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

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

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

NCT02765841•Aegerion Pharmaceuticals, Inc.

View on ClinicalTrials.gov

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.

Eligibility

Age

5 - 17 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Male or female aged ≥5 and \<18 years with diagnosed functional HoFH
•2. 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
•3. Negative pregnancy test at Screening and during the study for females of child bearing age
•4. 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

•1. Other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolemia (e.g., nephrotic syndrome, hypothyroidism).
•2. Abnormal liver function test at Screening
•3. Moderate or severe hepatic impairment or active liver disease
•4. Serum creatine phosphokinase (CPK) level \>2 × ULN.
•5. Chronic renal insufficiency
•6. History of drug abuse within the last 3 years or habitual alcohol consumption
•7. New York Heart Association (NYHA) Class III or IV congestive heart failure.
•8. Uncontrolled hypertension
•9. In the judgment of the PI, precocious or delayed puberty or endocrine disorder that would affect growth
•10. History of non-skin malignancy or other cancers occurring within the past 3 years
+5 more criteria

Key Dates

Start Date

May 1, 2016

Primary Completion Date

March 1, 2017

Completion Date

December 1, 2019

Last Updated

February 22, 2018

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

Lomitapide

DRUG

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

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Homozygous Familial Hypercholesterolemia

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ACTIVE_NOT_RECRUITING

Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia

NCT06832371

Homozygous Familial Hypercholesterolemia (HoFH)Major Adverse Cardiovascular Events (MACE)+1 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 22, 2018Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia

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