Clinical trial NCT02173158: Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Familial Hypercholesterolemia - Homozygous
Lead sponsor: Aegerion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2014-06-24
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Percent Change in LDL-C
timeFrame: Baseline to Week 26
Secondary outcomes:
measure: Change in Total Cholesterol
timeFrame: Baseline to Week 56
measure: Change in Apo B
timeFrame: Baseline to Week 56
measure: Change in Triglycerides
timeFrame: Baseline to Week 56
measure: Change in Non-HDL-C
timeFrame: Baseline to Week 56
measure: Change in VLDL-C
timeFrame: Baseline to Week 56
measure: Change in Lp(a)
timeFrame: Baseline to Week 56
measure: Change in HDL-C
timeFrame: Baseline to Week 56
measure: Change in Apo AI
timeFrame: Baseline to Week 56
measure: Change in LDL-C
timeFrame: Baseline to Week 56
Other outcomes:
Locations:
city: Kurume-shi
country: Japan
state: Fukuoka
city: Kanazawa
country: Japan
state: Ishikawa
city: Osakashi
country: Japan
state: Osaka
city: Suita-shi
country: Japan
state: Osaka
city: Tokorozawa
country: Japan
state: Saitama
city: Bunkyo-ku
country: Japan
state: Tokyo
Study officials:
affiliation: National Cerebral and Cardiovascular Center Research Institute
name: Mariko Harada-Shiba, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: