Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

Phase 4RecruitingNCT06484868

Amryt Pharma12 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Partial Lipodystrophy

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

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Confirmed diagnosis of familial or acquired partial lipodystrophy Exclusion Criteria: * 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. Other protocol defined inclusion/exclusion criteria apply

Locations (12)

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

Lille, France

RECRUITING

Hopital Pitie-Salpetriere

Paris, France

Outcomes

Primary Outcomes

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

Time frame: 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

Time frame: 12 months

Secondary Outcomes

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%.

To evaluate the long-term efficacy of metreleptin treatment in patients with PL

Time frame: 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).

To evaluate the long-term efficacy of metreleptin treatment in patients with PL

Time frame: 24 months

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

To assess changes in liver volume.

Time frame: 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

To evaluate the safety of metreleptin treatment in patients with PL

Time frame: 24 months

Central Contacts

Janet Boylan

CONTACT

+3905212791 clinicaltrials_info@chiesi.com

Data from ClinicalTrials.gov

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