- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02173158
Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
February 21, 2018 updated by: Aegerion Pharmaceuticals, Inc.
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
Intervention / Treatment
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
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Fukuoka
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Kurume-shi, Fukuoka, Japan, 830-8522
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Ishikawa
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Kanazawa, Ishikawa, Japan, 920-8641
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Osaka
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Osakashi, Osaka, Japan, 530-00001
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Suita-shi, Osaka, Japan, 565-8565
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Saitama
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Tokorozawa, Saitama, Japan, 359-1142
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Tokyo
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Bunkyo-ku, Tokyo, Japan, 113-8519
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
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:
- 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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
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.
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Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percent Change in LDL-C
Time Frame: Baseline to Week 26
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Mean percent change from baseline
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Baseline to Week 26
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Total Cholesterol
Time Frame: Baseline to Week 56
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Mean percent change from baseline
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Baseline to Week 56
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Change in Apo B
Time Frame: Baseline to Week 56
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Mean percent change from baseline
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Baseline to Week 56
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Change in Triglycerides
Time Frame: Baseline to Week 56
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Mean percent change from baseline
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Baseline to Week 56
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Change in Non-HDL-C
Time Frame: Baseline to Week 56
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Mean percent change from baseline
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Baseline to Week 56
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Change in VLDL-C
Time Frame: Baseline to Week 56
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Mean percent change from baseline
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Baseline to Week 56
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Change in Lp(a)
Time Frame: Baseline to Week 56
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Mean percent change from baseline
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Baseline to Week 56
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Change in HDL-C
Time Frame: Baseline to Week 56
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Mean percent change from baseline
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Baseline to Week 56
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Change in Apo AI
Time Frame: Baseline to Week 56
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Mean percent change from baseline
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Baseline to Week 56
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Change in LDL-C
Time Frame: Baseline to Week 56
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Mean percent change from baseline
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Baseline to Week 56
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Mariko Harada-Shiba, M.D., Ph.D., National Cerebral and Cardiovascular Center Research Institute
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
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
Other Study ID Numbers
- AEGR-733-030
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Clinical Trials on lomitapide
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Amryt PharmaActive, not recruitingHomozygous Familial Hypercholesterolaemia (HoFH)Germany, Israel, Italy, Saudi Arabia, Spain, Tunisia
-
Aegerion Pharmaceuticals, Inc.Richmond Pharmacology LimitedCompletedHealthy VolunteerUnited Kingdom
-
Aegerion Pharmaceuticals, Inc.CompletedIntra-subject Variability of PharmacokineticsUnited Kingdom
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Aegerion Pharmaceuticals, Inc.WithdrawnHomozygous Familial Hypercholesterolemia
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Aegerion Pharmaceuticals, Inc.Completed
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Amryt PharmaRecruitingHomozygous Familial HypercholesterolemiaItaly, United States, Argentina, Canada, France, Greece, Netherlands, United Kingdom
-
Amryt PharmaEnrolling by invitation
-
Aegerion Pharmaceuticals, Inc.WithdrawnHomozygous Familial Hypercholesterolemia
-
Aegerion Pharmaceuticals, Inc.CompletedFamilial HypercholesterolemiaUnited States, Canada, Italy, South Africa
-
Aegerion Pharmaceuticals, Inc.FDA Office of Orphan Products DevelopmentCompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, South Africa