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Study on the Effects of Metreleptin for Patients with Partial Lipodystrophy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Partial Lipodystrophy. This is a rare disorder where the body is unable to store fat properly, leading to a lack of fat tissue in certain areas. The treatment being tested in this study is called Metreleptin, which is a medication given as a daily injection under the skin. The purpose of the study is to evaluate how effective and safe metreleptin is for patients with partial lipodystrophy.

Participants in the study will receive metreleptin for a period of 24 months. During this time, researchers will monitor the effects of the treatment on the participants' health, focusing on changes in blood sugar levels and fat levels in the blood. The study will also look at how the body responds to the treatment over time, including any potential immune reactions. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of metreleptin.

The study aims to gather information on how metreleptin can help manage symptoms of partial lipodystrophy, such as high blood sugar and high levels of fats in the blood. By the end of the study, researchers hope to better understand the benefits and any risks associated with metreleptin treatment for this condition. Participants will be closely monitored throughout the study to ensure their safety and well-being.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initiation of treatment
Upon joining the study, the treatment with metreleptin begins. This medication is administered as a subcutaneous injection, which means it is injected under the skin.
The available formulations for this medication include Myalepta 5.8 mg, 11.3 mg, and 3 mg powder for solution for injection. The specific dosage and formulation will be determined by the healthcare provider based on individual needs.
Step 2
Daily administration
The treatment involves a daily injection of metreleptin. It is important to follow the prescribed dosage and administration schedule as instructed by the healthcare provider.
The duration of this treatment phase is 24 months, during which the effects of the medication will be monitored.
Step 3
Monitoring and assessments
Throughout the study, regular assessments will be conducted to evaluate the efficacy, safety, and immunogenicity of the treatment.
Key assessments include measuring changes in glycated hemoglobin (HbA1c) and triglyceride (TG) levels at various intervals, specifically at Month 12 and Month 24.
Step 4
Evaluation of treatment outcomes
The primary goal is to observe a decrease in HbA1c and TG levels. A successful outcome is defined as a decrease of at least 0.5% in HbA1c or achieving HbA1c <6.5% at Month 12, and a 30% decrease in TG levels at Month 12.
Secondary outcomes include further reductions in these levels at Month 24 and other health indicators such as liver volume and insulin resistance.
Step 5
Completion of the study
The study is expected to conclude by September 2027. At the end of the study, a comprehensive evaluation of the treatment's impact on health parameters will be conducted.
Participants will receive guidance on the next steps based on the study's findings and their individual health status.

Who can join the trial?

10 criteria

Patients must be male or female and at least 12 years old.
The patient or their legal representative must understand and agree to the study requirements and sign a consent form. If the patient is a child, the study must be explained to them.
The patient must have a confirmed diagnosis of **Partial Lipodystrophy (PL)**, which is a condition where the body has trouble storing fat.
The doctor must confirm that other similar conditions have been ruled out, such as Cushing's syndrome or anorexia nervosa.
Patients must have either a **HbA1c level** of 6.5% or higher, which is a measure of blood sugar levels over time, or **fasting serum triglyceride (TG) levels** of 500 mg/dL or higher, which is a measure of fat in the blood.
Standard treatments must have failed to control the patient's condition. For those with high HbA1c, they must have been on a stable dose of diabetes medication for at least 90 days. For those with high TG levels, they must have been on a stable dose of fat-lowering medication for at least 6 weeks.
Patients must continue their current diabetes or fat-lowering treatments without changing the dose significantly.
Patients must be willing to follow dietary recommendations given by the doctor.
Patients must not have used metreleptin before or must have started using it within 6 months before the study. If already using metreleptin, they must have certain medical records available.
Females who can have children must either be unable to have children, be using effective birth control, or be willing to use effective birth control during the study and for 4 weeks after the last dose of the study drug. Hormonal birth control alone is not enough; an additional method must be used.

Who cannot join the trial?

6 criteria

Patients who have a different condition than Partial Lipodystrophy cannot participate. Partial Lipodystrophy is a condition where the body has an abnormal distribution of fat.
Patients who are not within the specified age range cannot participate. The age range includes children, teenagers, and adults.
Patients who are not willing to follow the study procedures or take the study medication cannot participate.
Patients who have other serious health conditions that might interfere with the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who are currently participating in another clinical trial cannot participate.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Universitaet Leipzig
Leipzig, Germany
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Azienda Ospedaliero-Universitaria Maggiore Della Carita
Novara, Italy
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Azienda Sanitaria Universitaria Friuli Centrale
Udine, Italy

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Investigated drugs

Metreleptin is a medication used in this clinical trial to treat patients with partial lipodystrophy. It is administered daily through a subcutaneous injection. The purpose of using metreleptin in this study is to assess its effectiveness, safety, and the body's immune response to the treatment. Metreleptin is designed to help manage the symptoms associated with partial lipodystrophy by supplementing leptin, a hormone that helps regulate energy balance and fat storage in the body.

Metreleptin

What is already known about the treatment

Metreleptin – Metreleptin is administered through daily subcutaneous injections. It is currently being studied in a Phase IV clinical trial to assess its efficacy, safety, and immunogenicity for treating partial lipodystrophy. This medication is primarily indicated for patients with this condition, which involves abnormal fat distribution and metabolic complications. Metreleptin works by mimicking the hormone leptin, which helps regulate energy balance and metabolism at the molecular level. It is classified as a hormone replacement therapy.

Investigated diseases

Partial Lipodystrophy – Partial Lipodystrophy is a rare disorder characterized by the abnormal distribution of body fat. In this condition, individuals lose fat from certain areas of the body while it may accumulate in others. This uneven fat distribution can lead to metabolic complications, such as insulin resistance and high levels of triglycerides in the blood. The progression of the disease can vary, with some individuals experiencing more severe metabolic issues than others. It is often associated with other health problems, including diabetes and liver abnormalities. The condition can be genetic or acquired, and its symptoms may become more pronounced over time.