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

• Status: Not yet recruiting
• Trial end date: 2023-11-02
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Regeneron Pharmaceuticals

Criteria

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