Generalised LipodystrophyMEASuRENCT02325674
MEASuRE: Metreleptin Effectiveness And Safety Registry
An observational study of Generalised Lipodystrophy and Partial Lipodystrophy, run by Chiesi Farmaceutici S.p.A. at 28 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Generalised Lipodystrophy, Partial Lipodystrophy
- What is being tested?
- Metreleptin
- How many people
- 100 participants
- Where
- 28 sitesin 5 countries
- Study dates
- 11 Oct 2016to 31 Oct 2031
- How long will I take part?
- 10 Years
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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
Who may be able to join
- 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
Who cannot join
- Patients currently treated with an investigational agent as part of a clinical trial
Trial locations and contacts
28 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
28 sites in 5 countries
United States16 sites, 9 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
Elif Oral, MD
Elif Oral, MD
- Completed
Atlanta Diabetes Associates
Atlanta, Georgia, 30318
- Recruiting
Nih/Niddk/Deob
Bethesda, Maryland, 20892
Marissa Lightbourne, MD
Marissa Lightbourne, MD
- Terminated
University Alabama at Birmingham
Birmingham, Alabama, 40506
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Erika Vucko
Barbara Burton, MD
- Recruiting
Endocrinology Research Associates
Columbus, Ohio, 43201
Elena Christofides, MD
Elena Christofides, MD
- Recruiting
UT Southwestern Medical Center
Dallas, Texas, 75390
Abhimanyu Garg, MD
Abhimanyu Garg, MD
- Recruiting
Childrens Hospital of Michigan
Detroit, Michigan, 48201
Vinood Misra
Vinood Misra, MD
- Terminated
City of Hope
Duarte, California, 91010
- Recruiting
University of Kansas Medical Center Research Institute, Inc.
Kansas City, Kansas, 66160
Patrick Moriarty, MD
Patrick Moriarty, MD
- Completed
Children's Hospital of New Orleans/LSU Health Sciences Center
New Orleans, Louisiana, 70118
- Withdrawn
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Shana McCormack, MD
Shana McCormack, MD
- Withdrawn
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Helen Dichek, MD
Helen Dichek, MD
- Completed
Richmond University Medical Centre
Staten Island, New York, 10314
Germany4 sites, 4 recruiting
- Recruiting
Kinderkrankenhaus Auf der Bult
Hanover, Lower Saxony, 0000
Olga Kordonouri
Olga Kordonouri
- Recruiting
Universitaetsklinikum Leipzig AoeR
Leipzig, Saxony, 04103
Konstanze Miehle, MD
Konstanze Miehle, MD
- Recruiting
Universitaetsklinikum Muenster
Münster, North Rhine-Westphalia, 48149
Elena Vorona, MD
Elena Vorona, MD
- Recruiting
Universitaetsklinikum Ulm
Ulm, Baden-Wurttemberg, 89075
Julia Von Schnurbein, MD
Julia Von Schnurbein, MD
Italy4 sites, 3 recruiting
- Recruiting
Alma Mater Studiorum - Università di Bologna
Bologna, Bologna, 40126
Alessandra Gambineri
Alessandra Gambineri, MD
- Recruiting
Universita del Piemonte Orientale "Amedeo Avogadro"
Novara, Verceilli, 13100
Flavia Prodam, MD
Flavia Prodam, MD
- Recruiting
Azienda Ospedaliero Universitaria Pisana (Presidio di Cisanello)
Pisa, Pisa, 56124
Giovanni Ceccarini, MD
Giovanni Ceccarini, MD
- Withdrawn
Azienda Ospedaliera Universitaria Policlinico Tor Vergata
Roma, Roma, 00133
France3 sites, 3 recruiting
- Recruiting
CHRU Hôpital Claude Huriez
Lille, Nord, 59800
Marie-Christine Vantyghem
Marie-Christine Vantyghem, MD
- Recruiting
Hôpital Robert Debré
Paris, Paris, France, 75019
Elise Bismuth-Reisman, MD
Elise Bismuth-Reisman
- Recruiting
Hôpital Saint-Antoine
Paris, Paris, 75012
Camille Vatier
Camille Vatier, MD
United Kingdom1 site, 1 recruiting
- Recruiting
Addenbrooke's Hospital
Cambridge, Cambridge, CB2 0QQ
Anna Stears
Anna Stears, MD
About this study
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.
Read the 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.
- Conditions
- Generalised Lipodystrophy, Partial Lipodystrophy
- Being tested
- Metreleptin
- Keywords
- Lipodystrophy, Metreleptin Registry
- Medical subject headings
- Lipodystrophy, Congenital Generalized, Lipodystrophy, Partial, Acquired, Lipodystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetreleptinDrug
| Study group | Role | What they receive |
|---|---|---|
| Metreleptin | Metreleptin new-users
Metreleptin prevalent users Receives: Drug: Metreleptin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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 (3)
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)
Measured: 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
Measured: 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)
Measured: Treatment information will be collected at all study visits - min. 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Dec 2014 (estimated)
- Study start11 Oct 2016
- Primary completion31 Oct 2031 (estimated)
- Study completion31 Oct 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Generalised LipodystrophyStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lori Hartnett, Study director, Amryt Pharmaceuticals DAC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02325674
- Sponsor's protocol ID
- AEGR-734-400
- Acronym
- MEASuRE
- Status last verified
- Jul 2026
- First submitted
- 22 Dec 2014
- Last updated
- 16 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
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.
PubMed 37237416