New drug aims to cut life-threatening events in kids with rare metabolic disease
NCT ID NCT05933200
First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 38 times
Summary
This study tests whether triheptanoin, a special fat-based medicine, works better than standard MCT oil at preventing major health crises in children with long-chain fatty acid oxidation disorders (LC-FAOD). These rare genetic conditions prevent the body from breaking down certain fats for energy, leading to serious events like low blood sugar and heart problems. About 69 children under 18 are participating in this phase 3 trial, which compares the two treatments head-to-head.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Cerrahpasa Medical Faculty
Istanbul, 34116, Turkey (Türkiye)
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Cukurova University
Adana, Turkey (Türkiye)
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Ege University
Bornova-İzmir, Turkey (Türkiye)
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Gazi University
Ankara, Turkey (Türkiye)
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Gdańksi Uniwersytet Medyczny
Gdansk, Pomeranian Voivodeship, 80-952, Poland
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General University Hospital in Prague-GUH (Všeobecná fakultní nemocnice v Praze- VFN)
Prague, 120-08, Czechia
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Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, Masovian Voivodeship, 04-730, Poland
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Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
Istanbul, 34093, Turkey (Türkiye)
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Juntendo University Hospital
Bunkyo City, Tokyo, 113-8431, Japan
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King Faisal Specialist Hospital & Research Centre
Riyadh, 11211, Saudi Arabia
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Sant Joan de Deu Hospital (SJD)
Barcelona, Esplugues de Llobregat, 08950, Spain
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The Jikei University Hospital
Minato, Tokyo, Japan
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University Hospital 12 de Octubre
Madrid, 28041, Spain
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University Hospital Santiago de Compostela
A Coruña, 15706, Spain
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Universitätsklinikum Freiburg
Freiburg im Breisgau, 79106, Germany
Conditions
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