MEASuRE: Metreleptin Effectiveness And Safety Registry (MEASuRE)

January 16, 2026 updated by: Amryt Pharma
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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • France
      • Paris, France, 75012
        • Recruiting
        • Hôpital Saint-Antoine
        • Principal Investigator:
          • Camille Vatier, MD
        • Contact:
          • Camille Vatier
    • Nord
      • Lille, Nord, France, 59800
        • Recruiting
        • CHRU Hôpital Claude Huriez
        • Principal Investigator:
          • Marie-Christine Vantyghem, MD
        • Contact:
          • Marie-Christine Vantyghem
    • Paris, France
      • Paris, Paris, France, France, 75019
        • Recruiting
        • Hopital Robert Debre
        • Contact:
          • Elise Bismuth-Reisman, MD
        • Principal Investigator:
          • Elise Bismuth-Reisman
  • Germany
    • Baden-Wurttemberg
      • Ulm, Baden-Wurttemberg, Germany, 89075
        • Recruiting
        • Universitaetsklinikum Ulm
        • Principal Investigator:
          • Julia Von Schnurbein, MD
        • Contact:
          • Julia Von Schnurbein, MD
    • Lower Saxony
      • Hanover, Lower Saxony, Germany, 0000
        • Recruiting
        • Kinderkrankenhaus Auf der Bult
        • Contact:
          • Olga Kordonouri
        • Principal Investigator:
          • Olga Kordonouri
    • North Rhine-Westphalia
      • Münster, North Rhine-Westphalia, Germany, 48149
        • Recruiting
        • Universitaetsklinikum Muenster
        • Principal Investigator:
          • Elena Vorona, MD
        • Contact:
          • Elena Vorona, MD
    • Saxony
      • Leipzig, Saxony, Germany, 04103
        • Recruiting
        • Universitaetsklinikum Leipzig AoeR
        • Principal Investigator:
          • Konstanze Miehle, MD
        • Contact:
          • Konstanze Miehle, MD
  • Italy
      • Bologna, Italy, 40126
        • Recruiting
        • Alma Mater Studiorum - Universita Di Bologna
        • Contact:
          • Alessandra Gambineri
        • Principal Investigator:
          • Alessandra Gambineri, MD
      • Pisa, Italy, 56124
        • Recruiting
        • Azienda Ospedaliero Universitaria Pisana (Presidio di Cisanello)
        • Principal Investigator:
          • Giovanni Ceccarini, MD
        • Contact:
          • Giovanni Ceccarini, MD
      • Roma, Italy, 00133
        • Withdrawn
        • Azienda Ospedaliera Universitaria Policlinico Tor Vergata
    • Verceilli
      • Novara, Verceilli, Italy, 13100
        • Recruiting
        • Universita del Piemonte Orientale "Amedeo Avogadro"
        • Principal Investigator:
          • Flavia Prodam, MD
        • Contact:
          • Flavia Prodam, MD
  • United Kingdom
      • Cambridge, United Kingdom, CB2 0QQ
        • Recruiting
        • Addenbrooke's Hospital
        • Contact:
          • Anna Stears
        • Principal Investigator:
          • Anna Stears, MD
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 40506
        • Terminated
        • University Alabama at Birmingham
    • California
      • Duarte, California, United States, 91010
        • Terminated
        • City of Hope
    • Georgia
      • Atlanta, Georgia, United States, 30318
        • Active, not recruiting
        • Atlanta Diabetes Associates
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Ann & Robert H. Lurie Children's Hospital of Chicago
        • Principal Investigator:
          • Barbara Burton, MD
        • Contact:
          • Erika Vucko
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • University of Kansas Medical Center Research Institute, Inc.
        • Principal Investigator:
          • Patrick Moriarty, MD
        • Contact:
          • Patrick Moriarty, MD
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
        • Withdrawn
        • Ochsner Clinic Foundation
      • New Orleans, Louisiana, United States, 70118
        • Completed
        • Children's Hospital of New Orleans/LSU Health Sciences Center
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • Recruiting
        • Nih/Niddk/Deob
        • Principal Investigator:
          • Marissa Lightbourne, MD
        • Contact:
          • Marissa Lightbourne, MD
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan
        • Principal Investigator:
          • Elif Oral, MD
        • Contact:
          • Elif Oral, MD
      • Detroit, Michigan, United States, 48201
        • Recruiting
        • Childrens Hospital of Michigan
        • Contact:
          • Vinood Misra
        • Principal Investigator:
          • Vinood Misra, MD
    • New York
      • Staten Island, New York, United States, 10314
        • Completed
        • Richmond University Medical Centre
    • Ohio
      • Columbus, Ohio, United States, 43201
        • Recruiting
        • Endocrinology Research Associates
        • Principal Investigator:
          • Elena Christofides, MD
        • Contact:
          • Elena Christofides, MD
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
        • Principal Investigator:
          • Shana McCormack, MD
        • Contact:
          • Shana McCormack, MD
      • Philadelphia, Pennsylvania, United States, 19104
        • Withdrawn
        • University of Pennsylvania
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • UT Southwestern Medical Center
        • Principal Investigator:
          • Abhimanyu Garg, MD
        • Contact:
          • Abhimanyu Garg, MD
    • Washington
      • Seattle, Washington, United States, 98105
        • Recruiting
        • Seattle Children's Hospital
        • Principal Investigator:
          • Helen Dichek, MD
        • Contact:
          • Helen Dichek, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Metreleptin

Metreleptin new-users

Metreleptin prevalent users
Drug: Metreleptin

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
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.
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

Outcome Measure
Measure Description
Time Frame
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
Description of the overall demographic and clinical characteristics in all patients treated with metreleptin (pattern of use analysis)
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
Description of routine laboratory measurements that could be inferred as efficacy endpoints (including HbA1c, FPG, and TG) over time
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
Description of the metreleptin exposure information in all patients treated with metreleptin (pattern of use analysis)
Treatment information will be collected at all study visits - min. 10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Amryt Pharma

Investigators

  • Study Director: Lori Hartnett, Amryt Pharmaceuticals DAC

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 11, 2016

Primary Completion (Estimated)

October 31, 2031

Study Completion (Estimated)

October 31, 2031

Study Registration Dates

First Submitted

December 22, 2014

First Submitted That Met QC Criteria

December 22, 2014

First Posted (Estimated)

December 25, 2014

Study Record Updates

Last Update Posted (Actual)

January 20, 2026

Last Update Submitted That Met QC Criteria

January 16, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AEGR-734-400

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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