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

Amryt Pharma

Status and phase: Enrolling; Phase 3

Conditions: Generalized Lipodystrophy

Treatments: Drug: Metreleptin

Study type: Interventional

Funder types: Industry

Identifiers

NCT06502990

APL-20

2022-501781-22-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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

Enrollment: 15 estimated patients

Sex: All

Ages: Under 5 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed diagnosis of Generalised Lipodystrophy
• Metreleptin treatment naive

Exclusion criteria

• Weight <9 kg at Screening (Visit 1)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

Metreleptin

Experimental group

Description:

Metreleptin \[Recombinant-methionyl human Leptin; rmetHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake

Treatment:

Drug: Metreleptin