Study on Metreleptin for Patients with Partial Lipodystrophy

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What is this study about?

This clinical trial is focused on studying a condition called partial lipodystrophy. This is a rare disorder where the body is unable to store fat properly, leading to a lack of fat in certain areas of the body. The study will evaluate the effects of a treatment called Metreleptin, which is a protein that helps regulate fat and energy balance in the body. Participants in the study will receive either Metreleptin or a placebo, which is a substance with no active medication, to compare the effects.

The purpose of the study is to assess the safety and effectiveness of daily injections of Metreleptin over a 12-month period. Participants will receive the treatment through a subcutaneous injection, which means the medication is injected under the skin. The study will monitor changes in health markers such as blood sugar levels and fat levels in the blood, which are often affected in people with partial lipodystrophy.

Throughout the study, participants will have regular check-ups to track their progress and any changes in their condition. The study aims to provide valuable information on how Metreleptin can help manage partial lipodystrophy and improve the quality of life for those affected by this condition.

1 initial visit

Upon joining the study, an initial visit is conducted. During this visit, eligibility is confirmed based on specific criteria, including age, medical history, and current health status.

A physical examination is performed, and blood samples are taken to measure various health indicators, such as glycated hemoglobin (HbA1c) and fasting triglycerides (TGs).

2 randomization

Participants are randomly assigned to one of two groups: one receiving the active medication, metreleptin, and the other receiving a placebo. This process is double-blind, meaning neither the participant nor the study staff knows which group the participant is in.

The medication is administered as a daily subcutaneous injection, which means it is injected under the skin.

3 treatment phase

The treatment phase lasts for 12 months. Participants are required to self-administer the daily injection of either Myalepta 11.3 mg or a placebo.

Regular follow-up visits are scheduled to monitor health status, adjust dosages if necessary, and ensure adherence to the study protocol.

4 mid-study evaluation

At the 6-month mark, a comprehensive evaluation is conducted to assess changes in health indicators, such as HbA1c and TGs.

Participants may undergo additional blood tests and physical examinations to track progress and any side effects.

5 final evaluation

At the end of the 12-month period, a final evaluation is performed. This includes a thorough review of health changes and any side effects experienced during the study.

Participants are asked to return any unused medication and complete a final set of questionnaires regarding their experience.

6 post-study follow-up

A follow-up period of 4 weeks is observed after the last dose of the study treatment. During this time, participants are monitored for any lingering effects or delayed reactions.

Participants are encouraged to maintain contact with their healthcare provider and report any health changes.

Who Can Join the Study?

Must be at least 12 years old.
Females must be either postmenopausal (no periods for at least 1 year), surgically sterile, or willing to use a highly effective method of birth control during the study. Hormonal birth control alone is not enough; an additional barrier method must be used.
Males must be surgically sterile or willing to use an acceptable method of birth control during the study, such as condoms or having a partner who uses effective birth control.
If you donate blood, eggs, or sperm, you must be willing to stop donations during the study and for 4 weeks after the last dose of the study treatment.
Must be willing to provide informed consent before any study-specific procedures. If under 18, a parent or legal guardian must also provide consent.
Must be willing to follow dietary restrictions recommended by the study doctor.
Must have a diagnosis of familial partial lipodystrophy (FPLD), which is a condition involving a deficiency of body fat in certain areas. This can be confirmed by a physical exam, genetic testing, or family history.
Must have poor metabolic control, defined as having a blood sugar level (HbA1c) of 7% or higher, or fasting triglycerides (a type of fat in the blood) of 500 mg/dL or higher.
If you have diabetes and a blood sugar level (HbA1c) of 7% or higher, you should be on a stable diabetes treatment for at least 3 months before the study.
If you have diabetes and a blood sugar level (HbA1c) below 7%, you should be on a stable treatment for at least 6 weeks before the study.
If you have high triglycerides, you should be on a stable lipid-lowering treatment for at least 6 weeks before the study.
If you are on diabetes or lipid-lowering medication, the dose must be stable for at least 6 weeks before the study.
Females of childbearing potential must have a negative pregnancy test before starting the study.

Who Cannot Join the Study?

Patients with any other serious medical condition that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who have had a recent major surgery.
Patients who are currently participating in another clinical trial.
Patients with a history of drug or alcohol abuse.
Patients who have an allergy to the study medication or its ingredients.
Patients with uncontrolled high blood pressure.
Patients with severe liver or kidney disease.
Patients with a history of heart disease.
Patients who are unable to comply with the study procedures.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
Academisch Ziekenhuis Leiden	Leiden	The Netherlands
Universidade De Santiago De Compostela	Santiago De Compostela	Spain
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie	Cracow	Poland
Saqqgstxj Rpatitt Ucujgbwelz Majzltn Cpkyms	Nijmegen	The Netherlands

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	01.02.2024
Poland	Not recruiting	01.02.2024
Spain	Not recruiting	01.02.2024
The Netherlands	Not recruiting	01.02.2024

Trial locations

Investigated drugs:

Metreleptin

Metreleptin is a medication used in this clinical trial to help people with a condition called partial lipodystrophy. This condition affects how the body stores fat, leading to problems with metabolism and energy use. Metreleptin is given as a daily injection under the skin. It works by replacing a hormone called leptin, which is usually low in people with lipodystrophy. By doing this, metreleptin helps to regulate the body’s energy balance and improve metabolic health. The goal of using metreleptin in this trial is to see if it can safely and effectively help people with partial lipodystrophy manage their condition better.

Familial Partial Lipodystrophy – Familial Partial Lipodystrophy is a rare genetic disorder characterized by the abnormal distribution of body fat. In this condition, individuals lose fat from certain areas of the body, such as the arms, legs, and buttocks, while fat may accumulate in other areas like the face, neck, and trunk. This uneven fat distribution can lead to metabolic complications, including insulin resistance and high levels of triglycerides in the blood. Over time, these metabolic issues can contribute to the development of diabetes and cardiovascular problems. The progression of the disease varies among individuals, with some experiencing more severe symptoms than others. The condition is typically inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder.

Trial ID:

2022-501799-24-00

Protocol code:

APG-20

NCT ID:

NCT05164341

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Metreleptin for Patients with Partial Lipodystrophy

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