Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
This trial is active, not recruiting.
|Condition||familial hypercholesterolemia - homozygous|
|Sponsor||Aegerion Pharmaceuticals, Inc.|
|Start date||April 2014|
|End date||April 2015|
|Trial size||10 participants|
|Trial identifier||NCT02173158, AEGR-733-030|
Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy.
|United States||No locations recruiting|
|Other Countries||No locations recruiting|
|Kurume-shi, Japan 830-8522||not available||no longer recruiting|
|Kanazawa, Japan 920-8641||not available||no longer recruiting|
|Iida-shi, Japan 395-8522||not available||no longer recruiting|
|Matsumoto-shi, Japan 390-8621||not available||no longer recruiting|
|Osakashi, Japan 530-00001||not available||no longer recruiting|
|Suita-shi, Japan 565-8565||not available||no longer recruiting|
|Tokorozawa, Japan 359-1142||not available||no longer recruiting|
|Bunkyo-ku, Japan 113-8519||not available||no longer recruiting|
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Percent change in LDL-C
time frame: 26 Weeks
Long term safety
time frame: 56 Weeks
Male or female participants at least 18 years old.
Inclusion Criteria: 1. Japanese male and female HoFH patients aged ≥ 18 years of age 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
|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|
|Principal investigator||Mariko Harada-Shiba, M.D., Ph.D.|
|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 two phases: a primary 26-week Efficacy Phase followed by a 30-week Safety Phase.|
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