Brief Title: Open-label Study to Evaluate Metreleptin in Patients with Partial Lipodystrophy
Research type
Research Study
Full title
A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients with Partial Lipodystrophy
IRAS ID
1008177
Contact name
Laura Maher
Contact email
Sponsor organisation
Amryt Pharma DAC
Research summary
Metreleptin is an artificially made human protein called leptin. It is approved to replace leptin (a hormone released by fat tissue cells), for the treatment of leptin deficiency in adults and children with lipodystrophy (LD) in combination with dietary modification. LD is caused by a loss or absence of fat tissue under the skin, resulting in low leptin levels. Metreleptin is an approved drug, studied in adults and children with LD for over 14 years. This post-marketing (after treatment has been approved), open-label (with the known treatment) study will provide further information on the effectiveness, safety and immunogenicity (the ability to cause an immune response) of metreleptin treatment in patients 12 years or older with partial lipodystrophy (PL). Approximately 12 patients with PL will take part in this study.
The study design reflects metreleptin use in routine standard of care, therefore it includes visits/procedures in line with clinical practice. Metreleptin will be injected daily under the skin during the 24-month study treatment period and may be continued after the study ends as routine clinical care. The recommended doses are based on sex and body weight. The doctor can modify the dose based on clinical response and/or other considerations such as side effects. Known common side effects include low blood sugar levels and weight decrease. There may also be discomfort and reactions from daily injections. There have been some rare cases of cancer (T-cell lymphoma) in patients treated with metreleptin, however it is not known if metreleptin is the cause of the lymphoma or if there is another cause.
The main objective of this study is to assess the possible effect of metreleptin on improving triglyceride and blood sugar levels. Additional efficacy and safety measures will also be assessed as part of this study.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
23/NW/0263
Date of REC Opinion
2 Oct 2023
REC opinion
Further Information Favourable Opinion