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

Primary Purpose

Familial Hypercholesterolemia - Homozygous

Status
Completed
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
lomitapide
Sponsored by
Aegerion Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Familial Hypercholesterolemia - Homozygous

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

lomitapide

Arm Description

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

Outcomes

Primary Outcome Measures

Percent Change in LDL-C
Mean percent change from baseline

Secondary Outcome Measures

Change in Total Cholesterol
Mean percent change from baseline
Change in Apo B
Mean percent change from baseline
Change in Triglycerides
Mean percent change from baseline
Change in Non-HDL-C
Mean percent change from baseline
Change in VLDL-C
Mean percent change from baseline
Change in Lp(a)
Mean percent change from baseline
Change in HDL-C
Mean percent change from baseline
Change in Apo AI
Mean percent change from baseline
Change in LDL-C
Mean percent change from baseline

Full Information

First Posted
June 23, 2014
Last Updated
February 21, 2018
Sponsor
Aegerion Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02173158
Brief Title
Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
Official Title
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 Type
Interventional

2. Study Status

Record Verification Date
February 2018
Overall Recruitment Status
Completed
Study Start Date
April 2, 2014 (Actual)
Primary Completion Date
April 3, 2015 (Actual)
Study Completion Date
December 17, 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Aegerion Pharmaceuticals, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Hypercholesterolemia - Homozygous

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Actual)

8. Arms, Groups, and Interventions

Arm Title
lomitapide
Arm Type
Experimental
Arm Description
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Intervention Type
Drug
Intervention Name(s)
lomitapide
Other Intervention Name(s)
Juxtapid, Lojuxta
Primary Outcome Measure Information:
Title
Percent Change in LDL-C
Description
Mean percent change from baseline
Time Frame
Baseline to Week 26
Secondary Outcome Measure Information:
Title
Change in Total Cholesterol
Description
Mean percent change from baseline
Time Frame
Baseline to Week 56
Title
Change in Apo B
Description
Mean percent change from baseline
Time Frame
Baseline to Week 56
Title
Change in Triglycerides
Description
Mean percent change from baseline
Time Frame
Baseline to Week 56
Title
Change in Non-HDL-C
Description
Mean percent change from baseline
Time Frame
Baseline to Week 56
Title
Change in VLDL-C
Description
Mean percent change from baseline
Time Frame
Baseline to Week 56
Title
Change in Lp(a)
Description
Mean percent change from baseline
Time Frame
Baseline to Week 56
Title
Change in HDL-C
Description
Mean percent change from baseline
Time Frame
Baseline to Week 56
Title
Change in Apo AI
Description
Mean percent change from baseline
Time Frame
Baseline to Week 56
Title
Change in LDL-C
Description
Mean percent change from baseline
Time Frame
Baseline to Week 56

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mariko Harada-Shiba, M.D., Ph.D.
Organizational Affiliation
National Cerebral and Cardiovascular Center Research Institute
Official's Role
Principal Investigator
Facility Information:
City
Kurume-shi
State/Province
Fukuoka
ZIP/Postal Code
830-8522
Country
Japan
City
Kanazawa
State/Province
Ishikawa
ZIP/Postal Code
920-8641
Country
Japan
City
Osakashi
State/Province
Osaka
ZIP/Postal Code
530-00001
Country
Japan
City
Suita-shi
State/Province
Osaka
ZIP/Postal Code
565-8565
Country
Japan
City
Tokorozawa
State/Province
Saitama
ZIP/Postal Code
359-1142
Country
Japan
City
Bunkyo-ku
State/Province
Tokyo
ZIP/Postal Code
113-8519
Country
Japan

12. IPD Sharing Statement

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

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