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NCT06502990·Phase 3·INTERVENTIONAL

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

MetreleptinOtherTerminated

Status

Terminated

Phase

Phase 3

Enrollment

1

Locations

12

Results

Not posted

Publications

0

Study summary

What the protocol is testing.

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

Interventions

Treatment arms and agents.

DRUG

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

Timeline

From registration to results.

  1. First posted

    Jul 16, 2024

  2. Study start

    May 23, 2025

  3. Primary completion

    Feb 11, 2026

  4. Study completion

    Feb 11, 2026

  5. Results posted

    Not reported

  6. Registry updated

    Jul 22, 2026

Outcomes

What the study measures.

Primary outcomes

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

Time frame · 12 months

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

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

Time frame · 12 months

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

Secondary outcomes

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

Time frame · 12 months

To evaluate the efficacy of metreleptin treatment in patients with GL

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

Time frame · 12 months

To evaluate the efficacy of metreleptin treatment in patients with GL

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

Time frame · 12 months

To evaluate the efficacy of metreleptin treatment in patients with GL

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

Time frame · 12 months

To evaluate the efficacy of metreleptin treatment in patients with GL

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

Time frame · 12 months

To evaluate the efficacy of metreleptin treatment in patients with GL

Incidence of, treatment emergent adverse events (TEAEs), serious adverse events (SAEs), treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation

Time frame · 12 months

To assess safety and tolerability of metreleptin

Eligibility

Who can take part.

Minimum age
Not reported
Maximum age
5 Years
Sex
ALL
Healthy volunteers
No

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

Study locations

12 registered sites.

Belgium · France · Germany · Italy. Showing up to 24 locations stored in the fast local snapshot.

UZ Leuven

Leuven, Leuven, Belgium

Hôpital Necker - Enfants Malades

Paris, Paris, France

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

Paris, France

Universitätsklinikum Hamburg-Eppendorf

Hamburg, Free and Hanseatic City of Hamburg, Germany

Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

Ulm, Ulm, Germany

IRCCS Ospedale Pediatrico Bambino Gesù

Chieti, Chieti, Italy

Azienda Ospedaliera Universitaria "Federico II"

Naples, Naples, Italy

Azienda Ospedaliero Universitaria Maggiore della Carità di Novara

Novara, Novara, Italy

Azienda Ospedaliero-Universitaria di Parma

Parma, Parma, Italy

Azienda Ospedaliero Universitaria Pisana

Pisa, Pisa, Italy

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, Rome, Italy

Ospedale Filippo Del Ponte Varese - ASST Sette Laghi

Varese, Varese, Italy

Publications

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

ClinicalTrials.gov ↗Search PubMed ↗Search FDA ↗Search EMA ↗

Related trials

More studies on Metreleptin.

Related PeptideStat pages

Put the record in context.

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TrialsMetreleptin trial dashboard →