Clinical trial NCT02325674: MEASuRE: Metreleptin Effectiveness And Safety Registry
Official title: MEASuRE: Metreleptin Effectiveness And Safety Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Generalised Lipodystrophy
Partial Lipodystrophy
Keywords: Lipodystrophy
Metreleptin Registry
Lead sponsor: Aegerion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2014-12-25
Enrollment: 100
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients in the US with Generalised Lipodystrophy treated with commercial Metreleptin, and patients in the EEA with either Generalised Lipodystrophy or Partial Lipodystrophy, treated with commercial Metreleptin

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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

• Patients currently treated with an investigational agent as part of a clinical trial

Primary outcomes:
measure: The severity and incidence of the following safety events in patients prescribed Metreleptin in routine clinical practice
timeFrame: 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.
Secondary outcomes:
measure: Characteristics of the study population in terms of demographic profile, vital signs and clinical signs
timeFrame: Demographics and Vital Signs information will be collected at all study visits - min. 10 years
measure: Characteristics of the study population in terms of clinical chemistry - Glycated Hemoglobin (HbA1c), Fasting Plasma Glucose (FPG) and Triglycerides (TG)
timeFrame: Clinical chemistry will be collected at all study visits - min. 10 years
measure: Characteristics of the study population in terms of its use of metreleptin
timeFrame: Treatment information will be collected at all study visits - min. 10 years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University Alabama at Birmingham
state: Alabama
city: Duarte
country: United States
facility: City of Hope
state: California
city: Atlanta
country: United States
facility: Atlanta Diabetes Associates
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center Research Institute, Inc.
state: Kansas
city: New Orleans
country: United States
facility: Children's Hospital of New Orleans/LSU Health Sciences Center
state: Louisiana
city: New Orleans
country: United States
facility: Ochsner Clinic Foundation
state: Louisiana
city: Bethesda
country: United States
facility: Nih/Niddk/Deob
state: Maryland
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Detroit
country: United States
facility: Childrens Hospital of Michigan
state: Michigan
city: Staten Island
country: United States
facility: Richmond University Medical Centre
state: New York
city: Columbus
country: United States
facility: Endocrinology Research Associates
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Lille
country: France
facility: CHRU Hôpital Claude Huriez
state: Nord
city: Paris
country: France
facility: Hôpital Robert Debré
state: Paris, France
city: Paris
country: France
facility: Hôpital Saint-Antoine
city: Ulm
country: Germany
facility: Universitaetsklinikum Ulm
state: Baden Wuerttemberg
city: Muenster
country: Germany
facility: Universitaetsklinikum Muenster
state: Nordrhein Westfalen
city: Leipzig
country: Germany
facility: Universitaetsklinikum Leipzig AoeR
state: Sachsen
city: Novara
country: Italy
facility: Universita del Piemonte Orientale "Amedeo Avogadro"
state: Verceilli
city: Bologna
country: Italy
facility: Alma Mater Studiorum - Università di Bologna
city: Pisa
country: Italy
facility: Azienda Ospedaliero Universitaria Pisana (Presidio di Cisanello)
city: Roma
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico Tor Vergata
city: Cambridge
country: United Kingdom
facility: Addenbrooke's Hospital
Study officials:
affiliation: Amryt Pharmaceuticals DAC
name: Lori Hartnett
role: STUDY_DIRECTOR
References: