Triheptanoin
- Generic Name
- Triheptanoin
- Brand Names
- Dojolvi
- Drug Type
- Small Molecule
- Chemical Formula
- C24H44O6
- 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
- -
Post Study Continuation of C7 for G1D
- First Posted Date
- 2013-12-23
- Last Posted Date
- 2019-04-18
- Lead Sponsor
- Juan Pascual
- Registration Number
- NCT02018302
- Locations
- 🇺🇸
UT Southwestern Medical Center, Dallas, Texas, United States
Pilot Study of Triheptanoin in Patients With Glucose Transporter 1 Deficiency Syndrome
- First Posted Date
- 2013-12-04
- Last Posted Date
- 2016-06-01
- Lead Sponsor
- University of British Columbia
- Target Recruit Count
- 3
- Registration Number
- NCT02000960
- Locations
- 🇨🇦
BC Children's Hospital, Vancouver, British Columbia, Canada
Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
- Conditions
- Mitochondrial Trifunctional Protein DeficiencyPyruvate Carboxylase Deficiency DiseaseVery Long-chain acylCoA Dehydrogenase (VLCAD) DeficiencyCarnitine Palmitoyltransferase Deficiencies (CPT1, CPT2)Long-chain Hydroxyacyl-CoA Dehydrogenase DeficiencyBarth SyndromeGlycogen Storage DisordersACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of
- First Posted Date
- 2011-10-28
- Last Posted Date
- 2021-12-10
- Lead Sponsor
- Jerry Vockley, MD, PhD
- Registration Number
- NCT01461304
- Locations
- 🇺🇸
University of Pittsburgh Division of Medical Genetics, Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
- First Posted Date
- 2011-06-23
- Last Posted Date
- 2017-03-23
- Lead Sponsor
- Oregon Health and Science University
- Target Recruit Count
- 32
- Registration Number
- NCT01379625
- Locations
- 🇺🇸
Oregon Health & Science University, Portland, Oregon, United States
🇺🇸University of Pittsburgh, Pittsburgh, Pennsylvania, United States
Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease
- Conditions
- Interventions
- First Posted Date
- 2009-07-28
- Last Posted Date
- 2018-03-05
- Lead Sponsor
- Baylor Research Institute
- Target Recruit Count
- 23
- Registration Number
- NCT00947960
- Locations
- 🇫🇷
Department of Genetics, Groupe Hospitalier Pitié-Salpêtrière, Paris, France
Drug Development Updates
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