Table 1 Primary endpoints, secondary endpoints and exploratory endpoints collected for MEASuRE
From: The Metreleptin Effectiveness and Safety Registry (MEASuRE): concept, design and challenges
Primary objectives | To determine the incidence and severity of the following safety events in patients prescribed metreleptin in normal clinical practice: • Acute pancreatitis associated with the discontinuation of metreleptin; and all cases of fatal or necrotizing pancreatitis • Hepatic adverse events • Hypoglycemia 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 ADAs with blocking activity • New diagnoses of autoimmune disorders (for instance, autoimmune hepatitis, glomerulonephritis, lupus erythematosus, antiphospholipid antibody syndrome, rheumatoid arthritis) • 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 objectives | • To describe the overall demographic and clinical characteristics, and metreleptin exposure in all patients treated with metreleptin (pattern of metreleptin use) • 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, estradiol, LH, FSH and free T3 and T4 • 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 |