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New hope for youngest kids with rare fat disorder

NCT ID NCT06502990

Terminated Disease control Sponsor: Amryt Pharma Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 23, 2026 · Updated 2 times

Summary

This study tests a drug called metreleptin in 15 children under 6 years old who have a rare disease called generalized lipodystrophy, which prevents the body from storing fat properly and often leads to diabetes and high triglycerides. The goal is to see if the drug can lower blood fats and improve blood sugar control. All children will receive the drug, and researchers will monitor safety and effectiveness for 12 months.

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Conditions

The condition(s) this trial relates to.

generalized lipodystrophy Lipodystrophy, Congenital Generalized

As listed by the trial registrant

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

GENERALIZED LIPODYSTROPHY

Contacts and locations

Locations

  • Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Universitaire Robert-Debre

    Paris, France

  • Azienda Ospedaliera Universitaria "Federico II"

    Naples, Naples, 80131, Italy

  • Azienda Ospedaliero Universitaria Maggiore della Carità di Novara

    Novara, Novara, 28100, Italy

  • Azienda Ospedaliero Universitaria Pisana

    Pisa, Pisa, 56124, Italy

  • Azienda Ospedaliero-Universitaria di Parma

    Parma, Parma, 43126, Italy

  • Hôpital Necker - Enfants Malades

    Paris, Paris, 75015, France

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Chieti, Chieti, 66100, Italy

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Rome, Rome, 00165, Italy

  • Ospedale Filippo Del Ponte Varese - ASST Sette Laghi

    Varese, Varese, 21100, Italy

  • UZ Leuven

    Leuven, Leuven, 3000, Belgium

  • Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

    Ulm, Ulm, 89075, Germany

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg, Free and Hanseatic City of Hamburg, 20251, Germany

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