Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy

Familial Hypercholesterolemia - Homozygous Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02173158
• Other study ID #: AEGR-733-030
• Status: Completed
• Phase: Phase 3
• Start date: April 2, 2014
• Est. completion date: December 17, 2015

Study information

• Verified date: February 2018
• Source: Aegerion Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: December 17, 2015
• Est. primary completion date: April 3, 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Familial Hypercholesterolemia - Homozygous
• Hypercholesterolemia
• Hyperlipoproteinemia Type II

Intervention

Drug:
• lomitapide

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Aegerion Pharmaceuticals, Inc.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent Change in LDL-C	Mean percent change from baseline	Baseline to Week 26
Secondary	Change in Total Cholesterol	Mean percent change from baseline	Baseline to Week 56
Secondary	Change in Apo B	Mean percent change from baseline	Baseline to Week 56
Secondary	Change in Triglycerides	Mean percent change from baseline	Baseline to Week 56
Secondary	Change in Non-HDL-C	Mean percent change from baseline	Baseline to Week 56
Secondary	Change in VLDL-C	Mean percent change from baseline	Baseline to Week 56
Secondary	Change in Lp(a)	Mean percent change from baseline	Baseline to Week 56
Secondary	Change in HDL-C	Mean percent change from baseline	Baseline to Week 56
Secondary	Change in Apo AI	Mean percent change from baseline	Baseline to Week 56
Secondary	Change in LDL-C	Mean percent change from baseline	Baseline to Week 56