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Study on Metreleptin for Children Under 6 with Generalized Lipodystrophy and Related Diabetes or High Triglycerides

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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 rare condition called generalized lipodystrophy, which affects the body's ability to store fat properly. This can lead to other health issues like diabetes mellitus and high levels of fats in the blood, known as hypertriglyceridemia. The study will use a treatment called metreleptin, which is a protein-based medication. Metreleptin is given as an injection under the skin and is being tested to see how effective and safe it is for children under 6 years old who have generalized lipodystrophy.

The purpose of the study is to evaluate how well metreleptin works in managing the symptoms of generalized lipodystrophy and its associated conditions. Participants in the study will receive metreleptin injections over a period of 12 months. Throughout the study, researchers will monitor changes in the participants' health, including their blood sugar levels and fat levels in the blood. The study aims to understand how metreleptin affects these health markers and whether it can help improve the quality of life for young children with this condition.

Participants will be closely observed by healthcare professionals to ensure their safety and to gather important information about how metreleptin works in their bodies. The study is open-label, meaning that both the participants and the researchers know that metreleptin is being administered. This trial is an important step in finding better treatments for children with generalized lipodystrophy and related health issues.

The research process

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

  1. Step 1

    Enrollment and baseline visit

    Upon joining the study, the first step is the enrollment and baseline visit. During this visit, the child's eligibility for the study is confirmed. This involves reviewing the child's medical history and conducting necessary tests to ensure they meet the study criteria.

    The child's current health status is assessed, including measurements of fasting triglycerides and glycated hemoglobin (HbA1c) levels. These measurements help establish a baseline for future comparisons.

  2. Step 2

    Initiation of metreleptin treatment

    After the baseline visit, the child begins treatment with metreleptin. Metreleptin is administered as a subcutaneous injection, which means it is injected under the skin.

    The dosage and frequency of the metreleptin injections are determined by the study protocol and the child's specific needs. The treatment continues throughout the study duration, which is estimated to last until September 2026.

  3. Step 3

    Regular follow-up visits

    The child will attend regular follow-up visits to monitor their response to the treatment. These visits are scheduled at specific intervals as outlined in the study protocol.

    During these visits, the child's health is assessed, and blood samples may be taken to measure triglyceride and HbA1c levels. The child's overall well-being and any side effects experienced are also evaluated.

  4. Step 4

    Mid-study assessments

    At the midpoint of the study, additional assessments are conducted to evaluate the child's progress. This includes measuring changes in fasting triglycerides and HbA1c levels compared to the baseline.

    The child's liver health may also be assessed using ultrasound, and any changes in liver size or function are documented.

  5. Step 5

    Final study visit

    At the end of the study period, a final study visit is conducted. This visit includes a comprehensive evaluation of the child's health and the effectiveness of the metreleptin treatment.

    Final measurements of triglyceride and HbA1c levels are taken, and the child's overall experience with the treatment is reviewed.

Who can join the trial?

11 criteria

  • Children who are younger than 6 years old at the time of signing the consent form and at the start of the study.
  • Children who have never been treated with metreleptin before.
  • Children with a confirmed diagnosis of generalised lipodystrophy (a condition where the body has very little fat) through one of the following:
    • A genetic test showing mutations related to the condition.
    • Imaging tests like X-rays or MRIs showing almost no body fat.
    • A clinical diagnosis supported by low levels of leptin (a hormone related to fat) and signs of insulin resistance (when the body doesn't respond well to insulin, a hormone that controls blood sugar).
    • The doctor must confirm that other conditions that might look similar have been ruled out.
    • Children with high blood sugar levels (HbA1c ≥ 6.5%) or high levels of triglycerides (a type of fat in the blood) (≥ 2.3 mmol/L or 200 mg/dL).
    • If the child has diabetes, they should be on a stable treatment plan (diet and/or medication) for at least 90 days before the study starts. If they use insulin, the dose should not change by more than 20%.
    • If the child has high triglyceride levels, they should be on a stable treatment plan (diet and/or medication) for at least 6 weeks before the study starts.
    • The parent or guardian must be able to give informed consent and agree to follow the study's requirements.
    • The child should be in stable mental and physical health according to the doctor's opinion.

Who cannot join the trial?

3 criteria

  • Patients who are not diagnosed with generalised lipodystrophy cannot participate. This is a condition where the body has a problem storing fat under the skin.
  • Patients who are 6 years of age or older cannot participate. The study is only for children under 6 years old.
  • Patients who do not have associated diabetes mellitus or hypertriglyceridaemia cannot participate. Diabetes mellitus is a condition where the body has trouble controlling blood sugar levels. Hypertriglyceridaemia is when there are high levels of fats called triglycerides in the blood.
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Investigated drugs

Metreleptin is a medication used in this clinical trial to help manage a rare condition called generalized lipodystrophy in young children under 6 years old. This condition affects how the body stores fat, leading to problems like diabetes and high levels of fats in the blood, known as hypertriglyceridemia. Metreleptin works by replacing a hormone called leptin, which is usually low in people with this condition. By doing this, it helps to control blood sugar levels and reduce fat levels in the blood, aiming to improve the overall health of the patients involved in the trial.

What is already known about the treatment

Metreleptin – Metreleptin is administered as an injection under the skin, typically used for treating generalized lipodystrophy, a condition where the body cannot properly store fat. This medication is currently being studied in clinical trials, particularly for its effectiveness in young children under 6 years old who also have diabetes or high levels of fats in the blood. Metreleptin works by mimicking a hormone called leptin, which helps regulate energy balance and metabolism in the body. It is classified as a hormone replacement therapy, aiming to restore normal metabolic functions in patients with leptin deficiency.

Investigated diseases

Generalized Lipodystrophy – Generalized lipodystrophy is a rare disorder characterized by the near-total loss of adipose tissue throughout the body. This condition leads to a deficiency in fat storage, resulting in a lack of the hormone leptin, which is produced by fat cells. As a result, individuals often experience metabolic complications such as insulin resistance, diabetes mellitus, and hypertriglyceridemia. The absence of fat tissue can also lead to an abnormal distribution of muscle and bone, giving the body a muscular appearance. Over time, the metabolic disturbances can affect various organs, including the liver, leading to conditions like hepatic steatosis. The progression of the disease can vary, with symptoms often becoming more pronounced as the individual ages.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2022-501781-22-00Protocol codeAPL-20Estimated enrolment25 patientsSponsorAmryt Pharmaceuticals Designated Activity Company

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

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