Dojolvi (triheptanoin) / Ultragenyx 
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 30 Diseases   10 Trials   10 Trials   301 News 


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  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Clinical, Journal:  No effect of triheptanoin in patients with phosphofructokinase deficiency. (Pubmed Central) -  Apr 29, 2022   
    Palmitate production and palmitate utilization increased during exercise and increased to a greater extent with triheptanoin treatment in all three patients. This study suggests that triheptanoin treatment has no effect on heart rate or exercise performance despite increased palmitate production and utilization in patients with PFKD.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Journal:  An anaplerotic approach to correct the mitochondrial dysfunction in ataxia-telangiectasia (A-T). (Pubmed Central) -  Mar 25, 2022   
    This study suggests that triheptanoin treatment has no effect on heart rate or exercise performance despite increased palmitate production and utilization in patients with PFKD. These data together show that heptanoate corrects metabolic stress in A-T cells by restoring ER-mitochondria signalling and mitochondrial function and suggest that the parent compound, triheptanoin, has great potential as a novel therapeutic agent for patients with A-T.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx, dizocilpine (MK801) / Merck (MSD)
    Ketogenic Diet Based Metabolic Therapy for Schizophrenia (Spadolini Pavilion Upper Floor) -  Mar 11, 2022 - Abstract #SIRS2022SIRS_145;    
    BHB supplementation largely replicated the effects of ketogenic diet in mice models. Triheptanoin and trihexanoin normalized MK-801-induced social withdrawal but not the locomotor hyperactivity.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Enrollment change, Trial completion date, Trial primary completion date:  Treatment Development of Triheptanoin (G1D) (clinicaltrials.gov) -  Feb 17, 2022   
    P1,  N=12, Active, not recruiting, 
    Trial completion date: May 2023 --> May 2024 | Trial primary completion date: Jun 2022 --> Jun 2023 N=36 --> 12 | Trial completion date: Mar 2025 --> Jul 2022 | Trial primary completion date: Mar 2022 --> Jul 2022
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Review, Journal:  Mitochondrial hepatopathy: Anticipated difficulties in management of fatty acid oxidation defects and urea cycle defects. (Pubmed Central) -  Feb 8, 2022   
    In this review, we discuss the role of carnitine supplementation, dietary interventions, newer therapies like triheptanoin, long-term treatment and approach to positive newborn screening...Practical issues during the acute presentation including differential diagnosis of hyperammonemia, dietary dilemmas, the role of liver transplantation, management of the asymptomatic individual and monitoring are described in detail. A multi-disciplinary team consisting of hepatologists, metabolic specialists and dieticians is required for optimum management and improvement in quality of life for these patients.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Enrollment closed, Enrollment change, Trial completion date, Trial primary completion date:  Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D (clinicaltrials.gov) -  Jan 3, 2022   
    P2,  N=10, Active, not recruiting, 
    Triheptanoin appears to be a promising effective treatment for SCEH Deficiency. Recruiting --> Active, not recruiting | N=15 --> 10 | Trial completion date: Jul 2023 --> Jul 2024 | Trial primary completion date: Feb 2022 --> Jun 2023
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Journal:  Current challenges in the pathophysiology, diagnosis and treatment of paroxysmal movement disorders. (Pubmed Central) -  Dec 16, 2021   
    Alteration of the cerebellar output and modulation of the striatal cAMP turnover offer new perspectives for experimental therapeutics, at least for paroxysmal movement disorders due to selected causes. Further characterization of cell-specific molecular pathways or network dysfunctions that are critically involved in the pathogenesis of paroxysmal movement disorders will likely result in the identification of new biomarkers and testing of innovative targeted therapeutics.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Trial completion:  Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I (clinicaltrials.gov) -  Dec 15, 2021   
    P1,  N=4, Completed, 
    Further characterization of cell-specific molecular pathways or network dysfunctions that are critically involved in the pathogenesis of paroxysmal movement disorders will likely result in the identification of new biomarkers and testing of innovative targeted therapeutics. Recruiting --> Completed
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    [VIRTUAL] Triheptanoin is associated with clinical stability and decreased caudate atrophy in Huntington disease () -  Sep 29, 2021 - Abstract #MDSCongress2021MDS_Congress_1319;    
    P=N/A, P2
    The method was applied to the measurement of 3-hydroxypentanoic acid and 3-oxopentanoic acid concentrations in plasma from subjects receiving the triglyceride triheptanoin (as a source of heptanoate) for the experimental treatment of glucose transporter type I deficiency (G1D) syndrome. Taken together, these results showed that treatment with triheptanoin was associated with clinical stability (UHDRS) and decreased caudate atrophy in HD patients.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Journal:  Long-chain fatty acid oxidation disorders: managed care and specialty pharmacy implications. (Pubmed Central) -  Sep 22, 2021   
    As with any novel agent for rare diseases, the treatment adoption and coverage criteria for rare disease therapy such as this presents challenges for managed care decision makers. Both managed care decision makers and specialty pharmacists must ensure that healthcare professionals provide appropriate patient and caregiver education surrounding the management of LC-FAODs, including vital information on adopting emerging agents into the therapy armamentarium for these disorders.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Journal:  Long-chain fatty acid oxidation disorders and current management strategies. (Pubmed Central) -  Sep 22, 2021   
    This article provides an overview of the epidemiology, pathophysiology, clinical manifestations, and current management approaches for the diagnosis and management of LC-FAODs. It also provides the most recent clinical safety and efficacy data for triheptanoin and other therapies under investigation.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Review, Journal:  The therapeutic potential of triheptanoin in metabolic and neurodegenerative diseases. (Pubmed Central) -  Aug 25, 2021   
    Epilepsy due to deficiency of the GLUT1 transporter, as well as diseases associated with dysregulation of neuronal signaling, have been treated with triheptanoin supplementation, and very recently the advantages of this oil in long-chain fatty acid oxidation disorders have been reported. The present review summarizes the published literature on the metabolism of triheptanoin including clinical reports related to the use of triheptanoin.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Enrollment change, Trial termination:  The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses (clinicaltrials.gov) -  Aug 18, 2021   
    P2,  N=3, Terminated, 
    The present review summarizes the published literature on the metabolism of triheptanoin including clinical reports related to the use of triheptanoin. N=20 --> 3 | Recruiting --> Terminated; Problems with recruitment
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Trial primary completion date:  Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D (clinicaltrials.gov) -  Aug 18, 2021   
    P2,  N=15, Recruiting, 
    N=20 --> 3 | Recruiting --> Terminated; Problems with recruitment Trial primary completion date: Jul 2021 --> Feb 2022
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Review, Journal:  Triheptanoin: First Approval. (Pubmed Central) -  Jul 14, 2021   
    Triheptanoin has also been investigated for use as a treatment in a range of other metabolic disorders or other diseases where energy deficiency is implicated. This article summarizes the milestones in the development of triheptanoin leading to this first regulatory approval for use in the treatment of pediatric and adult patients with LC-FAOD.
  • ||||||||||  amantadine / Generic mfg.
    Clinical, Review, Journal:  Management of Alternating Hemiplegia of Childhood: A Review. (Pubmed Central) -  Mar 18, 2021   
    Besides reviewing existing data about individual agent active against paroxysmal events, we also review the management principles for coexisting neurological issues. However, with rapid advancement in the understanding of molecular pathogenesis and network abnormality of this disease, the treatment paradigm of alternating hemiplegia of childhood may significantly alter over the next decade.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Journal:  Physiological Perspectives on the Use of Triheptanoin as Anaplerotic Therapy for Long Chain Fatty Acid Oxidation Disorders. (Pubmed Central) -  Feb 16, 2021   
    Triheptanoin (triheptanoyl glycerol; UX007, Ultragenyx Pharmaceuticals) is chemically composed of three heptanoate (seven carbon fatty acid) molecules linked to glycerol through ester bonds that has the potential to replete TCA cycle intermediates through production of both acetyl-CoA and propionyl-CoA through medium chain FAO. Compassionate use, retrospective, and recently completed prospective studies demonstrate significant reduction of hypoglycemic events and improved cardiac function in LC-FAOD patients, but a less dramatic effect on muscle symptoms.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Trial completion, Enrollment change:  GLUT1DS: An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome (clinicaltrials.gov) -  Jan 28, 2021   
    P2,  N=20, Completed, 
    Compassionate use, retrospective, and recently completed prospective studies demonstrate significant reduction of hypoglycemic events and improved cardiac function in LC-FAOD patients, but a less dramatic effect on muscle symptoms. Recruiting --> Completed | N=50 --> 20
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Journal:  No effect of triheptanoin on exercise performance in McArdle disease. (Pubmed Central) -  Sep 8, 2020   
    Decreased glycogen breakdown and increased glucose utilization and metabolism via the TCA cycle in epileptogenic brain areas may contribute to triheptanoin's anticonvulsant effects. Despite increased resting plasma malate with triheptanoin, the increase was insufficient to generate a normal TCA turnover during exercise and the treatment has no effect on exercise capacity or oxidative metabolism in patients with McArdle disease.
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Clinical, Journal:  Transition from ketogenic diet to triheptanoin in patients with GLUT1 deficiency syndrome. (Pubmed Central) -  Aug 27, 2020   
    Despite increased resting plasma malate with triheptanoin, the increase was insufficient to generate a normal TCA turnover during exercise and the treatment has no effect on exercise capacity or oxidative metabolism in patients with McArdle disease. No abstract available
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Trial primary completion date:  Diet Treatment Glucose Transporter Type 1 Deficiency (G1D) (clinicaltrials.gov) -  Aug 8, 2020   
    P2,  N=45, Active, not recruiting, 
    Enrolling by invitation --> Active, not recruiting | N=150 --> 94 | Trial completion date: Sep 2021 --> Oct 2020 | Trial primary completion date: Sep 2021 --> Oct 2020 Trial primary completion date: May 2020 --> Jul 2022
  • ||||||||||  Dojolvi (triheptanoin) / Ultragenyx
    Trial completion date, Trial primary completion date:  Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin (clinicaltrials.gov) -  Jul 17, 2020   
    P2,  N=12, Recruiting, 
    Trial primary completion date: May 2020 --> Jul 2022 Trial completion date: Feb 2021 --> Feb 2022 | Trial primary completion date: Feb 2020 --> Feb 2021