What side effects are associated with this type of intervention?

Metreleptin, used in the treatment of Multiple Symmetrical Lipomatosis, can have side effects including hypoglycemia, headache, decreased weight, and abdominal pain. Other potential side effects may include fatigue, nausea, and injection site reactions. It is important for patients to discuss these potential side effects with their healthcare provider to manage and mitigate any adverse effects effectively.

What prior FDA approvals exist?

There are no specific FDA-approved treatments for Multiple Symmetrical Lipomatosis (MSL). However, Metreleptin, a recombinant analog of human leptin, is being studied for its effects on body composition and metabolic parameters in MSL patients. Metreleptin is already approved by the FDA for the treatment of generalized lipodystrophy, a condition characterized by abnormal or degenerative conditions of the body's adipose tissue, which shares some metabolic dysfunctions with MSL.

What other types of research exist?

As of 2024, promising novel alternatives to Metreleptin for Multiple Symmetrical Lipomatosis (MSL) patients include GLP-1 receptor agonists (such as semaglutide and liraglutide) due to their effects on weight loss and metabolic regulation. Additionally, therapies targeting adipokines and anti-inflammatory pathways, such as myostatin inhibitors and cardiosphere-derived cells (CDCs), are under investigation and may offer potential benefits. Further clinical trials and studies are needed to confirm their efficacy and safety in MSL patients.

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Leptin Analog

Metreleptin for Lipomatosis

Q: What is the mechanism of action of this treatment?

Metreleptin, a recombinant analog of human leptin, plays a crucial role in regulating energy balance and metabolism, which is particularly important for patients with Multiple Symmetrical Lipomatosis (MSL). MSL is characterized by abnormal fat accumulation, and Metreleptin helps by restoring leptin levels, thereby normalizing metabolic functions. This can potentially reduce the size and number of lipomas, offering a targeted approach to manage the disease by addressing the underlying metabolic dysregulation.

Phase 2

Waitlist Available

Led By Elif Oral

Research Sponsored by University of Michigan

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

<60 years of age

<60 years of age.

Must not have

Clinically relevant Coronary artery disease (CAD): history of stent or Coronary artery bypass graft surgery (CABG) with cardiologist confirmed angina

Presence of advanced liver disease (abnormal synthetic function, prothrombin time [PT], or albumin) in medical records

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 24

Awards & highlights

Summary

This trial will treat patients with a specific genetic mutation (MFN2 R707W) using Metreleptin. Metreleptin acts like a hormone that helps control hunger and energy use. The study will assess changes in body composition, metabolism, and safety over several months, with additional safety monitoring for an extended period. Metreleptin is an analogue of the human hormone leptin, developed to treat metabolic disorders including lipodystrophy, and has been approved by the FDA for generalized lipodystrophy.

Who is the study for?

This trial is for individuals under 60 with Multiple Symmetric Lipomatosis (MSL), specifically those with a certain genetic mutation (homozygous MFN2 R707W). Participants must be able to undergo blood sampling, not have conditions affecting the study, and if female and of childbearing potential, they must not be pregnant or breastfeeding. They should also be patients at the University of Michigan.

What is being tested?

The trial tests Metreleptin's effects on body composition and metabolic parameters in MSL patients over six months. It aims to evaluate both safety and efficiency by monitoring changes after administering Metreleptin.

What are the potential side effects?

Potential side effects of Metreleptin may include allergic reactions for those sensitive to it, issues related to blood sampling such as bruising or infection, and other unspecified risks that could arise from altering metabolic parameters.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am younger than 60 years old.

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I am younger than 60 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had heart surgery or a stent due to blocked arteries and still experience chest pain.

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My medical records show I have advanced liver disease.

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I cannot walk or travel to the University of Michigan for study visits.

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My medical records show I have a condition that could increase my bleeding risk.

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I have end-stage kidney disease, active cancer, or severe heart failure.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in total adiposity

Change in truncal adiposity

Side effects data

From 2015 Phase 2 trial • 103 Patients • NCT00025883

15%

Musculoskeletal pain

12%

Weight loss

10%

Hypoglycemia

Nausea

Abdominal pain

Infection

Fatigue

tumor, benign

Anemia

Anxiety

Diarrhea

Insomnia

Constipation

Depression

Headache

Joins pain

Decreased appetite

Iron deficiency

Pneumonia

Exacerbations of heart failure

Group B streptococcus bacteremia

Abdominal pain requiring hospitalization

Osteomyelitis

Acute exacerbation of pancreatitis

Ovarian cyst requiring bilateral oophorectomy and total abdominal hysterectomy

Cellulitis

Miscarriage

Severe acute bronchitis

100%

80%

60%

40%

20%

Study treatment Arm

Metreleptin

Trial Design

1Treatment groups

Experimental Treatment

Group I: Patients with MSLExperimental Treatment1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Metreleptin

FDA approved

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Metreleptin, a recombinant analog of human leptin, plays a crucial role in regulating energy balance and metabolism, which is particularly important for patients with Multiple Symmetrical Lipomatosis (MSL). MSL is characterized by abnormal fat accumulation, and Metreleptin helps by restoring leptin levels, thereby normalizing metabolic functions. This can potentially reduce the size and number of lipomas, offering a targeted approach to manage the disease by addressing the underlying metabolic dysregulation.

Selective capacity of metreleptin administration to reconstitute CD4+ T-cell number in females with acquired hypoleptinemia.