lead Sponsor: University of Texas Southwestern Medical Center

Target Recruit Count: 45

Registration Number: NCT03181399

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

Treatment Development of Triheptanoin (G1D)

Phase 1

Completed

Conditions: Glucose Transporter Type 1 Deficiency Syndrome
Glucose Transporter Protein Type 1 Deficiency Syndrome
GLUT1DS1
Epilepsy
Glucose Transport Defect
Glut1 Deficiency Syndrome 1, Autosomal Recessive
Glucose Metabolism Disorders

Interventions: Drug: Triheptanoin

First Posted Date: 2017-02-02

Last Posted Date: 2022-12-22

Lead Sponsor: Juan Pascual

Target Recruit Count: 12

Registration Number: NCT03041363

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

Drug Development Updates

Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.

© Copyright 2024. All Rights Reserved by MedPath

Home Terms & Conditions Privacy Policy