Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)Phase 3NCT05933200
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
A Phase 3 study testing Triheptanoin and MCT Oil in people with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), run by Ultragenyx Pharmaceutical Inc at 15 sites in 7 countries.
Trial summary
The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs). Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
- What is being tested?
- Triheptanoin and MCT Oil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 69 participants
- Where
- 15 sitesin 7 countries
- Study dates
- 28 Feb 2023to Aug 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Males and females, from 0 (including newborns) to < 18 years of age at time of randomization
- Confirmed diagnosis of LC-FAOD
- Have a caregiver(s) willing and able to assist in all applicable study requirements
- Have a legally authorized representative willing and able to provide written informed consent after the nature of the study has been explained and prior to any research-related procedures, and the study participant to be able to provide age-appropriate written assent
- Have ANY ONE of the following significant clinical manifestations of LC-FAOD:
- At least 2 in the prior year, or 3 in the prior 2 years, of severe major episodes of metabolic decompensation (eg, hypoglycemia, rhabdomyolysis, or exacerbation of cardiomyopathy, requiring ER/urgent care unit visits or hospitalizations)
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- Recurrent symptomatic hypoglycemia (clinical symptoms of hypoglycemia) requiring intervention
- Susceptibility to hypoglycemia after short periods of fasting (less than 4 to 12 hours, depending on age)
- Evidence of functional cardiomyopathy requiring ongoing medical management or clinical manifestation of heart failure
- Sibling(s) with the same pathogenic variant who presented with MCEs
- Participant with pathogenic variants that are known or suspected to be associated with absent or severely reduced enzyme activity or with severe disease manifestations.
- From the period following informed consent to 5 days after the last dose of study drug, females of childbearing potential and fertile males must consent to use highly effective contraception. If female, agree not to become pregnant. If male, agree not to father a child or donate sperm
- Enrollment in the Main Study of Study UX007-CL302
- Age > 2 years
- Liver fat content ≥ 2% and < 20% PDFF as assessed by 1 H-MRS
- Body mass index < 95th percentile
- Able to comply with instructions (remaining still during scan) and requirements (eg, constraints on recent meals, no metallic items or implanted devices in the body, no recent contrast agents) for liver 1 H-MRS scan
Who cannot join
- Enrolled in a clinical study involving concurrent use of an investigational drug product within 30 days before Screening
- Use of a prohibited medication (eg, valproate products or pancreatic lipase inhibitors) within 30 days before Screening, or unwilling to avoid a prohibited medication or other substance that may confound study objectives
- Treatment with triheptanoin within 60 days of Screening
- History of known hypersensitivity to triheptanoin or MCT or its excipients that, in the judgement of the Investigator, places the subject at increased risk for adverse effects
- Caregiver unwilling or unable to sign informed consent, or release of medical records, or follow study procedures
- Have any comorbid conditions, including unstable major organ-system disease(s), that in the opinion of the Investigator places the subject at increased risk of complications, interferes with study participation or compliance, or confounds study objectives or interpretation of results. History of metabolic decompensation(s) with metabolic acidosis, hyperammonemia, and/or liver enzyme elevations does not constitute an exclusion criterion unless in the opinion of the Investigator places the subject at increased risk of complications, interferes with study participation or compliance, or confounds study objectives or interpretation of results.
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- Have a diagnosis of pancreatic insufficiency
- Pregnant, breastfeeding, or planning to become pregnant (self or partner) at any time during the study
- Acute or chronic liver disease other than LC-FAOD that presents with increased risk of liver fat (eg, hepatic cirrhosis, viral toxic or drug hepatitis, diabetes mellitus) and/or metabolic syndrome
- Need for anesthesia/sedation to perform liver 1 H-MRS
Trial locations and contacts
15 sites in 7 countries.
15 sites in 7 countries
Turkey (Türkiye)5 sites
Cukurova University
Adana
Gazi University
Ankara
Ege University
Bornova-İzmir
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
Istanbul, 34093
Cerrahpasa Medical Faculty
Istanbul, 34116
Spain3 sites
University Hospital Santiago de Compostela
A Coruña, 15706
Sant Joan de Deu Hospital (SJD)
Barcelona, Esplugues de Llobregat, 08950
University Hospital 12 de Octubre
Madrid, 28041
Japan2 sites
Juntendo University Hospital
Bunkyo City, Tokyo, 113-8431
The Jikei University Hospital
Minato, Tokyo
Poland2 sites
Gdańksi Uniwersytet Medyczny
Gdansk, Pomeranian Voivodeship, 80-952
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, Masovian Voivodeship, 04-730
Czechia1 site
General University Hospital in Prague-GUH (Všeobecná fakultní nemocnice v Praze- VFN)
Prague, 120-08
Saudi Arabia1 site
King Faisal Specialist Hospital & Research Centre
Riyadh, 11211
About this study
Official title: A Randomized, Double-blind, Multicenter Study to Determine the Effect of Triheptanoin Compared With Even-chain, Medium-chain Triglycerides (MCT) on Major Clinical Events (MCEs) in Pediatric Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs).
Read the detailed description
Participants will be randomly assigned 1:1 to receive triheptanoin or MCT oil. The duration of the study is estimated to be 3.5 to 4 years. The study comprises the following: Screening Period, Baseline (Month 0), Double-blind Treatment Period (including Titration and End of Study Visit), Safety Follow-up Phone Visit, and an Open Access Period.
In addition, a substudy will examine the effect of triheptanoin versus MCT on decreasing liver fat fraction to avoid or improve steatosis in subjects with LC-FAOD. Participants older than 2 years of age at selected sites will be invited to screen for the Liver Substudy.
- Being tested
- Triheptanoin, MCT Oil
- Keywords
- Carnitine Palmitoyl Transferase (CPT) I Deficiency, Carnitine Palmitoyl Transferase (CPT) II Deficiency, Carnitine/acylcarnitine Translocase (CACT) Deficiency, Very Long Chain Acyl-coenzyme A Dehydrogenase (VLCAD) Deficiency, Long-chain 3-hydroxyacyl-coenzyme A Dehydrogenase (LCHAD) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency
- Medical subject headings
- VLCAD deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TriheptanoinDrug
Also called UX007, Dojolvi
Liquid for oral (PO) or enteral feeding tube administration
MCT OilDietary supplement
Also called Medium-chain Triglyceride
Liquid for oral (PO) or enteral feeding tube administration
| Study group | Role | What they receive |
|---|---|---|
| Triheptanoin | Experimental | Participants will be given prescriptions for triheptanoin in mL/day, which will be divided into approximately 4 administrations per day, with precise instructions for administration. Triheptanoin will be titrated up to the first 6 weeks of the study. After titration, the dose is to remain stable for the remainder of the Double-blind Treatment Period. Following the Double-blind Treatment Period, open-label triheptanoin will be provided at participants' current dose during an Open Access Period, if no other options to receive trihepatnoin are available locally. Receives: Drug: Triheptanoin |
| MCT | Active comparator | Participants will be given prescriptions for MCT in mL/day, which will be divided into approximately 4 administrations per day, with precise instructions for administration. MCT will be titrated up to the first 6 weeks of the study. After titration, the dose is to remain stable for the remainder of the Double-blind Treatment Period. Following the Double-blind Treatment Period, open-label triheptanoin will be provided at participants' current dose during an Open Access Period, if no other options to receive trihepatnoin are available locally. Receives: Dietary Supplement: MCT Oil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Event Rate of Major Clinical Events (MCEs)
Measured: Up to Year 4
Secondary outcomes (19)
Annualized Duration of MCEs
Measured: Up to Year 4
Annualized Hypoglycemic Event-rate Captured as MCEs and At-home Clinical Events (HCEs)
Measured: Up to Year 4
Clinical Global Impression of Change [CGI-C] Scale Score
Measured: Up to Year 4
Change From Baseline in Left Ventricular Ejection Fraction
Measured: Baseline, Up to Year 1
Change From Baseline in Left Ventricular Systolic Volume
Measured: Baseline, Up to Year 1
Change From Baseline in Left Ventricular Wall Mass
Measured: Baseline, Up to Year 1
Liver Substudy (Single Study Site Only): Change from Baseline to 6 Months in Hepatic Proton Density Fat Fraction (PDFF%), Assessed by 1H-Magnetic Resonance Spectroscopy (1H-MRS)
Measured: Baseline, Month 6
Annualized Frequency of Rhabdomyolysis-MCEs and Cardiomyopathy-MCEs
Measured: Up to Year 4
Annualized Duration of Rhabdomyolysis-MCEs, Cardiomyopathy-MCEs, and Hypoglycemic-MCEs
Measured: Up to Year 4
Change From Baseline in Caregiver-reported Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scale Score for Participants 2 Years of Age or Older
Measured: Up to Year 4
Change From Baseline in PedsQL Infant Scale Score for Participants Ages 1 to <24 Months
Measured: Up to Year 4
Survival Time
Measured: Up to Year 4
Annualized Hospitalization Days
Measured: Up to Year 4
Number of Missed School or Learning Opportunity Days
Measured: Up to Year 4
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, and Adverse Events of Special Interest (AESIs)
Measured: Up to Year 4
Number of Participants With TEAEs and Serious TEAEs Leading to Dose Modifications, Dose Reductions, Treatment Interruptions, Discontinuations From Study Drug, and Discontinuations From the Study
Measured: Up to Year 4
Plasma Concentration Levels of Heptanoate
Measured: Up to Year 1
Plasma Concentration Levels of Beta Hydroxypentanoate (BHP)
Measured: Up to Year 1
Acceptability and Palatability Survey Scores of Triheptanoin Mixed with Oral Liquids
Measured: Up to Year 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2023
- Study start28 Feb 2023
- Primary completionAug 2027 (estimated)
- Study completionAug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)Studies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 69 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05933200
- Sponsor's protocol ID
- UX007-CL302
- Other IDs
- 2022-001539-10, 2023-509809-76-00, 2023
- Status last verified
- Sep 2026
- First submitted
- 27 Jun 2023
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Multicenter Study to Determine the Effect of Triheptanoin Compared With Even-chain, Medium-chain Triglycerides (MCT) on Major Clinical Events (MCEs) in Pediatric Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
- ClinicalTrials.gov ID
- NCT05933200
- Acronym / sponsor ID
- UX007-CL302
- Keywords
- Carnitine Palmitoyl Transferase (CPT) I Deficiency, Carnitine Palmitoyl Transferase (CPT) II Deficiency, Carnitine/acylcarnitine Translocase (CACT) Deficiency, Very Long Chain Acyl-coenzyme A Dehydrogenase (VLCAD) Deficiency, Long-chain 3-hydroxyacyl-coenzyme A Dehydrogenase (LCHAD) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 69 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 28 Feb 2023 (actual)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Aug 2027 (estimated)
- First posted
- 6 Jul 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs).
Detailed description
Participants will be randomly assigned 1:1 to receive triheptanoin or MCT oil. The duration of the study is estimated to be 3.5 to 4 years. The study comprises the following: Screening Period, Baseline (Month 0), Double-blind Treatment Period (including Titration and End of Study Visit), Safety Follow-up Phone Visit, and an Open Access Period.
In addition, a substudy will examine the effect of triheptanoin versus MCT on decreasing liver fat fraction to avoid or improve steatosis in subjects with LC-FAOD. Participants older than 2 years of age at selected sites will be invited to screen for the Liver Substudy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Triheptanoin | Experimental | Drug: TriheptanoinParticipants will be given prescriptions for triheptanoin in mL/day, which will be divided into approximately 4 administrations per day, with precise instructions for administration. Triheptanoin will be titrated up to the first 6 weeks of the study. After titration, the dose is to remain stable for the remainder of the Double-blind Treatment Period. Following the Double-blind Treatment Period, open-label triheptanoin will be provided at participants' current dose during an Open Access Period, if no other options to receive trihepatnoin are available locally. |
| MCT | Active comparator | Dietary Supplement: MCT OilParticipants will be given prescriptions for MCT in mL/day, which will be divided into approximately 4 administrations per day, with precise instructions for administration. MCT will be titrated up to the first 6 weeks of the study. After titration, the dose is to remain stable for the remainder of the Double-blind Treatment Period. Following the Double-blind Treatment Period, open-label triheptanoin will be provided at participants' current dose during an Open Access Period, if no other options to receive trihepatnoin are available locally. |
Who may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (17 listed)
- Males and females, from 0 (including newborns) to < 18 years of age at time of randomization
- Confirmed diagnosis of LC-FAOD
- Have a caregiver(s) willing and able to assist in all applicable study requirements
- Have a legally authorized representative willing and able to provide written informed consent after the nature of the study has been explained and prior to any research-related procedures, and the study participant to be able to provide age-appropriate written assent
- Have ANY ONE of the following significant clinical manifestations of LC-FAOD:
Key exclusion criteria (10 listed)
- Enrolled in a clinical study involving concurrent use of an investigational drug product within 30 days before Screening
- Use of a prohibited medication (eg, valproate products or pancreatic lipase inhibitors) within 30 days before Screening, or unwilling to avoid a prohibited medication or other substance that may confound study objectives
- Treatment with triheptanoin within 60 days of Screening
- History of known hypersensitivity to triheptanoin or MCT or its excipients that, in the judgement of the Investigator, places the subject at increased risk for adverse effects
- Caregiver unwilling or unable to sign informed consent, or release of medical records, or follow study procedures
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TriheptanoinAlso: UX007, DojolviLiquid for oral (PO) or enteral feeding tube administration | Drug | Triheptanoin |
| MCT OilAlso: Medium-chain TriglycerideLiquid for oral (PO) or enteral feeding tube administration | Dietary supplement | MCT |
Primary outcome
Annualized Event Rate of Major Clinical Events (MCEs)
Time frame: Up to Year 4
19 secondary outcomes
Annualized Duration of MCEs
Time frame: Up to Year 4
Annualized Hypoglycemic Event-rate Captured as MCEs and At-home Clinical Events (HCEs)
Time frame: Up to Year 4
Clinical Global Impression of Change [CGI-C] Scale Score
Time frame: Up to Year 4
Change From Baseline in Left Ventricular Ejection Fraction
Time frame: Baseline, Up to Year 1
Change From Baseline in Left Ventricular Systolic Volume
Time frame: Baseline, Up to Year 1
Change From Baseline in Left Ventricular Wall Mass
Time frame: Baseline, Up to Year 1
Liver Substudy (Single Study Site Only): Change from Baseline to 6 Months in Hepatic Proton Density Fat Fraction (PDFF%), Assessed by 1H-Magnetic Resonance Spectroscopy (1H-MRS)
Time frame: Baseline, Month 6
Annualized Frequency of Rhabdomyolysis-MCEs and Cardiomyopathy-MCEs
Time frame: Up to Year 4
Annualized Duration of Rhabdomyolysis-MCEs, Cardiomyopathy-MCEs, and Hypoglycemic-MCEs
Time frame: Up to Year 4
Change From Baseline in Caregiver-reported Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scale Score for Participants 2 Years of Age or Older
Time frame: Up to Year 4
Change From Baseline in PedsQL Infant Scale Score for Participants Ages 1 to <24 Months
Time frame: Up to Year 4
Survival Time
Time frame: Up to Year 4
Annualized Hospitalization Days
Time frame: Up to Year 4
Number of Missed School or Learning Opportunity Days
Time frame: Up to Year 4
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, and Adverse Events of Special Interest (AESIs)
Time frame: Up to Year 4
Number of Participants With TEAEs and Serious TEAEs Leading to Dose Modifications, Dose Reductions, Treatment Interruptions, Discontinuations From Study Drug, and Discontinuations From the Study
Time frame: Up to Year 4
Plasma Concentration Levels of Heptanoate
Time frame: Up to Year 1
Plasma Concentration Levels of Beta Hydroxypentanoate (BHP)
Time frame: Up to Year 1
Acceptability and Palatability Survey Scores of Triheptanoin Mixed with Oral Liquids
Time frame: Up to Year 1
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Ultragenyx Patient Advocacy/LC-FAOD Disease InformationLink provided by the sponsor.
- Ultragenyx Transparency CommitmentLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Active, not recruitingObservational study
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Ultragenyx Pharmaceutical Inc
15 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Triheptanoin and MCT Oil in people with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), run by Ultragenyx Pharmaceutical Inc at 15 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Turkey (Türkiye), Spain, Japan, Poland, Czechia and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Event Rate of Major Clinical Events (MCEs) (time frame: Up to Year 4). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2026.