Metreleptin for Young Children with Generalized Lipodystrophy

We are studying whether metreleptin can help children under 6 years old with generalized lipodystrophy and related diabetes. The trial will also look at its effects on quality of life and other health markers.

1-2 years≤5 visitsConfirmation phase (III)No PlaceboPartially RemoteStandard MedicinesInjectionDermatologyEndocrinology

What diseases are being studied?

Health conditions and diseases that the clinical trial is designed to study and treat.

Who can participate in the clinical trial?

These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..

What medicines are being studied?

Medicines that are officially approved for use in the European Union and considered safe by EMA. These medicines are already on the market and may be tested for new conditions or populations.

Myalepta
Myalepta treats rare disorders that cause abnormal fat loss and helps control related problems like high blood sugar and high blood fats.

What active substances are being studied?

Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.

Metreleptin
Metreleptin is a lab-made leptin hormone replacement used in people with lipodystrophy to help control blood sugar and lower high blood fats.

Where is the clinical trial being conducted?

Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.

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UZ Leuven
Pediatrics
Heverlee, Belgium
Hôpital Robert Debré
Department of Pediatric Endocrinology and Diabetes
Le Pré-Saint-Gervais, France
Hôpital Universitaire Necker Enfants Malades - AP-HP
Endocrinology, Gynecology and Pediatric Diabetology
Montrouge, France
Sponsor: Amryt Pharmaceuticals Designated Activity Company
Last updated: Nov 26, 2025

Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.