New oil therapy could slash Life-Threatening events in kids with rare metabolic disease

NCT ID NCT05933200

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests whether triheptanoin, a special oil, can reduce major clinical events (like severe low blood sugar or heart problems) in children with long-chain fatty acid oxidation disorders (LC-FAOD), a rare condition where the body can't break down certain fats for energy. About 69 kids under 18 will receive either triheptanoin or a standard MCT oil, and researchers will track how often serious events occur over time. The goal is to see if triheptanoin offers better disease control than current treatments.

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Conditions

The condition(s) this trial relates to.

inherited fatty acid metabolism disorder VLCAD deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Cerrahpasa Medical Faculty

    Istanbul, 34116, Turkey (Türkiye)

  • Cukurova University

    Adana, Turkey (Türkiye)

  • Ege University

    Bornova-İzmir, Turkey (Türkiye)

  • Gazi University

    Ankara, Turkey (Türkiye)

  • Gdańksi Uniwersytet Medyczny

    Gdansk, Pomeranian Voivodeship, 80-952, Poland

  • General University Hospital in Prague-GUH (Všeobecná fakultní nemocnice v Praze- VFN)

    Prague, 120-08, Czechia

  • Instytut Pomnik-Centrum Zdrowia Dziecka

    Warsaw, Masovian Voivodeship, 04-730, Poland

  • Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

    Istanbul, 34093, Turkey (Türkiye)

  • Juntendo University Hospital

    Bunkyo City, Tokyo, 113-8431, Japan

  • King Faisal Specialist Hospital & Research Centre

    Riyadh, 11211, Saudi Arabia

  • Sant Joan de Deu Hospital (SJD)

    Barcelona, Esplugues de Llobregat, 08950, Spain

  • The Jikei University Hospital

    Minato, Tokyo, Japan

  • University Hospital 12 de Octubre

    Madrid, 28041, Spain

  • University Hospital Santiago de Compostela

    A Coruña, 15706, Spain

  • Universitätsklinikum Freiburg

    Freiburg im Breisgau, 79106, Germany