NCT02325674

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.

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
100

participants targeted

Target at P50-P75 for all trials

Timeline
66mo left

Started Oct 2016

Longer than P75 for all trials

Geographic Reach
5 countries

28 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress64%
Oct 2016Oct 2031

First Submitted

Initial submission to the registry

December 22, 2014

Completed
3 days until next milestone

First Posted

Study publicly available on registry

December 25, 2014

Completed
1.8 years until next milestone

Study Start

First participant enrolled

October 11, 2016

Completed
15.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2031

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2031

Last Updated

January 20, 2026

Status Verified

January 1, 2026

Enrollment Period

15.1 years

First QC Date

December 22, 2014

Last Update Submit

January 16, 2026

Conditions

Generalised LipodystrophyPartial Lipodystrophy

Keywords

LipodystrophyMetreleptin Registry

Outcome Measures

Primary Outcomes (1)

  • 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.

    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

    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)

    Clinical chemistry will be collected at all study visits - min. 10 years

  • Characteristics of the study population in terms of its use of metreleptin

    Treatment information will be collected at all study visits - min. 10 years

Study Arms (1)

Metreleptin

Metreleptin new-users Metreleptin prevalent users

Drug: Metreleptin

Interventions

MetreleptinDRUG
Metreleptin

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-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

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (28)

University Alabama at Birmingham

Birmingham, Alabama, 40506, United States

TERMINATED

City of Hope

Duarte, California, 91010, United States

TERMINATED

Atlanta Diabetes Associates

Atlanta, Georgia, 30318, United States

ACTIVE NOT RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

RECRUITING

University of Kansas Medical Center Research Institute, Inc.

Kansas City, Kansas, 66160, United States

RECRUITING

Children's Hospital of New Orleans/LSU Health Sciences Center

New Orleans, Louisiana, 70118, United States

COMPLETED

Ochsner Clinic Foundation

New Orleans, Louisiana, 70121, United States

WITHDRAWN

Nih/Niddk/Deob

Bethesda, Maryland, 20892, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

Childrens Hospital of Michigan

Detroit, Michigan, 48201, United States

RECRUITING

Richmond University Medical Centre

Staten Island, New York, 10314, United States

COMPLETED

Endocrinology Research Associates

Columbus, Ohio, 43201, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

WITHDRAWN

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

CHRU Hôpital Claude Huriez

Lille, Nord, 59800, France

RECRUITING

Hôpital Robert Debré

Paris, Paris, France, 75019, France

RECRUITING

Hôpital Saint-Antoine

Paris, 75012, France

RECRUITING

Universitaetsklinikum Ulm

Ulm, Baden-Wurttemberg, 89075, Germany

RECRUITING

Kinderkrankenhaus Auf der Bult

Hanover, Lower Saxony, 0000, Germany

RECRUITING

Universitaetsklinikum Muenster

Münster, North Rhine-Westphalia, 48149, Germany

RECRUITING

Universitaetsklinikum Leipzig AoeR

Leipzig, Saxony, 04103, Germany

RECRUITING

Universita del Piemonte Orientale "Amedeo Avogadro"

Novara, Verceilli, 13100, Italy

RECRUITING

Alma Mater Studiorum - Università di Bologna

Bologna, 40126, Italy

RECRUITING

Azienda Ospedaliero Universitaria Pisana (Presidio di Cisanello)

Pisa, 56124, Italy

RECRUITING

Azienda Ospedaliera Universitaria Policlinico Tor Vergata

Roma, 00133, Italy

WITHDRAWN

Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom

RECRUITING

Related Publications (1)

  • 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: 37237416DERIVED

MeSH Terms

Conditions

Lipodystrophy, Congenital GeneralizedLipodystrophy, Partial, AcquiredLipodystrophy

Interventions

metreleptin

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin Diseases, MetabolicSkin DiseasesSkin and Connective Tissue DiseasesLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Lori Hartnett

    Amryt Pharmaceuticals DAC

    STUDY DIRECTOR

Central Study Contacts

Lori Hartnett

CONTACT

+3905212791clinicaltrials_info@chiesi.com

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
10 Years
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 22, 2014

First Posted

December 25, 2014

Study Start

October 11, 2016

Primary Completion (Estimated)

October 31, 2031

Study Completion (Estimated)

October 31, 2031

Last Updated

January 20, 2026

Record last verified: 2026-01

Locations

FR(3)IT(4)GB(1)US(16)DE(4)