Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL

Phase 3Active Not RecruitingNCT05164341

Amryt Pharma69 enrolled

Overview

This is a Phase III, double-blind, placebo-controlled, safety and efficacy study of daily SC metreleptin in subjects with Partial Lipodystrophy.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Partial Lipodystrophy

Interventions

metreleptinDRUG

Metreleptin is a recombinant human leptin analog that is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency

PlaceboDRUG

Placebo

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Diagnosis of Familial Partial Lipodystrophy (FPLD) * Subjects with poor metabolic control defined as: HbA1c ≥7% (at Visit 1 and Visit 3) and/or Fasting TGs ≥500 mg/dL (5.65 mmol/L, at Visit 1 and Visit 3) * Patients should be receiving optimized stable therapy Exclusion Criteria: * Previous treatment with metreleptin * Leptin levels \>20.0 ng/mL * Acquired or radiation induced partial lipodystrophy (APL) Other protocol defined inclusion/exclusion criteria apply

Locations (31)

Outcomes

Primary Outcomes

Change from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL)

To evaluate the efficacy (HbAa1c) of daily SC metreleptin treatment

Time frame: 6 months

Percent change from Baseline to month 6 in fasting TGs in subjects with partial lipodystrophy (PL)

To evaluate the efficacy (TGs) of daily SC metreleptin treatment

Time frame: 6 months

Secondary Outcomes

Safety analysis of AEs, AESIs, SAEs by treatment arm

To evaluate the safety of daily SC metreleptin treatment in subjects with PL

Time frame: 12 months

Change from baseline to months 9 and 12 in HbA1c in subjects with partial lipodystrophy (PL)

To assess the effect of metreleptin on HbA1c

Time frame: 12 months

Percent change from baseline to months 9 and 12 in fasting TGs in subjects with partial lipodystrophy (PL)

To assess the effect of metreleptin on TGs

Time frame: 12 months

Change from Baseline to each assessment time point in quality of life (QoL) in all subjects

To assess the effect of metreleptin on quality of life (QoL) in all subjects

Time frame: 12 months

Data from ClinicalTrials.gov

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University of Alabama

Birmingham, Alabama, United States

UC Davis

Sacramento, California, United States

Flourish Boca Raton

Boca Raton, Florida, United States

Emory University

Atlanta, Georgia, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

University of Michigan

Ann Arbor, Michigan, United States

Mayo Clinic

Rochester, Minnesota, United States

University of Missouri

Columbia, Missouri, United States

Amryt Research Site, Endocrinology Research Associates Inc

Columbus, Ohio, United States

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

...and 21 more locations