A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
- Conditions
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
- Interventions
- Dietary Supplement: MCT Oil
- Registration Number
- NCT05933200
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Brief 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).
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 60
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Triheptanoin 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. MCT MCT Oil 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.
- Primary Outcome Measures
Name Time Method Annualized Event Rate of Major Clinical Events (MCEs) Up to Year 4
- Secondary Outcome Measures
Name Time Method Annualized Hospitalization Days Up to Year 4 Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, and Adverse Events of Special Interest (AESIs) 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 Up to Year 4 Plasma Concentration Levels of Heptanoate Up to Year 1 Acceptability and Palatability Survey Scores of Triheptanoin Mixed with Oral Liquids Up to Year 1 Change From Baseline in Left Ventricular Ejection Fraction Baseline, Up to Year 1 Change From Baseline in Left Ventricular Wall Mass Baseline, Up to Year 1 Annualized Duration of Rhabdomyolysis-MCEs, Cardiomyopathy-MCEs, and Hypoglycemic-MCEs Up to Year 4 Annualized Hypoglycemic Event-rate Captured as MCEs and At-home Clinical Events (HCEs) Up to Year 4 Annualized Duration of MCEs Up to Year 4 Clinical Global Impression of Change [CGI-C] Scale Score Up to Year 4 Change From Baseline in Left Ventricular Systolic Volume Baseline, Up to Year 1 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 Up to Year 4 Survival Time Up to Year 4 Number of Missed School or Learning Opportunity Days Up to Year 4 Annualized Frequency of Rhabdomyolysis-MCEs and Cardiomyopathy-MCEs Up to Year 4 Change From Baseline in PedsQL Infant Scale Score for Participants Ages 1 to <24 Months Up to Year 4 Plasma Concentration Levels of Beta Hydroxypentanoate (BHP) 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) Baseline, Month 6
Trial Locations
- Locations (16)
General University Hospital in Prague-GUH (Všeobecná fakultní nemocnice v Praze- VFN)
🇨🇿Prague, Czechia
Universitätsklinikum Freiburg
🇩🇪Freiburg, Germany
Juntendo University Hospital
🇯🇵Bunkyo City, Tokyo, Japan
The Jikei University Hospital
🇯🇵Minato City, Tokyo, Japan
Instytut Pomnik-Centrum Zdrowia Dziecka
🇵🇱Warszawa, Mazowieckie, Poland
Gdańksi Uniwersytet Medyczny
🇵🇱Gdańsk, Pomorskie, 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
Gazi University
🇹🇷Ankara, Turkey
Ege University
🇹🇷Bornova-İzmir, Turkey
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
🇹🇷Istanbul, Turkey
Cerrahpasa Medical Faculty
🇹🇷Istanbul, Turkey
Great Ormond Street Hospital
🇬🇧London, United Kingdom