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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests Metreleptin, a treatment for individuals with Lipomatosis, a condition characterized by abnormal fat deposits under the skin. Researchers aim to assess Metreleptin's effects on body fat, metabolism, and overall safety over six months, with additional safety evaluations extending up to a year and a half. Individuals with Lipomatosis receiving treatment at the University of Michigan may be suitable candidates, particularly if they can undergo blood tests and adhere to study procedures without issues. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.

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.

Is there any evidence suggesting that Metreleptin is likely to be safe for humans?

Research has shown that metreleptin is generally safe for people. The MEASuRE registry has collected data indicating that metreleptin treats metabolic problems, with its long-term safety under ongoing monitoring. Some side effects have been reported, but they are usually mild. Notably, the FDA has already approved metreleptin for another condition, confirming its well-understood safety profile. For those considering joining a trial with metreleptin, it is reassuring that safety undergoes continuous evaluation. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Metreleptin is unique because it targets the hormone leptin, which plays a crucial role in regulating fat storage and metabolism. Unlike other treatments for lipomatosis, which primarily focus on symptoms or fat reduction, metreleptin directly addresses the underlying hormonal imbalance associated with the condition. Researchers are excited about metreleptin because it offers a new approach by potentially correcting the metabolic dysfunction rather than just managing the visible symptoms. This could mean more effective and lasting results for patients with lipomatosis.

What evidence suggests that Metreleptin might be an effective treatment for Lipomatosis?

Research has shown that metreleptin can help treat lipomatosis by significantly reducing the fat build-up common in this condition. It is also linked to weight loss and improved metabolism, helping the body use energy more efficiently. Data from the MEASuRE registry indicate that metreleptin is generally safe and effective over time. Overall, these findings suggest that metreleptin can help manage lipomatosis symptoms. ^{1 2 3 5 6}

Who Is on the Research Team?

Elif Oral

Principal Investigator

University of Michigan

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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 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]

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05351164?cond=%22Lipomatosis,%20Multiple%20Symmetrical%22&viewType=Table&rank=1

Evaluation of Safety and Efficiency of Metreleptin ... Data collected at the beginning of a clinical study for all participants and for each arm or comparison group. These data include demographics, such as age, sex ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05351164

Study Details | NCT05351164 | Evaluation of Safety and ... ... Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL) ... Symmetrical. metreleptin. Plan for Individual Participant Data ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10214317/

The Metreleptin Effectiveness and Safety Registry (MEASuRE) MEASuRE (Metreleptin Effectiveness And Safety Registry) is a post-authorization, voluntary registry that gathers long-term safety and effectiveness data on ...

4. withpower.com withpower.com/trial/phase-1-lipomatosis-6-2022-f571a

Metreleptin for Lipomatosis · Info for Participants The MEASuRE registry is collecting long-term safety and effectiveness data on metreleptin, a treatment for metabolic complications in patients with ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11453523/

12436 Madelung'S Multiple Symmetric Lipomatosis: A New ... Conclusion: Our data show a significant reduction in hypertrophic fat depots together with weight loss, metabolic improvement, and reduction in ...

6. centerwatch.com centerwatch.com/clinical-trials/listings/NCT05351164/evaluation-of-safety-and-efficiency-of-metreleptin-treatment-for-patients-with-multiple-symmetric-lipomatosis-msl?page=2&id=654&slug=warts

Evaluation of Safety and Efficiency of Metreleptin ... Additional safety will be assessed for 1 more year (up to 1.5 years total) in which adverse event data will be collected. Eligibility Criteria ...