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

NCT ID: NCT06502990

Last Updated: 2026-01-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-05-23

Study Completion Date

2026-09-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Generalized Lipodystrophy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Single Group Assignment
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Metreleptin

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

Group Type EXPERIMENTAL

Metreleptin

Intervention Type 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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Metreleptin

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

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Confirmed diagnosis of Generalised Lipodystrophy
* Metreleptin treatment naive

Exclusion Criteria

* Weight \<9 kg at Screening (Visit 1)
Maximum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Amryt Pharma

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

UZ Leuven

Leuven, , Belgium

Site Status RECRUITING

Hôpital Necker - Enfants Malades

Paris, , France

Site Status RECRUITING

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

Paris, , France

Site Status RECRUITING

Universitätsklinikum Hamburg-Eppendorf

Hamburg, , Germany

Site Status NOT_YET_RECRUITING

Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

Ulm, , Germany

Site Status RECRUITING

IRCCS Ospedale Pediatrico Bambino Gesù

Chieti, , Italy

Site Status RECRUITING

Azienda Ospedaliera Universitaria "Federico II"

Naples, , Italy

Site Status RECRUITING

Azienda Ospedaliero Universitaria Maggiore della Carità di Novara

Novara, , Italy

Site Status RECRUITING

Azienda Ospedaliero-Universitaria di Parma

Parma, , Italy

Site Status RECRUITING

Azienda Ospedaliero Universitaria Pisana

Pisa, , Italy

Site Status RECRUITING

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, , Italy

Site Status RECRUITING

Ospedale Filippo Del Ponte Varese - ASST Sette Laghi

Varese, , Italy

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

Belgium France Germany Italy

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Janet Boylan

Role: CONTACT

+3905212791

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Kristina Casteels

Role: primary

Jacques Beltrand

Role: primary

Elise Bismuth

Role: primary

Konstantinos Tsiakas

Role: primary

Martin Wabitsch

Role: primary

Stefano Tumini

Role: primary

Enza Mozzillo

Role: primary

Flavia Prodam

Role: primary

Maria Street

Role: primary

Giovanni Ceccarini

Role: primary

Marco Cappa

Role: primary

Gianluca Musolino

Role: primary

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2022-501781-22-00

Identifier Type: CTIS

Identifier Source: secondary_id

APL-20

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Leptin to Treat Lipodystrophy
NCT00025883 COMPLETED PHASE2
Leptin to Treat Lipodystrophy
NCT00005905 COMPLETED PHASE2
Triheptanoin in Mc Ardle
NCT02919631 COMPLETED PHASE2