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Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy

Primary Purpose

Homozygous Familial Hypercholesterolemia

Status

Withdrawn

Phase

Phase 3

Locations

Study Type

Interventional

Intervention

Lomitapide

About this trial

This is an interventional treatment trial for Homozygous Familial Hypercholesterolemia focused on measuring HoFH

Eligibility Criteria

5 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Male or female aged ≥5 and <18 years with diagnosed functional HoFH
Patient must weigh at least 15 kg and be at or above the 10th percentile in BMI and at least 10th percentile in height for age and gender based on CDC growth charts
Negative pregnancy test at Screening and during the study for females of child bearing age
Potentially sexually active female patients who are of child-bearing age must either be sexually abstinent or follow two acceptable methods of contraception

Exclusion Criteria:

Other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolemia (e.g., nephrotic syndrome, hypothyroidism).
Abnormal liver function test at Screening
Moderate or severe hepatic impairment or active liver disease
Serum creatine phosphokinase (CPK) level >2 × ULN.
Chronic renal insufficiency
History of drug abuse within the last 3 years or habitual alcohol consumption
New York Heart Association (NYHA) Class III or IV congestive heart failure.
Uncontrolled hypertension
In the judgment of the PI, precocious or delayed puberty or endocrine disorder that would affect growth
History of non-skin malignancy or other cancers occurring within the past 3 years
History of inflammatory bowel disease or other malabsorption syndrome or a history of bowel resection, gastric bypass, or other weight loss surgical procedure.
Use of mipomersen within 6 months of Screening.
Any medical condition for which the life expectancy is predicted to be less than 5 years.
Any patient who is unable to avoid treatment with strong or moderate cytochrome P450 3A4 (CYP3A4) inhibitors, or other drugs contraindicated for use with lomitapide during the study.
Participation in an interventional clinical study within 6 weeks for a statin therapy or within 6 months for any other unapproved therapy.

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Lomitapide

Arm Description

Outcomes

Primary Outcome Measures

Percent change in LDL-C

Secondary Outcome Measures

Percent Change in TC

Percent change in non-HDL-C

Percent change in HDL-C

Percent change in TG

Percent change in VLDL-C

Percent change in Lp(a)

Percent change in apo B

Percent change in apo A-1

Percent change in LDL-C

Changes in lipid-lowering therapy

Changes in LDL apheresis

Percent of patients achieving goal (LDL-C of <100 mg/dL [2.6 mmol/L] for patients without documented cardiovascular disease [CVD] at Baseline

Percent of patients achieving goal LDL-C of <70 mg/dL [1.8 mmol/L]) for patients with documented CVD at Baseline.

Changes in laboratory parameters (including hepatic and renal function)

Reported Adverse Events

Electrocardiogram (ECG) changes

Pulmonary function tests (PFTs)

Bone health/age (x-ray of the wrist)

Height Measurement

Weight Measurement

Body Mass Measurement

Tanner Staging

Percent change in hepatic fat

Blood Pressure

Heart Rate

Temperature

Respiration (breaths/min)

Full Information

NCT ID

NCT02765841

First Posted

April 27, 2016

Last Updated

February 21, 2018

Sponsor

Aegerion Pharmaceuticals, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT02765841

Brief Title

Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy

Official Title

A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy

Study Type

Interventional

2. Study Status

Record Verification Date

February 2018

Overall Recruitment Status

Withdrawn

Study Start Date

May 2016 (undefined)

Primary Completion Date

March 2017 (Anticipated)

Study Completion Date

December 2019 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Aegerion Pharmaceuticals, Inc.

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This is a Phase 3 single-arm, open-label, international, multi-center clinical trial to evaluate the efficacy and safety of lomitapide in pediatric patients with HoFH who are receiving stable lipid-lowering therapy, including LDL apheresis. The study is comprised of a 12-week Run-in Period, a primary 24-week Efficacy Phase, followed by an 80-week Safety Phase.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Homozygous Familial Hypercholesterolemia

Keywords

HoFH

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Lomitapide

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Lomitapide

Other Intervention Name(s)

Juxtapid, Lojuxta

Primary Outcome Measure Information:

Title

Percent change in LDL-C

Time Frame

Baseline, Week 24

Secondary Outcome Measure Information:

Title

Percent Change in TC

Time Frame