MEASuRE: Metreleptin Effectiveness And Safety Registry

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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MEASuRE: Metreleptin Effectiveness And Safety Registry

Conditions
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Generalised Lipodystrophy, Partial Lipodystrophy

Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Metreleptin<br>Metreleptin new-users Metreleptin prevalent users | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The severity and incidence of the following safety events in patients presribed 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. | Adverse event will be collected from first dose 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 | 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) | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Lipodystrophy, Metreleptin Registry