Global Lomitapide (Juxtapid and Lojuxta) Pregnancy Exposure Registry (PER)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Safety Registry

Study drug and medical condition

• Medical condition to be studied:
  Type IIa hyperlipidaemia

Population studied

• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  30

Study design details

• Main study objective:
  To evaluate the outcomes of pregnancy in women treated with lomitapide at any time within 30 days prior to first day of Last Menstrual Period (LMP) or during pregnancy. The outcomes of primary interest are major congenital anomalies.
• Outcomes:
  The outcomes of primary interest are major congenital anomalies.
• Data analysis plan:
  A formal statistical analysis plan (SAP) will include details of all planned analyses and presentation of PER data. Since this is an observational study, descriptive analyses will be provided. Descriptive statistics will comprise the number of observations (n), mean, standard deviation (SD), median, minimum, and maximum for continuous variables, and n and percent for categorical variables. Data will be presented for all patients enroled in the PER. An analysis of all PER participants combined will be provided. Separate analyses will also be conducted for prospective and retrospective reports. All data will be pooled for an additional analysis that includes spontaneous and literature reports from patients not participating in the PER. The proportion of pregnancies and births that have been lost to follow-up will also be calculated and reported. The primary outcome is major congenital abnormalities.