Metreleptin for Lipomatosis

Overseen By Elif Oral

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Michigan

Disqualifiers: Advanced liver disease, HIV, ESRD, active cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

4 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team to understand any specific requirements.

What evidence supports the effectiveness of the drug metreleptin for treating lipomatosis?

Metreleptin, a synthetic version of the hormone leptin, has been shown to improve metabolic issues like high blood sugar and high triglycerides in patients with generalized lipodystrophy, a condition related to lipomatosis. It has been approved for use in treating lipodystrophy, suggesting it may help manage similar metabolic complications in lipomatosis. ^{1 2 3 4 5}

How is the drug metreleptin unique in treating lipomatosis?

Metreleptin is unique because it is a synthetic version of the hormone leptin, used to treat metabolic issues in patients with lipodystrophy, a condition with little or no body fat. It helps improve insulin sensitivity and reduce high blood sugar and fat levels, which are not effectively managed by conventional treatments. ^{1 2 3 4 5}

What is the purpose of this trial?

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.

Research Team

Elif Oral

Principal Investigator

University of Michigan

Eligibility Criteria

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.

Inclusion Criteria

Having no condition that may impede successful data collection or interfere with testing parameters

You are not currently breastfeeding.

You have been diagnosed with MSL and are being treated at the University of Michigan.

Exclusion Criteria

You have a record of having HIV infection.

You are not being excluded for general obesity unless it is caused by a specific medical condition called congenital leptin deficiency.

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Metreleptin treatment and effects on body composition, metabolic parameters, and safety are assessed

24 weeks

Regular visits for assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment, with additional safety assessments

1 year

Long-term follow-up

Continued data collection, including adverse events, up to 5 years per participant

Up to 5 years

Treatment Details

Interventions

• Metreleptin

Trial Overview 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.

Participant Groups

Group I: Patients with MSL Experimental Treatment 1 Intervention

Metreleptin is already approved in United States, Canada for the following indications:

Approved in United States as Myalept for:

• Complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy

Approved in Canada as Myalept for:

• Complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy
• Patients with partial lipodystrophy

Who Is Running the Clinical Trial?

University of Michigan

Lead Sponsor

Trials

1,891

Recruited

6,458,000+

Amryt Pharma

Industry Sponsor

Trials

9

Recruited

1,000+

Findings from Research

The MEASuRE registry is collecting long-term safety and effectiveness data on metreleptin, a treatment for metabolic complications in patients with lipodystrophy, involving 85 patients from 15 US and 4 EU sites as of October 2022.

The integration of MEASuRE with the existing European Registry of Lipodystrophies (ECLip) has allowed for efficient data collection and validation, demonstrating that collaboration can enhance patient enrollment and streamline data gathering for new registries.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Metreleptin Effectiveness and Safety Registry (MEASuRE): concept, design and challenges. Haymond, MW., Araújo-Vilar, D., Balser, J., et al.[2023]

Metreleptin, a synthetic leptin analog, has been approved by the FDA for treating generalized lipodystrophy and has shown significant efficacy in reversing severe metabolic complications like hyperglycemia and fatty liver disease in patients with leptin deficiency.

While metreleptin is generally safe for daily subcutaneous administration, there are concerns about potential immune complications, such as lymphoma, and further research is needed to understand its long-term effects and the development of anti-leptin antibodies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New advances in the treatment of generalized lipodystrophy: role of metreleptin. Rodriguez, AJ., Mastronardi, CA., Paz-Filho, GJ.[2022]

Metreleptin, a recombinant human leptin analog, has been approved for treating generalized lipodystrophy and has shown significant improvements in metabolic issues like high blood sugar and triglycerides, as well as liver health.

There is emerging evidence that metreleptin may also help with metabolic problems in patients with partial lipodystrophy, although more research is needed to confirm its efficacy in this group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Metreleptin Treatment in Patients with Non-HIV Associated Lipodystrophy. Akinci, G., Akinci, B.[2019]

References

1. England pubmed.ncbi.nlm.nih.gov

The Metreleptin Effectiveness and Safety Registry (MEASuRE): concept, design and challenges. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Immunogenicity associated with metreleptin treatment in patients with obesity or lipodystrophy. [2019]

3. New Zealand pubmed.ncbi.nlm.nih.gov

New advances in the treatment of generalized lipodystrophy: role of metreleptin. [2022]

4. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Metreleptin Treatment in Patients with Non-HIV Associated Lipodystrophy. [2019]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Metreleptin: first global approval. [2021]