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

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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02173158

Enrollment

Registered

2014-06-24

Start date

2014-04-02

Completion date

2015-12-17

Last updated

2018-10-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Familial Hypercholesterolemia - Homozygous

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.

Interventions

DRUGlomitapide

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Percent Change in LDL-C	Baseline to Week 26	Mean percent change from baseline

Secondary

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

Countries

Japan

Participant flow

Participants by arm

Arm	Count
Lomitapide Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.	9
Total	9

Withdrawals & dropouts

Period	Reason	FG000
Efficacy Phase	Adverse Event	1

Baseline characteristics

Characteristic	Lomitapide
Age, Continuous	50.3 years STANDARD_DEVIATION 14.71
Race (NIH/OMB) American Indian or Alaska Native	0 Participants
Race (NIH/OMB) Asian	9 Participants
Race (NIH/OMB) Black or African American	0 Participants
Race (NIH/OMB) More than one race	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) White	0 Participants
Sex: Female, Male Female	4 Participants
Sex: Female, Male Male	5 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk
deaths Total, all-cause mortality	0 / 9	0 / 8
other Total, other adverse events	9 / 9	7 / 8
serious Total, serious adverse events	0 / 9	1 / 8