Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
- Registration Number
- NCT02173158
- Lead Sponsor
- Aegerion Pharmaceuticals, Inc.
- Brief Summary
Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 9
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description lomitapide lomitapide Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
- Primary Outcome Measures
Name Time Method Percent Change in LDL-C Baseline to Week 26 Mean percent change from baseline
- Secondary Outcome Measures
Name Time Method Change in HDL-C Baseline to Week 56 Mean percent change from baseline
Change in Apo AI Baseline to Week 56 Mean percent change from baseline
Change in Apo B Baseline to Week 56 Mean percent change from baseline
Change in Triglycerides Baseline to Week 56 Mean percent change from baseline
Change in Non-HDL-C Baseline to Week 56 Mean percent change from baseline
Change in Total Cholesterol Baseline to Week 56 Mean percent change from baseline
Change in VLDL-C Baseline to Week 56 Mean percent change from baseline
Change in Lp(a) Baseline to Week 56 Mean percent change from baseline
Change in LDL-C Baseline to Week 56 Mean percent change from baseline
Related Research Topics
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