Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy

Last updated: January 16, 2026
Sponsor: Amryt Pharma
Overall Status: Active - Recruiting

Phase

3

Condition

Skin Lipomas

Treatment

Metreleptin

Clinical Study ID

NCT06502990
APL-20
2022-501781-22-00
  • Ages < 5
  • All Genders

Study Summary

This is an open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Confirmed diagnosis of Generalised Lipodystrophy

  • Metreleptin treatment naive

Exclusion

Exclusion Criteria:

  • Weight <9 kg at Screening (Visit 1)

Study Design

Total Participants: 15
Treatment Group(s): 1
Primary Treatment: Metreleptin
Phase: 3
Study Start date:
May 23, 2025
Estimated Completion Date:
September 30, 2026

Connect with a study center

  • UZ Leuven

    Leuven, 3000
    Belgium

    Site Not Available

  • UZ Leuven

    Leuven 2792482, 3000
    Belgium

    Active - Recruiting

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

    Paris,
    France

    Site Not Available

  • Hôpital Necker - Enfants Malades

    Paris, 75015
    France

    Site Not Available

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

    Paris 2988507,
    France

    Active - Recruiting

  • Hôpital Necker - Enfants Malades

    Paris 2988507, 75015
    France

    Active - Recruiting

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg, 20251
    Germany

    Site Not Available

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg 2911298, 20251
    Germany

    Site Not Available

  • Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

    Ulm, 89075
    Germany

    Site Not Available

  • Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

    Ulm 2820256, 89075
    Germany

    Active - Recruiting

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Chieti, 66100
    Italy

    Site Not Available

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Chieti 3178796, 66100
    Italy

    Active - Recruiting

  • Azienda Ospedaliera Universitaria "Federico II"

    Naples, 80131
    Italy

    Site Not Available

  • Azienda Ospedaliera Universitaria "Federico II"

    Naples 3172394, 80131
    Italy

    Active - Recruiting

  • Azienda Ospedaliero Universitaria Maggiore della Carità di Novara

    Novara, 28100
    Italy

    Site Not Available

  • Azienda Ospedaliero Universitaria Maggiore della Carità di Novara

    Novara 3172189, 28100
    Italy

    Active - Recruiting

  • Azienda Ospedaliero-Universitaria di Parma

    Parma, 43126
    Italy

    Site Not Available

  • Azienda Ospedaliero-Universitaria di Parma

    Parma 3171457, 43126
    Italy

    Active - Recruiting

  • Azienda Ospedaliero Universitaria Pisana

    Pisa, 56124
    Italy

    Site Not Available

  • Azienda Ospedaliero Universitaria Pisana

    Pisa 3170647, 56124
    Italy

    Active - Recruiting

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Rome, 00165
    Italy

    Site Not Available

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Rome 3169070, 00165
    Italy

    Active - Recruiting

  • Ospedale Filippo Del Ponte Varese - ASST Sette Laghi

    Varese, 21100
    Italy

    Site Not Available

  • Ospedale Filippo Del Ponte Varese - ASST Sette Laghi

    Varese 3164699, 21100
    Italy

    Active - Recruiting

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