METRE-PL Study: Exploring a treatment option for familial partial lipodystrophy
Overview
Familial partial lipodystrophy
Lipodystrophy is a rare group of disorders characterized by complete or partial loss of subcutaneous adipose tissue (the fatty tissue layer that lies directly under the skin). Lipodystrophy can either be inherited or acquired.
Familial partial lipodystrophy (FPLD) is a genetic form of partial lipodystrophy (PL) that is caused by mutations of specific genes. Individuals with FPLD often have reduced subcutaneous fat in the arms and legs, and their head and torso regions may or may not have loss of fat. Individuals with FPLD may also have excess subcutaneous fat in other areas of the body, especially the neck, face, and abdomen.
As a result of adipose tissue loss, most patients with FPLD have low levels of the leptin hormone. A leptin deficiency disrupts the body’s ability to regulate hunger and energy. Metabolic abnormalities often occur with FPLD and other lipodystrophy subtypes, including severe insulin resistance, diabetes, and high levels of triglycerides (fat) in the blood.
METRE-PL
METRE-PL is a Phase 3 clinical study evaluating the use of metreleptin, an investigational medicine, as a leptin replacement therapy to treat the complications of leptin deficiency. The purpose of the study is to evaluate the safety and efficacy of metreleptin treatment in patients with FPLD. This study will test a daily dose of metreleptin or a placebo injected subcutaneously for 12 months in patients with FPLD. All patients that complete the study will have the option to receive metreleptin.
To provide comprehensive support for enrolled study participants and their caregivers, all expenses related to travel, accommodation, and study assessments will be covered.
Are you a physician?
If you have potentially eligible patients who may be interested in participating in the METRE-PL study, please contact MedInfo for more information. Please note your patient will need a confirmed FPLD diagnosis to be eligible for the study.
Eligibility
12 years of age and older
Diagnosis of familial partial lipodystrophy (FPLD)
Willing to follow dietary restrictions recommended by the investigator
- ≥ 12 years of age
- Diagnosis of FPLD based on deficiency of partial subcutaneous body fat assessed by physical examination, plus 1 of the following:
- Documented genetic diagnosis of FPLD (this can be based on a prior test, or one can be completed during the study)
OR - Documented evidence of positive family history (first degree biological relative) of FPLD
OR - Other criteria that include complications associated with partial lipodystrophy (PL)
- Documented genetic diagnosis of FPLD (this can be based on a prior test, or one can be completed during the study)
- Willing to follow dietary restrictions recommended by the investigator
Note: Additional inclusion criteria apply
- Previous treatment with metreleptin
- Currently pregnant or breastfeeding
Note: Additional exclusion criteria apply
Have questions about the METRE-PL study?
Please reach out to your (or your child’s) physician and ask them to contact MedInfo for more information.
FAQs
A clinical study is a carefully designed and controlled research study that aims to evaluate the safety and effectiveness of an investigational medication in humans. To enroll in a clinical study, you (or your child) are required to meet all eligibility requirements and must be willing to follow the instructions of the study physician throughout the study’s duration. To ensure you (or your child) remain safe and comfortable during the study, a dedicated team of physicians and other medical professionals will closely monitor your health during the study.
Familial partial lipodystrophy (FPLD) is a genetic form of partial lipodystrophy (PL) that is characterized by deficiency of subcutaneous body fat (the fatty tissue layer that lies directly under the skin) in a partial fashion. Individuals with FPLD often have reduced subcutaneous fat in the arms and legs, and the head and torso regions may or may not have loss of fat. Conversely, individuals with FPLD may also have excess subcutaneous fat accumulation in other areas of the body, especially the neck, face, and intra-abdominal regions. The prevalence of FPLD is not well understood, and it is possible that the disease may be underdiagnosed.
- Documented genetic diagnosis of FPLD (this can be based on a prior test, or one can be completed during the study) OR
- Documented evidence of positive family history (first degree biological relative) of FPLD OR
- Other criteria that include complications associated with partial lipodystrophy (PL)
Participation in the METRE-PL study may last for at least 17 months. In this study, you (or your child) will receive either metreleptin or a placebo, as an injection under your skin. A placebo injection looks like the metreleptin injection but does not contain any active medication. Both metreleptin and placebo will be referred to as the “study medication.” Neither you nor the study physician will know if metreleptin or a placebo is given.
As part of your (or your child’s) participation, you (or your child) will receive a daily dose of the study medication for 12 months. The first study medication dose will be administered to you (or your child) at your study location, and you are responsible for administering all subsequent doses for the remainder of the study. The daily dose requires an injection of the study medication under your (or your child’s) skin.
After completing the 12-month study, you will be offered to enroll in an expanded access program (EAP) where you will receive metreleptin as per your country specific rules.
During the study, you (or your child) will be required to:
- Take the study medication daily
- Go to the study location for study visits
- Follow a specific diet
- Continue taking your (or your child’s) regular medications while receiving the study medication, if applicable
In addition, there will be several laboratory assessments designed to monitor your (or your child’s) health and condition throughout the study’s duration. These assessments include, but are not limited to, blood tests, urine tests, fasting, and optional genetic testing to confirm diagnosis of FPLD.
To provide comprehensive support for enrolled study participants and their caregivers, all expenses related to travel, accommodation, and study assessments will be covered.
Choosing whether to take part in a clinical study is an important decision. If you (or your child) have questions about the METRE-PL study, talk to your physician or fill out the contact form below. By contacting us, you are under no obligation to take part in the study.
The METRE-PL study is being conducted in the US and Canada. See below for a list of our current study locations.
Locations
Contact
See if you (or your child) may be eligible for more information about the METRE-PL study.
