MEASuRE: Metreleptin Effectiveness And Safety Registry
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Metreleptin.
- Who it may be relevant to
- Registry conditions: Generalised Lipodystrophy, Partial Lipodystrophy. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, France, Germany, Italy, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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.
Interventions
- Drug Metreleptin
Primary outcome measures
- The severity and incidence of the following safety events in patients prescribed Metreleptin in routine clinical practice [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.]
Secondary outcome measures (3)
- Characteristics of the study population in terms of demographic profile, vital signs and clinical signs [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) [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 [Time frame: Treatment information will be collected at all study visits - min. 10 years]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 16 centers
- University Alabama at Birmingham — Birmingham
- City of Hope — Duarte
- Atlanta Diabetes Associates — Atlanta
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
- University of Kansas Medical Center Research Institute, Inc. — Kansas City
- Children's Hospital of New Orleans/LSU Health Sciences Center — New Orleans
- Ochsner Clinic Foundation — New Orleans
- Nih/Niddk/Deob — Bethesda
- … and 8 more centers
Germany · 4 centers
- Universitaetsklinikum Ulm — Ulm
- Kinderkrankenhaus Auf der Bult — Hanover
- Universitaetsklinikum Muenster — Münster
- Universitaetsklinikum Leipzig AoeR — Leipzig
Italy · 4 centers
- Alma Mater Studiorum - Università di Bologna — Bologna
- Azienda Ospedaliero Universitaria Pisana (Presidio di Cisanello) — Pisa
- Azienda Ospedaliera Universitaria Policlinico Tor Vergata — Roma
- Universita del Piemonte Orientale "Amedeo Avogadro" — Novara
France · 3 centers
- CHRU Hôpital Claude Huriez — Lille
- Hôpital Robert Debré — Paris
- Hôpital Saint-Antoine — Paris
United Kingdom · 1 center
- Addenbrooke's Hospital — Cambridge
Publications
- Haymond MW, Araujo-Vilar D, Balser J, Lewis JH, Louzado R, Musso C, von Schnurbein J, Wabitsch M; MEASuRE group. The Metreleptin Effectiveness and Safety Registry (MEASuRE): concept, design and challenges. Orphanet J Rare Dis. 2023 May 26;18(1):127. doi: 10.1186/s13023-023-02714-5. PMID 37237416
Identifiers
NCT: NCT02325674 · AEGR-734-400