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

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

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

Study Overview

• Status: Completed
• Conditions: Familial Hypercholesterolemia - Homozygous
• Intervention / Treatment: Drug: lomitapide

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 Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Fukuoka
    • Kurume-shi, Fukuoka, Japan, 830-8522
  • Ishikawa
    • Kanazawa, Ishikawa, Japan, 920-8641
  • Osaka
    • Osakashi, Osaka, Japan, 530-00001
    • Suita-shi, Osaka, Japan, 565-8565
  • Saitama
    • Tokorozawa, Saitama, Japan, 359-1142
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan, 113-8519

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Aegerion Pharmaceuticals, Inc.
• Investigators: Principal Investigator: Mariko Harada-Shiba, M.D., Ph.D., National Cerebral and Cardiovascular Center Research Institute

Study record dates

Study Major Dates

• Study Start (Actual): April 2, 2014
• Primary Completion (Actual): April 3, 2015
• Study Completion (Actual): December 17, 2015

Study Registration Dates

• First Submitted: June 23, 2014
• First Submitted That Met QC Criteria: June 23, 2014
• First Posted (Estimate): June 24, 2014

Study Record Updates

• Last Update Posted (Actual): October 10, 2018
• Last Update Submitted That Met QC Criteria: February 21, 2018
• Last Verified: February 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Hyperlipidemias; Dyslipidemias; Lipid Metabolism, Inborn Errors; Hyperlipoproteinemias; Hypercholesterolemia; Hyperlipoproteinemia Type II
• Other Study ID Numbers: AEGR-733-030