MEASuRE: Metreleptin Effectiveness And Safety Registry
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Chiesi Farmaceutici S.p.A.
- Enrollment
- 100
- Locations
- 46
- Primary Endpoint
- The severity and incidence of the following safety events in patients prescribed Metreleptin in routine clinical practice
Study Overview
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.
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 Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Metreleptin
Metreleptin new-users
Metreleptin prevalent users
Intervention: Metreleptin (Drug)
Outcomes
Primary Outcomes
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.
Secondary Outcomes
- 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)