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

NCT06502990 | Terminated Phase 3 DMC

• 2 other identifiers: APL-202022-501781-22-00
• Study Type: interventional
• Target: 1
• Locations: 4 countries
• Sites: 12

Brief 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

Conditions

Generalized Lipodystrophy

Keywords

Metreleptin

Outcome Measures

Primary Outcomes (2)

• 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

  To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia

  12 months

• Absolute change from baseline in glycated haemoglobin (HbA1c) at Month 12 for subjects with HbA1c ≥6.5% at baseline

  To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia

  12 months

Secondary Outcomes (6)

• 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

• 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

• 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

• 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

• Change from baseline in liver volume and liver span as assessed by ultrasound at each post-baseline visit through Month 12

  12 months

Study Arms (1)

Metreleptin

EXPERIMENTAL

Metreleptin \[Recombinant-methionyl human Leptin; rmetHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake

Drug: Metreleptin

Interventions

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

Metreleptin

Eligibility Criteria

• Age: Up to 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Confirmed diagnosis of Generalised Lipodystrophy
• Metreleptin treatment naive

You may not qualify if:

• Weight \<9 kg at Screening (Visit 1)

Sponsors & Collaborators

• Amryt Pharma: lead

Study Sites (12)

UZ Leuven

Leuven, Leuven, 3000, Belgium

Location

Hôpital Necker - Enfants Malades

Paris, Paris, 75015, France

Location

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

Paris, France

Location

Universitätsklinikum Hamburg-Eppendorf

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

Location

Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

Ulm, Ulm, 89075, Germany

Location

IRCCS Ospedale Pediatrico Bambino Gesù

Chieti, Chieti, 66100, Italy

Location

Azienda Ospedaliera Universitaria "Federico II"

Naples, Naples, 80131, Italy

Location

Azienda Ospedaliero Universitaria Maggiore della Carità di Novara

Novara, Novara, 28100, Italy

Location

Azienda Ospedaliero-Universitaria di Parma

Parma, Parma, 43126, Italy

Location

Azienda Ospedaliero Universitaria Pisana

Pisa, Pisa, 56124, Italy

Location

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, Rome, 00165, Italy

Location

Ospedale Filippo Del Ponte Varese - ASST Sette Laghi

Varese, Varese, 21100, Italy

Location

MeSH Terms

Conditions

Lipodystrophy, Congenital Generalized

Interventions

metreleptin

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipodystrophy; Skin Diseases, Metabolic; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single Group Assignment

Study Record Dates

• First Submitted: July 9, 2024
• First Posted: July 16, 2024
• Study Start: May 23, 2025
• Primary Completion: February 11, 2026
• Study Completion: February 11, 2026
• Last Updated: July 22, 2026

Record last verified: 2026-07

Locations

BE (1); IT (7); FR (2); DE (2)