MYALEPT™ (metreleptin) has been approved as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (MYALEPT Prescribing Information). This study is a multicenter, open-label, Phase 4 trial to provide an assessment of the immunogenicity associated with metreleptin and of any major potential risks due to development of antibodies to metreleptin. The study is being conducted to comply with a postmarketing requirement.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
11
Subjects will receive prescribed dosage of metreleptin as indicated in the USPI
Univ. Alabama-Birmingham
Birmingham, Alabama, United States
Ochsner Clinic
New Orleans, Louisiana, United States
University of Michigan
Ann Arbor, Michigan, United States
Endocrinology Research Associates
Columbus, Ohio, United States
Evaluate the Immunogenicity Associated With Daily Subcutaneous (SC) Metreleptin Treatment in Patients With Congenital Generalized Lipodystrophy (CGL) or Acquired Generalized Lipodystrophy (AGL).
Anti-metreleptin, anti-human leptin (HuL) binding antidrug antibody (ADA) titers over time. Category of in vitro cell-based neutralizing antibody (NAb) activity to metreleptin, and titer in the receptor blocking (RB) NAb assay in ADA positive samples over time.
Time frame: 36 months
Assess 2 Methods of Measuring in Vitro NAb Activity to Metreleptin.
The percent (standard error) of patients with a positive result was compared for the RB NAb assay and the cell-based NAb assay over time.
Time frame: 36 months
Evaluate the Safety and Tolerability in Relation to the Development of or Absence of Anti-metreleptin and Anti-HuL Binding ADAs, and/or in Vitro NAb Activity to Metreleptin in Patients With CGL or AGL
Serious adverse events (SAEs), AEs leading to discontinuation, loss of response (as assessed by glycated hemoglobin (HbA1c) and serum triglycerides), severe infections and/or sepsis, standard laboratory tests, and vital signs over time.
Time frame: 36 months
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Ohio State University
Columbus, Ohio, United States
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University Texas Southwestern INT
Dallas, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States