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CompletedNCT02173158Updated Oct 10, 2018Results posted

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

A Phase 3 interventional study of lomitapide in Familial Hypercholesterolemia - Homozygous, sponsored by Aegerion Pharmaceuticals, Inc.. Completed at 6 sites in Japan. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-10-10.

Sponsored by Aegerion Pharmaceuticals, Inc. (part of Chiesi Farmaceutici S.p.A.) · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
9
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

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

Read the 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.

02

Conditions studied

  • Familial Hypercholesterolemia - Homozygous
03

In context

Homozygous Familial Hypercholesterolemia

68 studies on the registry are indexed under Homozygous Familial Hypercholesterolemia; 13 are open to participants now.

This study's enrollment of 9 is below the median of 21 across 51 interventional studies indexed under Homozygous Familial Hypercholesterolemia.

Browse Homozygous Familial Hypercholesterolemia studies →

Lead sponsor

Aegerion Pharmaceuticals, Inc. is the lead sponsor of 17 studies on the registry; none are open to participants now.

First submitted before Aegerion Pharmaceuticals, Inc. became part of Chiesi Farmaceutici S.p.A..

Browse Aegerion Pharmaceuticals, Inc. studies →All Chiesi Farmaceutici S.p.A. studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

Exclusion Criteria:

  1. Uncontrolled hypertension
  2. History of chronic renal insufficiency
  3. History of 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 within the previous 3 years
  8. Patients who are not able to limit their alcohol intake
  9. Participation in an 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
  15. Use of simvastatin at doses >10 mg per day
  16. Documented diagnosis of any liver disease
Terms in these criteria, in plain language(4)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Biopsy and archival tissue

A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.

Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

CYP3A (CYP3A4) inhibitors and inducers

CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.

Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
9 participants (actual)

Study arms

  • Experimental
    lomitapide

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

    Drug: lomitapide

Interventions

06

What researchers measure

Primary outcomes

  1. Percent Change in LDL-C

    Mean percent change from baseline

    Time frame: Baseline to Week 26

Secondary outcomes

  1. Change in Total Cholesterol

    Mean percent change from baseline

    Time frame: Baseline to Week 56

  2. Change in Apo B

    Mean percent change from baseline

    Time frame: Baseline to Week 56

  3. Change in Triglycerides

    Mean percent change from baseline

    Time frame: Baseline to Week 56

  4. Change in Non-HDL-C

    Mean percent change from baseline

    Time frame: Baseline to Week 56

  5. Change in VLDL-C

    Mean percent change from baseline

    Time frame: Baseline to Week 56

  6. Change in Lp(a)

    Mean percent change from baseline

    Time frame: Baseline to Week 56

  7. Change in HDL-C

    Mean percent change from baseline

    Time frame: Baseline to Week 56

  8. Change in Apo AI

    Mean percent change from baseline

    Time frame: Baseline to Week 56

  9. Change in LDL-C

    Mean percent change from baseline

    Time frame: Baseline to Week 56

07

Results

Posted Oct 10, 2018

Participant flow

Efficacy Phase
Participant flow — Efficacy Phase
MilestoneLomitapide
Started9
Completed8
Not completed1
Withdrew: Adverse event1
Safety Phase
Participant flow — Safety Phase
MilestoneLomitapide
Started8
Completed8
Not completed0

Outcome measures

PrimaryPercent Change in LDL-C

Mean percent change from baseline

Time frame:
Baseline to Week 26
Reported as:
Mean · Percent change
Percent Change in LDL-C
Percent changeLomitapide
Percent Change in LDL-C-42.2 ± 18.16
SecondaryChange in Total Cholesterol

Mean percent change from baseline

Time frame:
Baseline to Week 56
Reported as:
Mean · Percent change
Change in Total Cholesterol
Percent changeLomitapide
Change in Total Cholesterol-25.9 ± 18.57
SecondaryChange in Apo B

Mean percent change from baseline

Time frame:
Baseline to Week 56
Reported as:
Mean · Percent change
Change in Apo B
Percent changeLomitapide
Change in Apo B-41.4 ± 21.90
SecondaryChange in Triglycerides

Mean percent change from baseline

Time frame:
Baseline to Week 56
Reported as:
Mean · Percent change
Change in Triglycerides
Percent changeLomitapide
Change in Triglycerides-44.4 ± 12.70
SecondaryChange in Non-HDL-C

Mean percent change from baseline

Time frame:
Baseline to Week 56
Reported as:
Mean · Percent change
Change in Non-HDL-C
Percent changeLomitapide
Change in Non-HDL-C-34.6 ± 22.50
SecondaryChange in VLDL-C

Mean percent change from baseline

Time frame:
Baseline to Week 56
Reported as:
Mean · Percent change
Change in VLDL-C
Percent changeLomitapide
Change in VLDL-C-44.8 ± 12.05
SecondaryChange in Lp(a)

Mean percent change from baseline

Time frame:
Baseline to Week 56
Reported as:
Mean · Percent change
Change in Lp(a)
Percent changeLomitapide
Change in Lp(a)-27.2 ± 23.50
SecondaryChange in HDL-C

Mean percent change from baseline

Time frame:
Baseline to Week 56
Reported as:
Mean · Percent change
Change in HDL-C
Percent changeLomitapide
Change in HDL-C5.9 ± 19.64
SecondaryChange in Apo AI

Mean percent change from baseline

Time frame:
Baseline to Week 56
Reported as:
Mean · Percent change
Change in Apo AI
Percent changeLomitapide
Change in Apo AI-2.8 ± 11.61
SecondaryChange in LDL-C

Mean percent change from baseline

Time frame:
Baseline to Week 56
Reported as:
Mean · Percent change
Change in LDL-C
Percent changeLomitapide
Change in LDL-C-37.5 ± 24.21

Adverse events

Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Efficacy Phase0/9 (0%)0/9 (0%)9/9 (100%)
Safety Phase0/8 (0%)1/8 (12.5%)7/8 (87.5%)
Most frequent serious events
Most frequent serious events
EventEfficacy PhaseSafety Phase
Chest painGeneral disorders0/91/8
Most frequent other events
Showing 10 of 37
Most frequent other events
EventEfficacy PhaseSafety Phase
DiarrhoeaGastrointestinal disorders7/94/8
NasopharyngitisInfections and infestations4/93/8
Liver function test abnormalInvestigations3/91/8
AnaemiaBlood and lymphatic system disorders2/92/8
NauseaGastrointestinal disorders2/91/8
GastroenteritisInfections and infestations2/91/8
HeadacheNervous system disorders2/90/8
HaemorrhoidsGastrointestinal disorders1/91/8
InfluenzaInfections and infestations1/91/8
PeriodontitisInfections and infestations1/91/8

Baseline characteristics

Age, Continuous
Age, Continuous(years)Lomitapide
Mean50.3 ± 14.71
Sex: Female, Male
Sex: Female, Male(Participants)Lomitapide
Female4
Male5
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Lomitapide
American Indian or Alaska Native0
Asian9
Native Hawaiian or Other Pacific Islander0
Black or African American0
White0
More than one race0
Unknown or Not Reported0
08

Study locations

6 sites
  • Kurume-shi, Fukuoka 830-8522, Japan
  • Kanazawa, Ishikawa 920-8641, Japan
  • Osakashi, Osaka 530-00001, Japan
  • Suita-shi, Osaka 565-8565, Japan
  • Tokorozawa, Saitama 359-1142, Japan
  • Bunkyo-ku, Tokyo 113-8519, Japan
09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 10, 2018, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT02173158
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Jun 24, 2014
Start date
Apr 2, 2014
Primary completion
Apr 3, 2015
Completion
Dec 17, 2015
Results posted
Oct 10, 2018
Last update
Oct 10, 2018

Study contacts

Mariko Harada-Shiba, M.D., Ph.D.
principal investigator · National Cerebral and Cardiovascular Center Research Institute

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Feb 2018. You cannot join it, but the record below documents what was studied.

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Studies of the drug tested here: BMS201038

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