WithdrawnPhase 3

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

0Started 2016-05

Plain-language 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.

Who can participate

Age range5 Years – 17 Years

SexALL

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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

What they're measuring

Percent change in LDL-C

Timeframe: Baseline, Week 24

Trial details

NCT IDNCT02765841

SponsorAegerion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-03

View on ClinicalTrials.gov More Homozygous Familial Hypercholesterolemia trials