RecruitingPhase 3NCT06502990

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

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

Sponsor

Amryt Pharma

Enrollment

15 participants

Start Date

May 23, 2025

Study Type

INTERVENTIONAL

Conditions

Generalized Lipodystrophy

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

Max Age: 5 Years

Inclusion Criteria2

Confirmed diagnosis of Generalised Lipodystrophy
Metreleptin treatment naive

Exclusion Criteria1

Weight \<9 kg at Screening (Visit 1)

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Interventions

DRUGMetreleptin

Metreleptin is a recombinant human leptin analog that is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency

Locations(12)

UZ Leuven

Leuven, Belgium

Hôpital Necker - Enfants Malades

Paris, France

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

Paris, France

Universitätsklinikum Hamburg-Eppendorf

Hamburg, Germany

Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

Ulm, Germany

IRCCS Ospedale Pediatrico Bambino Gesù

Chieti, Italy

Azienda Ospedaliera Universitaria "Federico II"

Naples, Italy

Azienda Ospedaliero Universitaria Maggiore della Carità di Novara

Novara, Italy

Azienda Ospedaliero-Universitaria di Parma

Parma, Italy

Azienda Ospedaliero Universitaria Pisana

Pisa, Italy

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, Italy

Ospedale Filippo Del Ponte Varese - ASST Sette Laghi

Varese, Italy

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NCT06502990

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