NCT06484868

Brief Summary

This is an Open Label, Phase IV, Post Authorisation Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in people with Partial Lipodystrophy

Trial Health

80
On Track

Trial Health Score

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

Enrollment
12

participants targeted

Target at below P25 for phase_4

Timeline
21mo left

Started Feb 2024

Longer than P75 for phase_4

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 Progress56%
Feb 2024Feb 2028

Study Start

First participant enrolled

February 8, 2024

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

June 26, 2024

Completed
7 days until next milestone

First Posted

Study publicly available on registry

July 3, 2024

Completed
3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2028

Last Updated

July 3, 2025

Status Verified

July 1, 2025

Enrollment Period

4 years

First QC Date

June 26, 2024

Last Update Submit

July 2, 2025

Conditions

Partial Lipodystrophy

Keywords

Metreleptin

Outcome Measures

Primary Outcomes (2)

  • Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%.

    To evaluate the efficacy (HbA1c) of metreleptin treatment in patients with PL

    12 months

  • Number of patients with decrease of at least 30% in triglycerides (TG) at Month 12 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L)

    To evaluate the efficacy (TG) of metreleptin treatment in patients with PL

    12 months

Secondary Outcomes (4)

  • Number of patients with decrease of at least 0.5% in HbA1c at Month 24 compared to Baseline or HbA1c <6.5 % at Month 24, in patients with baseline HbA1c ≥6.5%.

    24 months

  • Number of patients with decrease of at least 30% in TG levels at Month 24 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L).

    24 months

  • Change from baseline in liver volume at Month 12 and Month 24

    12 months and 24 months

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

    24 months

Study Arms (1)

Metreleptin

EXPERIMENTAL

Metreleptin \[Recombinant-methionyl human Leptin; r-metHuLeptin\] 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

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Confirmed diagnosis of familial or acquired partial lipodystrophy

You may not qualify if:

  • Treatment with any Investigational Medicinal Product (IMP) within 6 months or 5 times the terminal half-life of the corresponding IMP, whichever is longer, before the screening visit.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

Centre Hospitalier Régional Universitaire de Lille (CHRU) - Hôpital Claude Huriez

Lille, France

RECRUITING

Hopital Pitie-Salpetriere

Paris, France

RECRUITING

Hôpital Saint-Antoine

Paris, France

RECRUITING

Centre Hospitalier Lyon-Sud

Pierre-Bénite, France

RECRUITING

Charite-Universitaetsmedizin Berlin - Campus Charite Mitte (CCM)

Berlin, Germany

RECRUITING

Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

Ulm, Germany

NOT YET RECRUITING

Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi

Bologna, Italy

RECRUITING

Universitá degli Studi "Magna Graecia" di Catanzaro

Catanzaro, Italy

NOT YET RECRUITING

Azienda Ospedaliero Universitaria Maggiore della Carità di Novara

Novara, Italy

RECRUITING

Azienda Ospedaliero Universitaria Pisana - Stabilimento di Cisanello

Pisa, Italy

RECRUITING

A.S.U Integrata di Udine - Presidio Ospedaliero Santa Maria della Misericordia

Udine, Italy

RECRUITING

Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital

Cambridge, United Kingdom

NOT YET RECRUITING

MeSH Terms

Conditions

Lipodystrophy, Partial, Acquired

Interventions

metreleptin

Central Study Contacts

Janet Boylan

CONTACT

+3905212791clinicaltrials_info@chiesi.com

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 26, 2024

First Posted

July 3, 2024

Study Start

February 8, 2024

Primary Completion (Estimated)

February 1, 2028

Study Completion (Estimated)

February 1, 2028

Last Updated

July 3, 2025

Record last verified: 2025-07

Locations

GB(1)DE(2)IT(5)FR(4)