CLINICAL TRIALS PROFILE FOR TRIHEPTANOIN

All Clinical Trials for Triheptanoin

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00328159 ↗	Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids	Completed	Assistance Publique - Hôpitaux de Paris	N/A	2006-06-01	Usual dietary therapies of mitochondrial fatty acid oxidation disorders (FAO) are based on 3 strategies: - limitation of lipid intake in the diet; - supplementation of the diet with medium-chain triglycerides (MCT) for patients affected with disorders of long-chain FAO; - some specific supplementations (for example, L-carnitine). These strategies are often ineffective. The aim of the present study is to evaluate new therapeutic ways based on the underlying energetic defect observed in these disorders. The long-term goal is to develop efficient therapies of these disorders.
NCT00947960 ↗	Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease	Completed	Ultragenyx Pharmaceutical Inc	Phase 2	2009-06-01	The purpose of the study is to determine if triheptanoin is an effective treatment for the symptoms of Adult Polyglucosan Body Disease.
NCT00947960 ↗	Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease	Completed	Baylor Research Institute	Phase 2	2009-06-01	The purpose of the study is to determine if triheptanoin is an effective treatment for the symptoms of Adult Polyglucosan Body Disease.
NCT00983788 ↗	Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects	Completed	Groupe Hospitalier Pitie-Salpetriere	Phase 2	2009-10-01	The investigators propose to evaluate the effect of bezafibrate on metabolism during exercise in 22 adult patients affected with carnitine palmitoyltransferase II (CPTII) or very-long chain acyl-CoA-dehydrogenase (VLCAD) deficiencies. This study will be an 9-month, randomized, double-blind, placebo-controlled crossover trial. The trial will be conducted in two centers: Institut de Myologie, Pitié-Salpêtrière Hospital in France, and Rigshospitalet, University of Copenhagen, in Denmark. The main criteria for assessing the potential effect of this drug will be the fat oxidation rate studied during a moderate workload on cycle ergometer, after infusion of stable isotopes (palmitate and glucose tracers).
NCT00983788 ↗	Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects	Completed	Rigshospitalet, Denmark	Phase 2	2009-10-01	The investigators propose to evaluate the effect of bezafibrate on metabolism during exercise in 22 adult patients affected with carnitine palmitoyltransferase II (CPTII) or very-long chain acyl-CoA-dehydrogenase (VLCAD) deficiencies. This study will be an 9-month, randomized, double-blind, placebo-controlled crossover trial. The trial will be conducted in two centers: Institut de Myologie, Pitié-Salpêtrière Hospital in France, and Rigshospitalet, University of Copenhagen, in Denmark. The main criteria for assessing the potential effect of this drug will be the fat oxidation rate studied during a moderate workload on cycle ergometer, after infusion of stable isotopes (palmitate and glucose tracers).
NCT01379625 ↗	Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder	Completed	University of Pittsburgh	Phase 2	2011-09-01	Humans eat long-chain fat in their diet and use it for energy during exercise and during periods of fasting. Patients with long-chain fatty acid oxidation disorders cannot use dietary fat for energy. They sometimes develop muscle breakdown, and severe pain with exercise or illness. They can also develop a heart that does not function properly. These patients are tired and expend less energy than people who do not have a long-chain fatty acid oxidation disorder. However, they can use a supplement oil called medium chain triglyceride or MCT. This study will determine if a new experimental oil called Triheptanoin can decrease the muscle pain and increase the heart function and the amount of energy in patients with long-chain fatty acid oxidation disorders. Funding source - FDA's OOPD
NCT01379625 ↗	Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder	Completed	Oregon Health and Science University	Phase 2	2011-09-01	Humans eat long-chain fat in their diet and use it for energy during exercise and during periods of fasting. Patients with long-chain fatty acid oxidation disorders cannot use dietary fat for energy. They sometimes develop muscle breakdown, and severe pain with exercise or illness. They can also develop a heart that does not function properly. These patients are tired and expend less energy than people who do not have a long-chain fatty acid oxidation disorder. However, they can use a supplement oil called medium chain triglyceride or MCT. This study will determine if a new experimental oil called Triheptanoin can decrease the muscle pain and increase the heart function and the amount of energy in patients with long-chain fatty acid oxidation disorders. Funding source - FDA's OOPD

Clinical Trial Conditions for Triheptanoin

Condition Name

Condition Name for Triheptanoin
Intervention	Trials
Glucose Transporter Type 1 Deficiency Syndrome	7
GLUT1 Deficiency Syndrome	4
GLUT1DS1	3
Trifunctional Protein (TFP) Deficiency	2

Condition MeSH

Condition MeSH for Triheptanoin
Intervention	Trials
Syndrome	12
Carbohydrate Metabolism, Inborn Errors	11
Metabolic Diseases	6
Glycogen Storage Disease	5

Clinical Trial Locations for Triheptanoin

Trials by Country

Trials by Country for Triheptanoin
Location	Trials
United States	51
France	7
United Kingdom	6
Spain	6
Australia	3

Trials by US State

Trials by US State for Triheptanoin
Location	Trials
Texas	10
Pennsylvania	9
Florida	5
Colorado	3
Massachusetts	3

Clinical Trial Progress for Triheptanoin

Clinical Trial Phase

Clinical Trial Phase for Triheptanoin
Clinical Trial Phase	Trials
PHASE2	1
PHASE1	1
Phase 3	3

Clinical Trial Status

Clinical Trial Status for Triheptanoin
Clinical Trial Phase	Trials
Completed	15
Recruiting	7
No longer available	4

Clinical Trial Sponsors for Triheptanoin

Sponsor Name

Sponsor Name for Triheptanoin
Sponsor	Trials
Ultragenyx Pharmaceutical Inc	21
University of Texas Southwestern Medical Center	5
Institut National de la Santé Et de la Recherche Médicale, France	4

Sponsor Type

Sponsor Type for Triheptanoin
Sponsor	Trials
Other	44
Industry	21
NIH	3

Clinical Trials Update, Market Analysis, and Projection for Triheptanoin

Last updated: October 28, 2025

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Introduction

Triheptanoin, also known as C7 glycerol triglyceride, is a triglyceride composed of three seven-carbon fatty acids. Its unique metabolic profile has driven interest in rare metabolic disorders and potential neurological applications. As an investigative drug, triheptanoin has garnered substantial attention due to promising early clinical data and evolving market dynamics. This report synthesizes recent clinical trial developments, market landscape, and future outlooks to assist stakeholders in strategic planning.

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Clinical Trials Update

Ongoing and Recent Clinical Trials

Recent years have witnessed an increase in clinical evaluations centered on triheptanoin's therapeutic scope. The most significant development is its continued investigation as a treatment for GLUT1 deficiency syndrome, a rare neurological disorder characterized by impaired glucose transport across the blood-brain barrier. The Phase II/III trials led by companies like Ultragenyx and trial registries such as ClinicalTrials.gov have shown promising results.

• GLUT1 Deficiency Syndrome (G1D): Several trials, including NCT02724103 and NCT03757278, have focused on assessing the safety, tolerability, and efficacy of triheptanoin. Preliminary data indicates improvements in seizure frequency, cognitive function, and energy metabolism.

• Metabolic Disorders: Studies like NCT02562781 are exploring triheptanoin's efficacy in long-chain fatty acid oxidation disorders (LC-FAOD). Results have demonstrated reduction in hospitalizations and improved energy levels.

• Neurological Conditions: Exploratory trials assess its potential in Alzheimer’s disease and other neurodegenerative conditions, driven by preclinical evidence suggesting enhanced cerebral energy metabolism.

Regulatory Status

While triheptanoin has received orphan drug designation for certain indications, such as G1D (by the FDA and EMA), it has yet to secure full approval. The orphan status provides incentives like tax credits and market exclusivity, expediting commercialization pathways once efficacy and safety are established.

Challenges

• Limited Large-Scale Data: Most trials are small, phase II studies, necessitating further validation.
• Biomarker Development: Better biomarkers are needed to quantify metabolic improvements reliably.
• Expanding Indications: While preliminary results are encouraging, broader indications require extensive clinical validation.

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Market Analysis

Market Landscape

The global market for metabolic disorder therapies, especially in rare diseases linked to fatty acid oxidation, has been expanding. The orphan drug segment, with an increasing number of novel therapies, is projected to reach $185 billion by 2030[1].

Key Market Players

• Ultragenyx Pharmaceutical: Leads research with its triheptanoin-based candidate, MTP-131 (Elamipretide), for mitochondrial diseases, indicating an interest in metabolic modulators.
• Other Biotech Firms: Several startups are investing in lipid-based metabolic therapies, with triheptanoin poised as a promising candidate due to its unique mechanism.

Market Drivers

• Unmet Medical Needs: For rare metabolic disorders like G1D, current treatment options are limited, and triheptanoin offers a potential disease-modifying approach.
• Regulatory Incentives: Orphan drug designations facilitate faster development and commercialization.
• Advances in Precision Medicine: Targeted therapies aligned with metabolic pathways enhance market acceptance.

Market Barriers

• Limited Awareness: Low clinician familiarity hampers immediate adoption.
• High Development Costs: Rare disease trials often involve complex, costly processes.
• Pricing and Reimbursement: High costs associated with orphan drugs pose challenges for reimbursement and access.

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Market Projection

Short-Term Outlook (Next 5 Years)

• Pending positive clinical trial results, triheptanoin could reach regulatory approval for G1D and LC-FAOD indications.
• Revenue estimates for approved indications are projected to be $50–$150 million annually within five years, primarily driven by North American and European markets.
• The expansion into neurological disorders could generate additional multi-million dollar opportunities, contingent upon trial outcomes.

Long-Term Outlook (Next 10–15 Years)

• Widespread adoption for multiple metabolic and neurodegenerative disorders—especially if phase III trials confirm efficacy—may elevate revenues to $500 million or more annually.
• The therapy's potential role in broader mitochondrial and neurodegenerative diseases can position it as a versatile metabolic modulator, supporting long-term market growth.
• Strategic collaborations with global healthcare systems and patient advocacy groups will be pivotal.

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Strategic Implications

• Investors should monitor trial progress and regulatory milestones closely, given the high reward potential in rare metabolic diseases.
• Pharmaceutical developers must prioritize robust clinical data to differentiate triheptanoin in a competitive orphan drug landscape.
• Healthcare providers should stay informed on emerging evidence to optimize patient management, particularly in genetic metabolic disorders.

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Key Takeaways

• Clinical data for triheptanoin remains promising, especially for GLUT1 deficiency syndrome and fatty acid oxidation disorders, with ongoing trials poised for pivotal results.
• Market potential is significant within the rare disease space, where unmet needs and regulatory incentives can accelerate approval and adoption.
• Challenges such as limited large-scale data, regulatory hurdles, and high development costs must be navigated proactively.
• Future projections forecast moderate to high revenue growth within five years, with expansive potential considering emerging indications.
• Collaborative strategies and continued innovation will be critical to fully realizing triheptanoin’s commercial and therapeutic potential.

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FAQs

1. What is triheptanoin and how does it work?
Triheptanoin is a triglyceride composed of three seven-carbon fatty acids. Once metabolized, it produces energy substrates that bypass defective metabolic pathways, particularly in fatty acid oxidation disorders and certain neurological conditions, enhancing cellular energy production.

2. Which rare diseases are being targeted by current clinical trials of triheptanoin?
Primary focus areas include GLUT1 deficiency syndrome, long-chain fatty acid oxidation disorders, and exploratory studies in neurodegenerative diseases such as Alzheimer’s.

3. Is triheptanoin approved for medical use?
As of now, triheptanoin has not received full regulatory approval but holds orphan drug designations, facilitating accelerated review processes for specific indications.

4. What are the main hurdles for triheptanoin’s commercialization?
Major challenges include limited large-scale trial data, the need for reliable biomarkers, high development costs, and navigating regulatory requirements for broader indications.

5. How large is the market opportunity for triheptanoin?
The immediate market for approved rare disease indications could reach hundreds of millions annually within five years, with long-term growth potential expanding into broader neurological and mitochondrial disorders.

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References

1. Grand View Research. (2022). Orphan Drugs Market Size, Share & Trends Analysis Report.
2. ClinicalTrials.gov. Retrieved from https://clinicaltrials.gov/
3. European Medicines Agency. (2023). Orphan Designation List.
4. MarketsandMarkets. (2022). Rare Disease Treatment Market.

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Disclaimer: This analysis reflects current data and projections; ongoing research may alter future outlooks.