LOWER: Lomitapide Observational Worldwide Evaluation Registry

Study Overview
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Brief Summary
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This global product exposure registry is a multicentre, long-term, prospective, observational cohort study (exposure registry), designed to evaluate the long term safety and effectiveness of lomitapide.

Detailed Description
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To evaluate the occurrence of adverse events of special interest, long term effectiveness of lomitapide, and to evaluate whether prescribers of lomitapide are following screening and monitoring recommendations as specified in product labeling.

Official Title
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LOWER: Lomitapide Observational Worldwide Evaluation Registry

Conditions
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Homozygous Familial Hypercholesterolemia

Intervention / Treatment
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* Drug: Lomitapide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients who have initiated treatment with lomitapide at the time of registry enrolment. Patients who have the ability to understand the requirements of the study and provide written informed consent to comply with the study data collection procedures. Exclusion Criteria: Patients who are receiving lomitapide in clinical trials Patients receiving an investigational agent, defined as any drug or biologic agent other than lomitapide that has not received MA in the country of participation, at time of enrollment.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Lomitapide<br>Lomitapide as prescribed by Physician. | Drug: Lomitapide<br>* As prescribed by Physician.<br>* Other names: Lojuxta;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hepatic Abnormalities | to evaluate the occurrence of hepatic abnormalities | patients will be followed for 10 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Gastrointestinal (GI) Events | to evaluate the occurrence of GI events | patients will be followed for 10 years | 
| Tumors | to evaluate any occurrence of tumors (small bowel, hepatic, colorectal or pancreatic) | patients will be followed for 10 years | 
| Events associated with coagulopathy | to evaluate the occurrence of events associated with coagulopathy (abnormal bleeding, cerebral haemorrhage or GI bleeding) | patients will be followed for 10 years | 
| Major Adverse Cardiovascular Events (MACE) events | to evaluate the occurrence of MACE events | patients will be followed for 10 years | 
| Death, including cause of death | to evaluate the occurrence and cause of death | patients will be followed for 10 years | 
| Pregnancy | to evaluate the occurrence and outcomes of pregnancy in females of reproductive potential treated with lomitapide. Patients who become pregnant will be offered enrolment into a separate Pregnancy Exposure Registry (PER). | patients will be followed for 10 years | 
| Serum lipid levels | to evaluate the long-term effectiveness of lomitapide in maintaining control of serum lipid levels in a clinical practice setting. | patients will be followed for 10 years | 
| Prescriber behavior | to evaluate whether prescribers of lomitapide enrolled at registry sites are following the screening and monitoring recommendations as specified in the PI and the prescriber educational materials aimed at risk minimization. | patients will be followed for 10 years |