A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
NCT ID: NCT05933200
Last Updated: 2026-01-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
69 participants
INTERVENTIONAL
2023-02-28
2027-08-31
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Triheptanoin
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.
Triheptanoin
Liquid for oral (PO) or enteral feeding tube administration
MCT
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.
MCT Oil
Liquid for oral (PO) or enteral feeding tube administration
Interventions
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MCT Oil
Liquid for oral (PO) or enteral feeding tube administration
Triheptanoin
Liquid for oral (PO) or enteral feeding tube administration
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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)
* 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
Exclusion Criteria
* 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.
* 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
17 Years
ALL
No
Sponsors
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Ultragenyx Pharmaceutical Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Ultragenyx Pharmaceutical Inc
Locations
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General University Hospital in Prague-GUH (Všeobecná fakultní nemocnice v Praze- VFN)
Prague, , Czechia
Universitätsklinikum Freiburg
Freiburg im Breisgau, , Germany
Juntendo University Hospital
Bunkyo City, Tokyo, Japan
The Jikei University Hospital
Minato, Tokyo, Japan
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, Masovian Voivodeship, Poland
Gdańksi Uniwersytet Medyczny
Gdansk, Pomeranian Voivodeship, Poland
King Faisal Specialist Hospital & Research Centre
Riyadh, , Saudi Arabia
Sant Joan de Deu Hospital (SJD)
Barcelona, Esplugues de Llobregat, Spain
University Hospital Santiago de Compostela
A Coruña, , Spain
University Hospital 12 de Octubre
Madrid, , Spain
Cukurova University
Adana, , Turkey (Türkiye)
Gazi University
Ankara, , Turkey (Türkiye)
Ege University
Bornova-İzmir, , Turkey (Türkiye)
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
Istanbul, , Turkey (Türkiye)
Cerrahpasa Medical Faculty
Istanbul, , Turkey (Türkiye)
Countries
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Related Links
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Ultragenyx Patient Advocacy/LC-FAOD Disease Information
Ultragenyx Transparency Commitment
Other Identifiers
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2022-001539-10
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-509809-76-00
Identifier Type: CTIS
Identifier Source: secondary_id
UX007-CL302
Identifier Type: -
Identifier Source: org_study_id
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