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

Phase 3

Completed

• Conditions: Familial Hypercholesterolemia - Homozygous
• Interventions: Drug: lomitapide

• 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.

Study Timeline

• Study Start: 2014-04-02
• Study First Submitted: 2014-06-23
• Study First Submitted QC: 2014-06-23
• Study First Posted: 2014-06-24
• Primary Completion: 2015-04-03
• Study Completion: 2015-12-17
• Status Verified: 2018-02
• Results First Submitted: 2018-02-09
• Results First Submitted QC: 2018-02-21
• Last Update Submitted: 2018-02-21
• Results First Posted: 2018-10-10
• Last Update Posted: 2018-10-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

1. Japanese male and female patients aged ≥ 18 years of age who are receiving maximally tolerated, stable, lipid-lowering therapy
2. Diagnosis of functional HoFH
3. Body weight ≥ 40 kg and < 136 kg
4. Negative pregnancy test at screening

Exclusion Criteria

1. Uncontrolled hypertension
2. History of chronic renal insufficiency
3. History of biopsy proven cirrhosis or abnormal liver function tests (LFTs) at screening
4. Any major surgical procedure occurring < 3 months prior to the screening visit
5. Cardiac insufficiency
6. Previous organ transplantation
7. History of a non-skin malignancy within the previous 3 years
8. Patients who are not able to limit their alcohol intake
9. Participation in an investigational drug study within 6 weeks prior to the screening visit
10. Known significant gastrointestinal bowel disease
11. Nursing mothers
12. Serious or unstable medical or psychological conditions
13. Requirement for certain prohibited medications known to be potentially hepatotoxic
14. Use of strong or moderate inhibitors of CYP3A4
15. Use of simvastatin at doses >10 mg per day
16. 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 Total Cholesterol	Baseline to Week 56	Mean percent change from baseline
Change in LDL-C	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 VLDL-C	Baseline to Week 56	Mean percent change from baseline
Change in Lp(a)	Baseline to Week 56	Mean percent change from baseline