What side effects are associated with this type of intervention?

Metreleptin, a leptin replacement therapy used for Partial Lipodystrophy, can cause side effects such as hypoglycemia, particularly in diabetic patients, and the development of anti-metreleptin antibodies, which may reduce its efficacy. Other side effects include headache, decreased appetite, and abdominal pain. There is also a rare risk of lymphoma. Broadly, treatments for Partial Lipodystrophy, including insulin sensitizers and lipid-lowering agents, may cause gastrointestinal disturbances and hypoglycemia.

What prior FDA approvals exist?

Metreleptin, a recombinant human leptin analog, has been approved by the FDA for the treatment of generalized lipodystrophy, a condition related to partial lipodystrophy. It is used to manage metabolic complications associated with leptin deficiency. While specific FDA approvals for partial lipodystrophy treatments are not detailed in the provided context, metreleptin's approval for generalized lipodystrophy suggests its potential relevance for similar conditions.

What other types of research exist?

Promising alternatives to Metreleptin for Partial Lipodystrophy patients include GLP-1 receptor agonists like Liraglutide, which have shown efficacy in weight management and metabolic control. Additionally, dual GIP and GLP-1 receptor agonists such as Tirzepatide are emerging as effective treatments for obesity and metabolic disorders, potentially offering benefits for Partial Lipodystrophy patients.

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Leptin Analog

Metreleptin for Partial Lipodystrophy (METRE-PL Trial)

Phase 3

Recruiting

Research Sponsored by Amryt Pharma

Eligibility Criteria Checklist

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

Must have

Diagnosis of Familial Partial Lipodystrophy (FPLD)

Must not have

Previous treatment with metreleptin

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

Pivotal Trial

Summary

This trial tests the safety and effectiveness of daily injections of metreleptin for people with Partial Lipodystrophy. The treatment aims to replace a missing hormone to help their bodies manage fat and sugar levels better. Metreleptin is approved by the United States Food and Drug Administration for the treatment of generalized forms of lipodystrophy and has shown improvement in many patients.

Who is the study for?

This trial is for individuals with a condition called Partial Lipodystrophy, specifically those diagnosed with Familial Partial Lipodystrophy (FPLD). It's not open to people who have already been treated with metreleptin.

What is being tested?

The study is testing the safety and effectiveness of a daily injection of a drug called metreleptin compared to a placebo. This is done in a controlled way where neither the participants nor the researchers know who gets the real treatment or placebo.

What are the potential side effects?

Possible side effects from metreleptin may include low blood sugar, weight loss, headaches, abdominal pain, and allergic reactions at the injection site.

Eligibility Criteria

Inclusion Criteria

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

Select...

I have been diagnosed with Familial Partial Lipodystrophy.

Exclusion Criteria

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

Select...

I have been treated with metreleptin before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months 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 from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL)

Percent change from Baseline to month 6 in fasting TGs in subjects with partial lipodystrophy (PL)

Secondary study objectives

Change from Baseline to each assessment time point in quality of life (QoL) in all subjects

Change from baseline to months 9 and 12 in HbA1c in subjects with partial lipodystrophy (PL)

Percent change from baseline to months 9 and 12 in fasting TGs in subjects with partial lipodystrophy (PL)

+1 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Active Control

Placebo Group

Group I: metreleptinActive Control1 Intervention

Metreleptin \[Recombinant-methionyl human Leptin; r-metHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake

Group II: placeboPlacebo Group1 Intervention

Placebo for daily injection is a sterile, white, solid lyophilised cake

0 / 2Eligibility 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 leptin replacement therapy, works by supplementing the deficient leptin hormone in patients with Partial Lipodystrophy. Leptin is crucial for regulating energy balance, appetite, and metabolism. In Partial Lipodystrophy, patients have low leptin levels due to abnormal fat distribution, leading to metabolic complications such as insulin resistance, hypertriglyceridemia, and fatty liver disease. By restoring leptin levels, metreleptin helps improve these metabolic abnormalities, thereby reducing the risk of associated complications and improving overall metabolic health.

Lipocalin 2 Influences Bone and Muscle Phenotype in the MDX Mouse Model of Duchenne Muscular Dystrophy.Follow-up of late-onset Pompe disease patients with muscle magnetic resonance imaging reveals increase in fat replacement in skeletal muscles.P2X7 purinoceptor as a therapeutic target in muscular dystrophies.