Study type

Study type

Non-interventional study

Scope of the study

Assessment of risk minimisation measure implementation or effectiveness
Drug utilisation
Effectiveness study (incl. comparative)
Safety study (incl. comparative)
Non-interventional study

Non-interventional study design

Cohort
Other

Non-interventional study design, other

This registry study is a multi-centre, long-term, prospective, observational cohort study
Study drug and medical condition

Name of medicine

LOJUXTA

Name of medicine, other

Juxtapid

Study drug International non-proprietary name (INN) or common name

LOMITAPIDE

Anatomical Therapeutic Chemical (ATC) code

(C10AX12) lomitapide
lomitapide

Medical condition to be studied

Type IIa hyperlipidaemia

Additional medical condition(s)

MedDRA code for homozygous familial hypercholesterolaemia (HoFH) is 10057100
Population studied

Age groups

Preterm newborn infants (0 – 27 days)
Term newborn infants (0 – 27 days)
Infants and toddlers (28 days – 23 months)
Children (2 to < 12 years)
Adolescents (12 to < 18 years)
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)

Special population of interest

Hepatic impaired
Renal impaired

Estimated number of subjects

300
Study design details

Main study objective

The registry is designed to evaluate the long-term safety and effectiveness of lomitapide in clinical practice. Objectives: To evaluate the occurrence of the following in patients treated with lomitapide: Hepatic abnormalities, GI events, Small bowel, hepatic, colorectal and pancreatic tumours, Events associated with coagulopathy, MACE events, Death (including cause of death) and Pregnancy.

Outcomes

The safety-related events of special interest include: Hepatic abnormalities, GI events, Tumours, Events associated with coagulopathy, Other safety events of interest include: MACE events, Death and cause of death, Pregnancy (both prospectively and retrospectively reported pregnancies will be collected). Effectiveness evaluations: Magnitude of reduction in serum LDL-C from baseline, Absolute and percent change from baseline in total cholesterol, non-HDL-C, apolipoprotein B (apo B), triglycerides (TG), lipoprotein a (Lp(a)), HDL-C, apolipoprotein AI (apo- AI) and very-low-density lipoprotein cholesterol (VLDL-C), Changes in concomitant medications or apheresis treatments.

Data analysis plan

The main objectives of the study are to evaluate the long-term safety and effectiveness of lomitapide under conditions of usual clinical practice, and to evaluate the effectiveness of risk minimisation interventions in mitigating the serious risks of lomitapide in countries where such efforts are in place. Summary tabulations will be presented that will display the number of observations, mean, standard deviation, median, minimum and maximum for continuous variables and the number and percentage per category for categorical or ordered categorical data. In addition, two-sided 95% confidence intervals will be calculated for all outcomes.