NCT02173158

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Apr 2014

Geographic Reach
1 country

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 2, 2014

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

June 23, 2014

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 24, 2014

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 3, 2015

Completed
9 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 17, 2015

Completed
2.8 years until next milestone

Results Posted

Study results publicly available

October 10, 2018

Completed
Last Updated

October 10, 2018

Status Verified

February 1, 2018

Enrollment Period

1 year

First QC Date

June 23, 2014

Results QC Date

February 9, 2018

Last Update Submit

February 21, 2018

Conditions

Familial Hypercholesterolemia - Homozygous

Outcome Measures

Primary Outcomes (1)

  • Percent Change in LDL-C

    Mean percent change from baseline

    Baseline to Week 26

Secondary Outcomes (9)

  • Change in Total Cholesterol

    Baseline to Week 56

  • Change in Apo B

    Baseline to Week 56

  • Change in Triglycerides

    Baseline to Week 56

  • Change in Non-HDL-C

    Baseline to Week 56

  • Change in VLDL-C

    Baseline to Week 56

  • +4 more secondary outcomes

Study Arms (1)

lomitapide

EXPERIMENTAL

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

Drug: lomitapide

Interventions

lomitapideDRUG
Also known as: Juxtapid, Lojuxta
lomitapide

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Japanese male and female patients aged ≥ 18 years of age who are receiving maximally tolerated, stable, lipid-lowering therapy
  • Diagnosis of functional HoFH
  • Body weight ≥ 40 kg and \< 136 kg
  • Negative pregnancy test at screening

You may not qualify if:

  • Uncontrolled hypertension
  • History of chronic renal insufficiency
  • History of biopsy proven cirrhosis or abnormal liver function tests (LFTs) at screening
  • Any major surgical procedure occurring \< 3 months prior to the screening visit
  • Cardiac insufficiency
  • Previous organ transplantation
  • History of a non-skin malignancy within the previous 3 years
  • Patients who are not able to limit their alcohol intake
  • Participation in an investigational drug study within 6 weeks prior to the screening visit
  • Known significant gastrointestinal bowel disease
  • Nursing mothers
  • Serious or unstable medical or psychological conditions
  • Requirement for certain prohibited medications known to be potentially hepatotoxic
  • Use of strong or moderate inhibitors of CYP3A4
  • Use of simvastatin at doses \>10 mg per day
  • +1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Unknown Facility

Kurume-shi, Fukuoka, 830-8522, Japan

Location

Unknown Facility

Kanazawa, Ishikawa-ken, 920-8641, Japan

Location

Unknown Facility

Osakashi, Osaka, 530-00001, Japan

Location

Unknown Facility

Suita-shi, Osaka, 565-8565, Japan

Location

Unknown Facility

Tokorozawa, Saitama, 359-1142, Japan

Location

Unknown Facility

Bunkyo-ku, Tokyo, 113-8519, Japan

Location

MeSH Terms

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

BMS201038

Condition Hierarchy (Ancestors)

Hyperlipoproteinemia Type IILipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemiasHyperlipidemiasDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Results Point of Contact

Title
Alison Long, MD - VP Clinical
Organization
Aegerion Pharmaceuticals, Inc.
alison.long@aegerion.com
857-242-5142

Study Officials

  • Mariko Harada-Shiba, M.D., Ph.D.

    National Cerebral and Cardiovascular Center Research Institute

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 23, 2014

First Posted

June 24, 2014

Study Start

April 2, 2014

Primary Completion

April 3, 2015

Study Completion

December 17, 2015

Last Updated

October 10, 2018

Results First Posted

October 10, 2018

Record last verified: 2018-02

Locations

JP(6)