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 States20 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 range18 Years

SexALL

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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

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