The study is a post-authorization, prospective, voluntary registry of patients treated with commercial metreleptin including, but not limited to, patients in the US and EEA.
This is a non-interventional, multicentre, prospective, observational study of patients receiving treatment with commercial metreleptin for lipodystrophy in the US (GL) and EEA (GL and PL). This registry will add to the knowledge about metreleptin gained from clinical trials by providing information on the incidence rates of acute pancreatitis associated with the discontinuation of metreleptin; and all cases of fatal or necrotizing pancreatitis, hepatic adverse events, hypoglycemia, hypersensitivity reactions, serious and severe infections, including serious infections resulting in hospitalization and death, loss of efficacy, new diagnoses of autoimmune disorders, exacerbation of existing autoimmune disorders, all cancers (excluding non-melanoma skin cancer) by cancer type, exposed pregnancies, and all-cause deaths, in patients treated with metreleptin in routine clinical practice.
Study Type
OBSERVATIONAL
Enrollment
100
The severity and incidence of the following safety events in patients prescribed Metreleptin in routine clinical practice
Estimation of the incidence rates of the following events of special interest in patients treated with metreleptin as part of current clinical practice: acute pancreatitis associated with discontinuation of metreleptin including fatal or necrotizing pancreatitis, hepatic adverse events, severe hypoglycemia, serious hypersensitivity reactions, serious and severe infections resulting in hospitalization or death, loss of efficacy, new diagnoses of autoimmune disorders, autoimmune disease exacerbation, all cancers, exposed pregnancies and pregnancy outcomes, all-cause deaths, medication errors.
Time frame: Adverse events will be collected from first dose to last visit - min. 10 years. For Prevalent Users: Serious adverse events will be collected from 6 months prior to enrolment to last visit - min. 10 years.
Characteristics of the study population in terms of demographic profile, vital signs and clinical signs
Description of the overall demographic and clinical characteristics in all patients treated with metreleptin (pattern of use analysis)
Time frame: Demographics and Vital Signs information will be collected at all study visits - min. 10 years
Characteristics of the study population in terms of clinical chemistry - Glycated Hemoglobin (HbA1c), Fasting Plasma Glucose (FPG) and Triglycerides (TG)
Description of routine laboratory measurements that could be inferred as efficacy endpoints (including HbA1c, FPG, and TG) over time
Time frame: Clinical chemistry will be collected at all study visits - min. 10 years
Characteristics of the study population in terms of its use of metreleptin
Description of the metreleptin exposure information in all patients treated with metreleptin (pattern of use analysis)
Time frame: Treatment information will be collected at all study visits - min. 10 years
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University Alabama at Birmingham
Birmingham, Alabama, United States
TERMINATEDCity of Hope
Duarte, California, United States
TERMINATEDAtlanta Diabetes Associates
Atlanta, Georgia, United States
ACTIVE_NOT_RECRUITINGAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
RECRUITINGUniversity of Kansas Medical Center Research Institute, Inc.
Kansas City, Kansas, United States
RECRUITINGChildren's Hospital of New Orleans/LSU Health Sciences Center
New Orleans, Louisiana, United States
COMPLETEDOchsner Clinic Foundation
New Orleans, Louisiana, United States
WITHDRAWNNih/Niddk/Deob
Bethesda, Maryland, United States
RECRUITINGUniversity of Michigan
Ann Arbor, Michigan, United States
RECRUITINGChildrens Hospital of Michigan
Detroit, Michigan, United States
RECRUITING...and 18 more locations