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

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ClinicalTrials.gov Identifier: NCT02173158
Recruitment Status : Completed
First Posted : June 24, 2014
Results First Posted : October 10, 2018
Last Update Posted : October 10, 2018
Sponsor:
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.

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

Condition or disease Intervention/treatment Phase
Familial Hypercholesterolemia - Homozygous Drug: lomitapide Phase 3

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : April 2, 2014
Actual Primary Completion Date : April 3, 2015
Actual Study Completion Date : December 17, 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Lomitapide

Arms and Interventions
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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 Name: Juxtapid, Lojuxta


Outcome Measures
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Primary Outcome Measures :
  1. Percent Change in LDL-C [ Time Frame: Baseline to Week 26 ]
    Mean percent change from baseline


Secondary Outcome Measures :
  1. Change in Total Cholesterol [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline

  2. Change in Apo B [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline

  3. Change in Triglycerides [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline

  4. Change in Non-HDL-C [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline

  5. Change in VLDL-C [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline

  6. Change in Lp(a) [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline

  7. Change in HDL-C [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline

  8. Change in Apo AI [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline

  9. Change in LDL-C [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02173158


Locations
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Japan
Kurume-shi, Fukuoka, Japan, 830-8522
Kanazawa, Ishikawa, Japan, 920-8641
Osakashi, Osaka, Japan, 530-00001
Suita-shi, Osaka, Japan, 565-8565
Tokorozawa, Saitama, Japan, 359-1142
Bunkyo-ku, Tokyo, Japan, 113-8519
Sponsors and Collaborators
Aegerion Pharmaceuticals, Inc.
Investigators
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Principal Investigator: Mariko Harada-Shiba, M.D., Ph.D. National Cerebral and Cardiovascular Center Research Institute
More Information
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Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02173158    
Other Study ID Numbers: AEGR-733-030
First Posted: June 24, 2014    Key Record Dates
Results First Posted: October 10, 2018
Last Update Posted: October 10, 2018
Last Verified: February 2018
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
 Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias