What side effects are associated with this type of intervention?

Common treatments for Generalized Lipodystrophy, such as leptin replacement therapy with metreleptin, are known to cause side effects including hypoglycemia, weight loss, and the development of anti-leptin antibodies. Other potential side effects include injection site reactions, headache, and abdominal pain. Treatments similar to REGN4461, a leptin receptor agonist antibody, may exhibit a similar side effect profile.

What prior FDA approvals exist?

The FDA has approved metreleptin (Myalept) for the treatment of generalized lipodystrophy. Metreleptin is a recombinant human leptin analog that helps to manage metabolic complications associated with leptin deficiency in patients with generalized lipodystrophy. This treatment is similar to REGN4461, which is a leptin receptor agonist antibody, as both target the leptin pathway to address metabolic dysfunctions.

What other types of research exist?

Promising alternatives to REGN4461 for Generalized Lipodystrophy patients include: 1) Pioglitazone, which improves lipid and glycemic parameters, 2) Sirolimus, an mTOR inhibitor that slows disease progression in metabolic conditions, and 3) Tesevatinib, which has shown efficacy in ameliorating disease progression in polycystic kidney disease and may have potential in lipid metabolism regulation.

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Monoclonal Antibodies

REGN4461 for Generalized Lipodystrophy

Phase 2

Waitlist Available

Research Sponsored by Regeneron Pharmaceuticals

Eligibility Criteria Checklist

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

Must have

Diagnosis of congenital or acquired generalized lipodystrophy (GLD), as defined in the protocol

Must not have

Uncontrolled infection with hepatitis B or hepatitis C infection, or known active tuberculosis at screening

Treatment with metreleptin within 1 month of the screening visit

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at week 8, 16 and 24

Summary

This trial is testing a new medication called REGN4461 to help patients with high blood sugar or high blood fat levels. The medication aims to improve how the body handles sugar and fats, making it easier to control diabetes and reduce heart disease risk.

Who is the study for?

This trial is for individuals with Generalized Lipodystrophy who have been on a stable diet and medication regimen for at least 3 months. They must have high blood sugar (HbA1c ≥7%) or high fasting triglycerides (TG ≥250 mg/dL). Those with recent weight loss treatments, metreleptin use, uncontrolled hepatitis B/C or tuberculosis, HIV, or participation in other drug trials recently are excluded.

What is being tested?

The study tests REGN4461's effects on blood sugar control and fasting triglyceride levels in patients with Generalized Lipodystrophy. It compares low-dose and high-dose REGN4461 against a placebo to see how well it works and its impact on insulin sensitivity.

What are the potential side effects?

While the specific side effects of REGN4461 aren't listed here, common ones may include reactions at the injection site, fatigue, headache, potential allergic responses to the antibody treatment, as well as possible impacts on liver function.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with a rare condition that causes a lack of fat tissue.

Exclusion Criteria

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

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I do not have active hepatitis B, hepatitis C, or tuberculosis.

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I have not taken metreleptin in the last month.

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I have not taken more than 7.5 mg of steroids daily in the last 3 months and don't plan to during the study.

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I have HIV or tested positive for HIV.

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I haven't been in a study for a new treatment within the last 3 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at week 8, 16 and 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at week 8, 16 and 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and at week 8, 16 and 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

Absolute change from baseline fasting glucose

Absolute change from baseline hemoglobin A1c (HbA1c)

Absolute change from baseline weighted mean glucose (WMG)

+1 more

Secondary study objectives

Absolute change from baseline in HbA1c over time

Absolute change from baseline in WMG over time

Absolute change from baseline in fasting glucose

+8 more

Trial Design

2Treatment groups

Experimental Treatment

Group I: Treatment BExperimental Treatment3 Interventions

Group II: Treatment AExperimental Treatment3 Interventions

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo

1995

Completed Phase 3

~2670

Low-Dose REGN4461

2020

Completed Phase 2

~20

High-dose REGN4461

2020

Completed Phase 2

~20

0 / 6Eligibility 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

Common treatments for Generalized Lipodystrophy often involve the use of leptin replacement therapy, such as metreleptin, which acts by binding to leptin receptors to regulate energy balance, appetite, and metabolism. This is crucial for patients with Generalized Lipodystrophy because they typically have low levels of leptin, leading to severe metabolic disturbances including insulin resistance, hypertriglyceridemia, and fatty liver disease. By mimicking the action of leptin, these treatments help to normalize metabolic functions, improve glycemic control, and reduce triglyceride levels, thereby addressing the core metabolic issues associated with the condition.

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