MEASuRE: Metreleptin Effectiveness and Safety Registry

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  MYALEPTA
• Study drug International non-proprietary name (INN) or common name:
  METRELEPTIN
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AA07) metreleptin

  metreleptin
• Medical condition to be studied:
  Partial lipodystrophy

  Congenital generalised lipodystrophy

  Acquired generalised lipodystrophy

Population studied

• Short description of the study population:
  All patients treated with metreleptin through commercial supply and enrolled in the registry will be categorized into one of two patient cohorts: metreleptin new user cohort or metreleptin prevalent user cohort.; • Metreleptin new user cohort includes patients who are initiating treatment with metreleptin through commercial supply at the time of providing written consent for registry enrolment.; • Metreleptin prevalent (prevalent current and prevalent prior) user cohort includes patients who are treated with metreleptin through commercial supply but before registry enrolment and/or patients treated with metreleptin through commercial supply but coming off metreleptin treatment from a non-commercial source (e.g., clinical studies).
• Estimated number of subjects:
  100

Study design details

• Study design:
  Prospective, non-interventional, observational registry of patients treated with metreleptin in routine clinical practice across US and EEA.
• Main study objective:
  Evaluate long-term safety and effectiveness of metreleptin under real-world conditions ; ; Primary: ; ; To determine the incidence and severity of the following safety events in patients treated with metreleptin as part of standard clinical practice: ; ; • Acute pancreatitis associated with the discontinuation of metreleptin, and all cases of fatal or necrotizing pancreatitis ; ; • Hepatic adverse events ; ; • Hypoglycaemia stratified by severity and concomitant antidiabetics dose modifications ; ; • Hypersensitivity reactions ; ; • Serious and severe infections, including serious infections resulting in hospitalization and death ; ; Loss of efficacy, potentially due to anti-drug antibodies with blocking activity ; ; New diagnoses of autoimmune disorders ; ; Exacerbation of existing autoimmune disorders ; ; All cancers (excluding non-melanoma skin cancer) by cancer type ; ; Exposed pregnancies and pregnancy outcomes stratified by planned or unplanned ; ; All-cause deaths (including causes of death) ; ; Medication errors ; ; Secondary ; ; • To describe the overall demographic and clinical characteristics, and metreleptin exposure in all patients treated with metreleptin (pattern of use analysis) ; ; • To describe routine laboratory measurements that could be inferred as effectiveness endpoints (including HbA1c, FPG and TG) over time ; ; Exploratory Objectives ; ; Use in pregnancy and lactation ; ; Use in elderly ; ; Effect of metreleptin on brain development ; ; Effect of metreleptin on bone metabolism ; ; Effect of metreleptin on sexual maturation (Tanner staging) ; ; Neuroendocrine parameters and levels of the following hormones: ; ; Testosterone, Oestradiol, Luteinizing hormone , Follicle stimulating hormone and Free triiodothyronine and thyroxine. Contingent on study sample size, the study will also estimate the incidence rate of the primary outcomes of interest by patient characteristics. ; ; In patients with results from immunogenicity testing, the incidence of ADAs with blocking activity will be estimated.
• Setting:
  Routine clinical practice across US and EEA, using standard-of-care visits and medical record abstraction
• Data analysis plan:
  The primary objective of this study is to estimate the incidence of primary outcomes of interest in the patients treated with metreleptin. A confidence interval approach is chosen for the rate of the events of interest. ; ; The primary population for analysis will consist of all patients who have received at least one dose of commercial metreleptin, regardless of the indication for which the treatment is received. ; ; Data will be analysed by subgroups; metreleptin new user cohort, metreleptin prevalent user cohort; by indication, GL and PL and if required by region, US and EEA. In addition, analyses of Paediatric cohorts and further sub-group analyses may be performed.
• Summary results:
  Not applicable (ongoing registry)