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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02173158 |
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Date of registration:
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23/06/2014 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
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Scientific title:
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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 |
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Date of first enrolment:
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April 2, 2014 |
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Target sample size:
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9 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02173158 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 3
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Countries of recruitment
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Japan
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Contacts
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Name:
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Mariko Harada-Shiba, M.D., Ph.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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National Cerebral and Cardiovascular Center Research Institute |
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Key inclusion & exclusion criteria
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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
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Familial Hypercholesterolemia - Homozygous
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Intervention(s)
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Drug: lomitapide
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Primary Outcome(s)
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Percent Change in LDL-C
[Time Frame: Baseline to Week 26]
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Secondary Outcome(s)
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Change in Non-HDL-C
[Time Frame: Baseline to Week 56]
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Change in Triglycerides
[Time Frame: Baseline to Week 56]
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Change in HDL-C
[Time Frame: Baseline to Week 56]
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Change in VLDL-C
[Time Frame: Baseline to Week 56]
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Change in Apo AI
[Time Frame: Baseline to Week 56]
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Change in Lp(a)
[Time Frame: Baseline to Week 56]
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Change in Apo B
[Time Frame: Baseline to Week 56]
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Change in LDL-C
[Time Frame: Baseline to Week 56]
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Change in Total Cholesterol
[Time Frame: Baseline to Week 56]
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Secondary ID(s)
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AEGR-733-030
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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