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

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-04-02

Study Completion Date

2015-12-17

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.

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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.

Conditions

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Familial Hypercholesterolemia - Homozygous

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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lomitapide

Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.

Group Type EXPERIMENTAL

lomitapide

Intervention Type DRUG

Interventions

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lomitapide

Intervention Type DRUG

Other Intervention Names

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Juxtapid, Lojuxta

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No