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

NCT06502990 — Phase 3, Terminated, 1 participants, Amryt Pharma

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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

Registry	NCT06502990
Status	Terminated - This clinical trial was closed early due to no longer being a requirement to the European Medicines Agency.
Phase	Phase 3
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	1 (Actual)
Sponsor	Amryt Pharma (Industry)
Conditions	Generalized Lipodystrophy
Interventions	Metreleptin (Drug)
Dates	start 2025-05-23, primary completion 2026-02-11, completion 2026-02-11, first posted 2024-07-16, last updated 2026-07-22
Locations	12 sites in Belgium, France, Germany, Italy

-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

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+Arms and interventions

Arm	Type	Interventions	Description
Metreleptin	Experimental	Drug: Metreleptin	Metreleptin \[Recombinant-methionyl human Leptin; rmetHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake

Metreleptin (Drug): 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

+Outcome measures

Primary outcomes

Percent change from baseline in fasting serum TG levels at Month 12 for subjects with fasting TG levels ≥2.3 mmol/L (200 mg/dL) at baseline [12 months]: To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia
Absolute change from baseline in glycated haemoglobin (HbA1c) at Month 12 for subjects with HbA1c ≥6.5% at baseline [12 months]: To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia

Secondary outcomes

Proportion of subjects of those with Baseline HbA1c ≥6.5% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <6.5% or HbA1c <5.7% at Month 12 [12 months]: To evaluate the efficacy of metreleptin treatment in patients with GL
Proportion of subjects of those with Baseline HbA1c ≥5.7% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <5.7% at Month 12 [12 months]: To evaluate the efficacy of metreleptin treatment in patients with GL
Proportion of subjects of those with fasting serum TG ≥ 1.7 mmol/L (150 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12 [12 months]: To evaluate the efficacy of metreleptin treatment in patients with GL
Proportion of subjects of those with fasting serum TG ≥ 2.3 mmol/L (200 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12 [12 months]: To evaluate the efficacy of metreleptin treatment in patients with GL
Change from baseline in liver volume and liver span as assessed by ultrasound at each post-baseline visit through Month 12 [12 months]: To evaluate the efficacy of metreleptin treatment in patients with GL
Incidence of, treatment emergent adverse events (TEAEs), serious adverse events (SAEs), treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation [12 months]: To assess safety and tolerability of metreleptin

+Eligibility

Sex: All, ages: any to 5 Years

Inclusion Criteria:

* Confirmed diagnosis of Generalised Lipodystrophy
* Metreleptin treatment naive

Exclusion Criteria:

* Weight <9 kg at Screening (Visit 1)

+Locations (12)

Facility	City	Country
UZ Leuven	Leuven, Leuven	Belgium
Hôpital Necker - Enfants Malades	Paris, Paris	France
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Universitaire Robert-Debre	Paris	France
Universitätsklinikum Hamburg-Eppendorf	Hamburg, Free and Hanseatic City of Hamburg	Germany
Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin	Ulm, Ulm	Germany
IRCCS Ospedale Pediatrico Bambino Gesù	Chieti, Chieti	Italy
Azienda Ospedaliera Universitaria "Federico II"	Naples, Naples	Italy
Azienda Ospedaliero Universitaria Maggiore della Carità di Novara	Novara, Novara	Italy
Azienda Ospedaliero-Universitaria di Parma	Parma, Parma	Italy
Azienda Ospedaliero Universitaria Pisana	Pisa, Pisa	Italy
IRCCS Ospedale Pediatrico Bambino Gesù	Rome, Rome	Italy
Ospedale Filippo Del Ponte Varese - ASST Sette Laghi	Varese, Varese	Italy