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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.

Tracking Information
First Submitted Date  ICMJE June 23, 2014
First Posted Date  ICMJE June 24, 2014
Results First Submitted Date  ICMJE February 9, 2018
Results First Posted Date  ICMJE October 10, 2018
Last Update Posted Date October 10, 2018
Actual Study Start Date  ICMJE April 2, 2014
Actual Primary Completion Date April 3, 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 21, 2018)
Percent Change in LDL-C [ Time Frame: Baseline to Week 26 ]
Mean percent change from baseline
Original Primary Outcome Measures  ICMJE
 (submitted: June 23, 2014)
Percent Change in LDL-C [ Time Frame: 26 Weeks ]
Mean percent change from baseline.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 21, 2018)
  • Change in Total Cholesterol [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline
  • Change in Apo B [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline
  • Change in Triglycerides [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline
  • Change in Non-HDL-C [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline
  • Change in VLDL-C [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline
  • Change in Lp(a) [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline
  • Change in HDL-C [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline
  • Change in Apo AI [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline
  • Change in LDL-C [ Time Frame: Baseline to Week 56 ]
    Mean percent change from baseline
Original Secondary Outcome Measures  ICMJE
 (submitted: June 23, 2014)
Long term safety [ Time Frame: 56 Weeks ]
Assessed through findings noted on physical examination and ECG, changes in laboratory parameters and weight, change in hepatic fat as assessed by MRI, and reported AEs
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
Official Title  ICMJE 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
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Familial Hypercholesterolemia - Homozygous
Intervention  ICMJE Drug: lomitapide
Other Name: Juxtapid, Lojuxta
Study Arms  ICMJE Experimental: lomitapide
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Intervention: Drug: lomitapide
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 21, 2018)
9
Original Estimated Enrollment  ICMJE
 (submitted: June 23, 2014)
10
Actual Study Completion Date  ICMJE December 17, 2015
Actual Primary Completion Date April 3, 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Japan
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02173158
Other Study ID Numbers  ICMJE AEGR-733-030
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Aegerion Pharmaceuticals, Inc.
Study Sponsor  ICMJE Aegerion Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Mariko Harada-Shiba, M.D., Ph.D. National Cerebral and Cardiovascular Center Research Institute
PRS Account Aegerion Pharmaceuticals, Inc.
Verification Date February 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP