Familial Partial Lipodystrophy MedDRA version: 21.1 Level: PT Classification code 10053857 Term: Partial lipodystrophy System Organ Class: 10040785 - Skin and subcutaneous tissue disorders
Conditions
Interventions
Product Name: REGN4461
Product Code: REGN4461
Pharmaceutical Form: Lyophilisate for solution for infusion
INN or Proposed INN: REGN4461
CAS Number: 2305770-44-7
Current Sponsor code: REGN4461
Other de
Sponsors
Regeneron Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female, >/= 18 years of age at the screening visit. 2. Clinical diagnosis of familial partial lipodystrophy as defined in the protocol 3. Fasting leptin level 5% current weight) 6. Stable diet during the past 3 months defined as no major change in macronutrient composition (eg, starting or stopping diets such as Adkins, Paleo, Vegetarianism, Veganism). 7. No clinically meaningful change in medication regimen in the 3 months prior to screening as defined in the protocol NOTE: Other protocol defined inclusion criteria apply Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 36 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 4
Exclusion criteria
Exclusion criteria: 1. Treatment with metreleptin within 3 months of the screening visit 2. Patients with a diagnosis of generalized lipodystrophy 3. Patients with a diagnosis of acquired lipodystrophy 4. Pregnant or breastfeeding women NOTE: Other protocol defined exclusion criteria apply
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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 HbA1c;Secondary Objective: 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;Primary end point(s): Co-primary: • Percent change in fasting serum TG from baseline to week 12 in patients with elevated baseline fasting TG and with baseline leptin <8.0 ng/mL (Cohort A) • Absolute change in HbA1c from baseline to week 12 in patients with elevated baseline HbA1c and with baseline leptin <8.0 ng/mL (Cohort A);Timepoint(s) of evaluation of this end point: As Stated Above | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The following secondary endpoints will be analyzed for Cohort B and for the combined set of Cohorts A plus B: • Percent change in fasting serum TG from baseline to week 12 in patients with elevated baseline fasting TG • Absolute change in HbA1c from baseline to week 12 in patients with elevated baseline HbA1c The following secondary endpoints will be analyzed for Cohorts A and B separately, and for the combined set of Cohorts A plus B: • Percent change in fasting serum TG from baseline to week 12 compared to the percent change between week 12 and week 24 (study arm 1) • Percent change in fasting serum TG from baseline to week 24 (study arm 2) • Percent change in fasting serum TG after the first 12 weeks of exposure to REGN4461— from baseline to week 12 (study arm 2) and from week 12 to week 24 (study arm 1; patients must meet stability criteria) • Change in HbA1c from baseline to week 12 compared to the change between week 12 and week 24 (study arm 1) • Change in fasting glucose from baseline to week 12 compared to the change between week 12 and week 24 (study arm 1) • Change in HbA1c from baseline to week 24 (study arm 2) • Change in fasting glucose from baseline to week 24 (study arm 2) • Change in HbA1c from baseline to week 12 (study arm 2) and from week 12 to week 24 (study arm 1; patients must meet stability criteria) • Change in fasting glucose from baseline to week 12 (study arm 2) and from week 12 to week 24 (study arm 1) • Percent change in liver fat measured by magnetic resonance imaging-derived proton density fat fraction (MRI-PDFF) from baseline to week 12, REGN4461 versus placebo in patients with baseline liver fat (MRI-PDFF) >/= 8.5% • Percent change in liver fat (MRI-PDFF) from baseline to week 12 compared to the percent change between week 12 and week 24 (study arm 1) in patients with baseline liver fat (MRI-PDFF) >/= 8.5% • Percent change in liver fat (MRI-PDFF) from baseline to week 24 (study arm 2) in patients with baseli | — |
Countries
France, Spain, Turkey, United States
Contacts
Public ContactClinical Trial Information
Regeneron Pharmaceuticals, Inc.
Outcome results
None listed