TerminatedPhase 2

A Study to Examine the Effects of the Leptin Receptor (LEPR) Agonist Antibody REGN4461 in Adult Patients With Familial Partial Lipodystrophy (FPLD)

Stopped: Sponsor Decision

United States, France, Spain20 participantsStarted 2022-02-28

Plain-language summary

Two cohorts are being studied based on leptin levels. Cohort A is composed of patients with baseline leptin \<8.0 ng/mL and Cohort B is composed of patients with baseline leptin 8.0 to ≤20.0 ng/mL The primary objectives will be evaluated for patients in Cohort A only: * To evaluate the effect of REGN4461 on fasting triglycerides (TG) in patients with elevated baseline fasting TG * To evaluate the effect of REGN4461 on hyperglycemia in patients with elevated baseline Hemoglobin A1c (HbA1c) The following secondary objectives of the study will be evaluated for Cohort B and for the combined set of Cohorts A plus B: * To evaluate the effect of REGN4461 on fasting TG levels in patients with hypertriglyceridemia * To evaluate the effect of REGN4461 on glycemic control in patients with hyperglycemia The following secondary objectives of the study will be evaluated for Cohorts A and B separately, and for the combined set of Cohorts A plus B: * To evaluate the effect of REGN4461 on liver fat in patients with hepatic steatosis * To evaluate the effect of REGN4461 on hunger * To evaluate safety and tolerability of REGN4461 * To characterize the concentration profile of REGN4461 over time * To assess immunogenicity to REGN4461

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Key Inclusion Criteria: * Clinical diagnosis of familial partial lipodystrophy as defined in the protocol * Fasting leptin level ≤20.0 ng/ml, as determined during the screening period * Presence of significant metabolic abnormalities related to glucose and triglycerides (TGs) as defined in the protocol * Stable body weight within the 3 months prior to screening (no gain or loss of \>5% current weight) * Stable diet during the past 3 months defined as no major change in macronutrient composition (eg, starting or stopping diets such as Atkins, Paleo, Vegetarianism, Veganism) * No clinically meaningful change in medication regimen in the 3 months prior to screening as defined in the protocol Key Exclusion Criteria: * Treatment with metreleptin within 3 months of the screening visit * Patients with a diagnosis of generalized lipodystrophy * Patients with a diagnosis of acquired lipodystrophy * Pregnant or breastfeeding women NOTE: Other protocol defined inclusion/exclusion criteria apply

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Percent Change From Baseline to Week 12 in Fasting Serum Triglyceride (TG) (Cohort A)

Timeframe: Baseline to week 12

Change From Baseline to Week 12 in Hemoglobin A1c (HbA1c) (Cohort A)

Timeframe: Baseline to week 12

Trial details

NCT IDNCT05088460

SponsorRegeneron Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-12-13

Results submitted2025-04-16

View on ClinicalTrials.gov More Familial Partial Lipodystrophy trials