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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Metreleptin | Metreleptin new-users Metreleptin prevalent users |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Metreleptin | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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) | Demographics and Vital Signs information will be collected at all study visits - min. 10 years |
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Inclusion Criteria:
Exclusion Criteria:
• Patients currently treated with an investigational agent as part of a clinical trial
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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
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Lori Hartnett | Contact | +3905212791 | clinicaltrials_info@chiesi.com |
| Name | Affiliation | Role |
|---|---|---|
| Lori Hartnett | Amryt Pharmaceuticals DAC | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Alabama at Birmingham | Terminated | Birmingham | Alabama | 40506 | United States | |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37237416 | Derived | 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. |
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| 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 |
| 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) | Treatment information will be collected at all study visits - min. 10 years |
| City of Hope |
| Terminated |
| Duarte |
| California |
| 91010 |
| United States |
| Atlanta Diabetes Associates | Active, not recruiting | Atlanta | Georgia | 30318 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Recruiting | Chicago | Illinois | 60611 | United States |
|
| University of Kansas Medical Center Research Institute, Inc. | Recruiting | Kansas City | Kansas | 66160 | United States |
|
| Children's Hospital of New Orleans/LSU Health Sciences Center | Completed | New Orleans | Louisiana | 70118 | United States |
| Ochsner Clinic Foundation | Withdrawn | New Orleans | Louisiana | 70121 | United States |
| Nih/Niddk/Deob | Recruiting | Bethesda | Maryland | 20892 | United States |
|
| University of Michigan | Recruiting | Ann Arbor | Michigan | 48109 | United States |
|
| Childrens Hospital of Michigan | Recruiting | Detroit | Michigan | 48201 | United States |
|
| Richmond University Medical Centre | Completed | Staten Island | New York | 10314 | United States |
| Endocrinology Research Associates | Recruiting | Columbus | Ohio | 43201 | United States |
|
| Children's Hospital of Philadelphia | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
|
| University of Pennsylvania | Withdrawn | Philadelphia | Pennsylvania | 19104 | United States |
| UT Southwestern Medical Center | Recruiting | Dallas | Texas | 75390 | United States |
|
| Seattle Children's Hospital | Recruiting | Seattle | Washington | 98105 | United States |
|
| CHRU Hôpital Claude Huriez | Recruiting | Lille | Nord | 59800 | France |
|
| Hôpital Robert Debré | Recruiting | Paris | Paris, France | 75019 | France |
|
| Hôpital Saint-Antoine | Recruiting | Paris | 75012 | France |
|
| Universitaetsklinikum Ulm | Recruiting | Ulm | Baden-Wurttemberg | 89075 | Germany |
|
| Kinderkrankenhaus Auf der Bult | Recruiting | Hanover | Lower Saxony | 0000 | Germany |
|
| Universitaetsklinikum Muenster | Recruiting | Münster | North Rhine-Westphalia | 48149 | Germany |
|
| Universitaetsklinikum Leipzig AoeR | Recruiting | Leipzig | Saxony | 04103 | Germany |
|
| Universita del Piemonte Orientale "Amedeo Avogadro" | Recruiting | Novara | Verceilli | 13100 | Italy |
|
| Alma Mater Studiorum - Università di Bologna | Recruiting | Bologna | 40126 | Italy |
|
| Azienda Ospedaliero Universitaria Pisana (Presidio di Cisanello) | Recruiting | Pisa | 56124 | Italy |
|
| Azienda Ospedaliera Universitaria Policlinico Tor Vergata | Withdrawn | Roma | 00133 | Italy |
| Addenbrooke's Hospital | Recruiting | Cambridge | CB2 0QQ | United Kingdom |
|
| ID | Term |
|---|---|
| D052497 | Lipodystrophy, Congenital Generalized |
| C562448 | Lipodystrophy, Partial, Acquired |
| D008060 | Lipodystrophy |
| ID | Term |
|---|---|
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012875 | Skin Diseases, Metabolic |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C415771 | metreleptin |
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