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New hope for youngest kids with rare fat disorder

NCT ID NCT06502990

Disease control Sponsor: Amryt Pharma Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 23, 2026 · Updated 2 times

Summary

This study tests a drug called metreleptin in 15 children under 6 years old who have a rare disease called generalized lipodystrophy, which prevents the body from storing fat properly and often leads to diabetes and high triglycerides. The goal is to see if the drug can lower blood fats and improve blood sugar control. All children will receive the drug, and researchers will monitor safety and effectiveness for 12 months.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

1 person

The number who actually took part.

Started

May 2025

Finished

Feb 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 5 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Confirmed diagnosis of Generalised Lipodystrophy * Metreleptin treatment naive Exclusion Criteria: * Weight \<9 kg at Screening (Visit 1)

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Conditions

The condition(s) this trial relates to.

generalized lipodystrophy Lipodystrophy, Congenital Generalized

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Generalized lipodystrophy

Contacts and locations

Locations

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

    Paris, France

  • Azienda Ospedaliera Universitaria "Federico II"

    Naples, Naples, 80131, Italy

  • Azienda Ospedaliero Universitaria Maggiore della Carità di Novara

    Novara, Novara, 28100, Italy

  • Azienda Ospedaliero Universitaria Pisana

    Pisa, Pisa, 56124, Italy

  • Azienda Ospedaliero-Universitaria di Parma

    Parma, Parma, 43126, Italy

  • Hôpital Necker - Enfants Malades

    Paris, Paris, 75015, France

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Chieti, Chieti, 66100, Italy

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Rome, Rome, 00165, Italy

  • Ospedale Filippo Del Ponte Varese - ASST Sette Laghi

    Varese, Varese, 21100, Italy

  • UZ Leuven

    Leuven, Leuven, 3000, Belgium

  • Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

    Ulm, Ulm, 89075, Germany

  • Universitätsklinikum Hamburg-Eppendorf

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

More trials for these conditions

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