Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy

Study Overview
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Brief Summary
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Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy.

Detailed Description
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This is a Phase 3, single-arm, open-label, multicenter clinical trial to evaluate both the efficacy and long-term safety of lomitapide in Japanese patients with HoFH receiving maximally-tolerated, stable lipid-lowering therapy. This study is comprised of a run-in period, a primary 26-week Efficacy Phase, and a 30-week Safety Phase.

Official Title
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A Phase 3, Single-Arm, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy

Conditions
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Familial Hypercholesterolemia - Homozygous

Intervention / Treatment
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* Drug: lomitapide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Japanese male and female patients aged ≥ 18 years of age who are receiving maximally tolerated, stable, lipid-lowering therapy Diagnosis of functional HoFH Body weight ≥ 40 kg and < 136 kg Negative pregnancy test at screening Exclusion Criteria: Uncontrolled hypertension History of chronic renal insufficiency History of biopsy proven cirrhosis or abnormal liver function tests (LFTs) at screening Any major surgical procedure occurring < 3 months prior to the screening visit Cardiac insufficiency Previous organ transplantation History of a non-skin malignancy within the previous 3 years Patients who are not able to limit their alcohol intake Participation in an investigational drug study within 6 weeks prior to the screening visit Known significant gastrointestinal bowel disease Nursing mothers Serious or unstable medical or psychological conditions Requirement for certain prohibited medications known to be potentially hepatotoxic Use of strong or moderate inhibitors of CYP3A4 Use of simvastatin at doses >10 mg per day Documented diagnosis of any liver disease

Ages Eligible for Study
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Minimum Age: 18 Years

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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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: lomitapide<br>Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. | Drug: lomitapide<br> <br> * Other names: Juxtapid, Lojuxta;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent Change in LDL-C | Mean percent change from baseline | Baseline to Week 26 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Total Cholesterol | Mean percent change from baseline | Baseline to Week 56 | 
| Change in Apo B | Mean percent change from baseline | Baseline to Week 56 | 
| Change in Triglycerides | Mean percent change from baseline | Baseline to Week 56 | 
| Change in Non-HDL-C | Mean percent change from baseline | Baseline to Week 56 | 
| Change in VLDL-C | Mean percent change from baseline | Baseline to Week 56 | 
| Change in Lp(a) | Mean percent change from baseline | Baseline to Week 56 | 
| Change in HDL-C | Mean percent change from baseline | Baseline to Week 56 | 
| Change in Apo AI | Mean percent change from baseline | Baseline to Week 56 | 
| Change in LDL-C | Mean percent change from baseline | Baseline to Week 56 |