MEASuRE: Metreleptin Effectiveness And Safety Registry
- Registration Number
- NCT02325674
- Lead Sponsor
- Aegerion Pharmaceuticals, Inc.
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 100
- Patients treated with metreleptin through commercial supply at the time or before enrolment into registry
- Patients who provide a written consent
- Patient coming off metreleptin clinical studies and continuing or restarting treatment with metreleptin through commercial supply
• Patients currently treated with an investigational agent as part of a clinical trial
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Metreleptin Metreleptin Metreleptin new-users Metreleptin prevalent users
- Primary Outcome Measures
Name Time Method The severity and incidence of the following safety events in patients prescribed Metreleptin in routine clinical practice 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. 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.
- Secondary Outcome Measures
Name Time Method Characteristics of the study population in terms of demographic profile, vital signs and clinical signs Demographics and Vital Signs information will be collected at all study visits - min. 10 years Description of the overall demographic and clinical characteristics in all patients treated with metreleptin (pattern of use analysis)
Characteristics of the study population in terms of clinical chemistry - Glycated Hemoglobin (HbA1c), Fasting Plasma Glucose (FPG) and Triglycerides (TG) Clinical chemistry will be collected at all study visits - min. 10 years Description of routine laboratory measurements that could be inferred as efficacy endpoints (including HbA1c, FPG, and TG) over time
Characteristics of the study population in terms of its use of metreleptin Treatment information will be collected at all study visits - min. 10 years Description of the metreleptin exposure information in all patients treated with metreleptin (pattern of use analysis)
Trial Locations
- Locations (27)
Hôpital Robert Debré
🇫🇷Paris, Paris, France, France
University Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
City of Hope
🇺🇸Duarte, California, United States
Atlanta Diabetes Associates
🇺🇸Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of Kansas Medical Center Research Institute, Inc.
🇺🇸Kansas City, Kansas, United States
Children's Hospital of New Orleans/LSU Health Sciences Center
🇺🇸New Orleans, Louisiana, United States
Ochsner Clinic Foundation
🇺🇸New Orleans, Louisiana, United States
Nih/Niddk/Deob
🇺🇸Bethesda, Maryland, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Childrens Hospital of Michigan
🇺🇸Detroit, Michigan, United States
Richmond University Medical Centre
🇺🇸Staten Island, New York, United States
Endocrinology Research Associates
🇺🇸Columbus, Ohio, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
UT Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
CHRU Hôpital Claude Huriez
🇫🇷Lille, Nord, France
Hôpital Saint-Antoine
🇫🇷Paris, France
Universitaetsklinikum Ulm
🇩🇪Ulm, Baden Wuerttemberg, Germany
Universitaetsklinikum Muenster
🇩🇪Muenster, Nordrhein Westfalen, Germany
Universitaetsklinikum Leipzig AoeR
🇩🇪Leipzig, Sachsen, Germany
Universita del Piemonte Orientale "Amedeo Avogadro"
🇮🇹Novara, Verceilli, Italy
Alma Mater Studiorum - Università di Bologna
🇮🇹Bologna, Italy
Azienda Ospedaliero Universitaria Pisana (Presidio di Cisanello)
🇮🇹Pisa, Italy
Azienda Ospedaliera Universitaria Policlinico Tor Vergata
🇮🇹Roma, Italy
Addenbrooke's Hospital
🇬🇧Cambridge, United Kingdom