NCT06502990

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

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_3

Timeline
5mo left

Started May 2025

Geographic Reach
4 countries

12 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress71%
May 2025Sep 2026

First Submitted

Initial submission to the registry

July 9, 2024

Completed
7 days until next milestone

First Posted

Study publicly available on registry

July 16, 2024

Completed
10 months until next milestone

Study Start

First participant enrolled

May 23, 2025

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2026

Last Updated

January 20, 2026

Status Verified

January 1, 2026

Enrollment Period

1.4 years

First QC Date

July 9, 2024

Last Update Submit

January 16, 2026

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

  • +1 more secondary outcomes

Study Arms (1)

Metreleptin

EXPERIMENTAL

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

Drug: Metreleptin

Interventions

MetreleptinDRUG

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

AgeUp to 5 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

UZ Leuven

Leuven, 3000, Belgium

RECRUITING

Hôpital Necker - Enfants Malades

Paris, 75015, France

RECRUITING

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

Paris, France

RECRUITING

Universitätsklinikum Hamburg-Eppendorf

Hamburg, 20251, Germany

NOT YET RECRUITING

Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

Ulm, 89075, Germany

RECRUITING

IRCCS Ospedale Pediatrico Bambino Gesù

Chieti, 66100, Italy

RECRUITING

Azienda Ospedaliera Universitaria "Federico II"

Naples, 80131, Italy

RECRUITING

Azienda Ospedaliero Universitaria Maggiore della Carità di Novara

Novara, 28100, Italy

RECRUITING

Azienda Ospedaliero-Universitaria di Parma

Parma, 43126, Italy

RECRUITING

Azienda Ospedaliero Universitaria Pisana

Pisa, 56124, Italy

RECRUITING

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, 00165, Italy

RECRUITING

Ospedale Filippo Del Ponte Varese - ASST Sette Laghi

Varese, 21100, Italy

RECRUITING

MeSH Terms

Conditions

Lipodystrophy, Congenital Generalized

Interventions

metreleptin

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipodystrophySkin Diseases, MetabolicSkin DiseasesSkin and Connective Tissue DiseasesLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Central Study Contacts

Janet Boylan

CONTACT

+3905212791clinicaltrials_info@chiesi.com

Study Design

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

Study Record Dates

First Submitted

July 9, 2024

First Posted

July 16, 2024

Study Start

May 23, 2025

Primary Completion (Estimated)

September 30, 2026

Study Completion (Estimated)

September 30, 2026

Last Updated

January 20, 2026

Record last verified: 2026-01

Locations

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