Long Chain Fatty Acid Oxidation DisordersNCT03773770
Expanded Access to Triheptanoin
A study testing Triheptanoin in people with Long Chain Fatty Acid Oxidation Disorders, run by Ultragenyx Pharmaceutical Inc.
Trial summary
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Long Chain Fatty Acid Oxidation Disorders
- What is being tested?
- Triheptanoin
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- Child, Adult, Older adult
- The participant must not be eligible for a UX007 clinical trial
Trial locations and contacts
No sites are listed yet.
Early Access contact
About this study
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
Read the detailed description
Long Chain Fatty Acid Oxidation Disorders (LC-FAOD) and other Non-FAOD conditions, where applicable: Expanded access may be available outside of the US in countries prior to approval by the local regulatory agencies.
For full details, please visit the links provided below.
- Being tested
- Triheptanoin
- Keywords
- Expanded Access, Compassionate Use, Carnitine Palmitoyltransferase Deficiency, CPT I, CPT II, Very Long Chain acyl-CoA Dehydrogenase Deficiency, VLCAD, Long Chain 3-hydroxy-acyl-CoA Dehydrogenase Deficiency, LHCAD, Trifunctional Protein Deficiency, TFP, Carnitine-acylcarnitine Translocase Deficiency, CACT, LC-FAOD
- Medical subject headings
- VLCAD deficiency, Trifunctional Protein Deficiency With Myopathy And Neuropathy, Carnitine-Acylcarnitine Translocase Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TriheptanoinDrug
Also called UX007
Liquid for oral (PO) or enteral tube administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2018
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 DisordersStudies 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
- Expanded access
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
- NCT03773770
- Sponsor's protocol ID
- UX007-EAP
- Status last verified
- Jul 2026
- First submitted
- 10 Dec 2018
- Last updated
- 31 Jul 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access to Triheptanoin
- ClinicalTrials.gov ID
- NCT03773770
- Acronym / sponsor ID
- UX007-EAP
- Keywords
- Expanded Access, Compassionate Use, Carnitine Palmitoyltransferase Deficiency, CPT I, CPT II, Very Long Chain acyl-CoA Dehydrogenase Deficiency, VLCAD, Long Chain 3-hydroxy-acyl-CoA Dehydrogenase Deficiency
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- First posted
- 12 Dec 2018 (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.
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
Detailed description
Long Chain Fatty Acid Oxidation Disorders (LC-FAOD) and other Non-FAOD conditions, where applicable: Expanded access may be available outside of the US in countries prior to approval by the local regulatory agencies.
For full details, please visit the links provided below.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
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: UX007Liquid for oral (PO) or enteral tube administration | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2026.
- Access to Investigational Therapies / Requesting AccessLink provided by the sponsor.
- Dojolvi Patient and Prescribing Information PageLink provided by the sponsor.
Related trials for Long Chain Fatty Acid Oxidation Disorders
Active, not recruitingPhase 3
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
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Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
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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.
Early Access contact
Medical Director study director
Ultragenyx Pharmaceutical Inc
Questions about this trial
What is this clinical trial studying?
A study testing Triheptanoin in people with Long Chain Fatty Acid Oxidation Disorders, run by Ultragenyx Pharmaceutical Inc.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 31 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Early Access (EarlyAccess@ultragenyx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
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 31 Jul 2026.