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Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia

NCT ID: NCT00943306

Last Updated: 2018-06-13

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-10-29

Study Completion Date

2014-12-01

Brief Summary

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This was a long term follow on study to assess the continued long term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia.

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Detailed Description

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This was a phase III open label clinical trial to evaluate the long-term efficacy and safety of lomitapide at the maximum tolerated dose (for each patient) established during the clinical trial 733-005/UP1002. Subjects completing the 78-week treatment period in study 733-005/UP1002 who have not met any of the stopping criteria will be eligible to participate in 733-012. The treatment period continued until a decision was made by the local competent authority regarding marketing authorization. Lomitapide will be given orally once daily. Patient specific doses will be carried forward from 733-005/UP1002, but will not exceed the maximum tolerated dose the patient received during 733-005 /UP1002. The maximum dose for any patient was 80 mg/day. There was no reference therapy in this trial. The effects of the study drug were compared to baseline data (from 733-005/UP1002). Concomitant lipid-lowering therapy including plasmapheresis or LDL apheresis is permitted.

Note that hereafter, Week 0 in Study 733-005/UP1002 will be referred to as Baseline and Week 48 in Study AEGR-733-012 will be referred to as Week 126 (of overall treatment).

Conditions

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Familial Hypercholesterolemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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lomitapide

Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.

Group Type EXPERIMENTAL

lomitapide

Intervention Type DRUG

5-60 mg po every day

Interventions

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lomitapide

5-60 mg po every day

Intervention Type DRUG

Other Intervention Names

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AEGR-733 BMS-201038

Eligibility Criteria

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Inclusion Criteria

1. Completed UP1002 or 733-005.
2. Willing and able to provide consent and comply with the requirements of the study protocol.

Exclusion Criteria

1. Met any of the stopping rules for study discontinuation at the final visit of study UP1002 or 733-005.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Aegerion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Marina Cuchel, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

University of Pennsylvania

Mark Sumeray, MD

Role: STUDY_CHAIR

Aegerion Pharmaceuticals, Inc.

Locations

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Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Robarts Research Institute

London, Ontario, Canada

Site Status

Lipid Clinic and University of Montreal Community Genomic Medicine Center

Chicoutimi, Quebec, Canada

Site Status

Medicina Interna Universitaria

Ferrara, Sicily, Italy

Site Status

Dipartimento di Medicina Clinica e Delle Patologie Emergenti

Palermo, Sicily, Italy

Site Status

Centro Universitario Dislipidemie

Milan, , Italy

Site Status

DAI Ematologia, Oncologia, Anatomia Patologica e Medicina

Roma, , Italy

Site Status

Cardiology Research

Bloemfontein, , South Africa

Site Status

University of Capetown

Cape Town, , South Africa

Site Status

Countries

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United States Canada Italy South Africa

References

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Cuchel M, Bloedon LT, Szapary PO, Kolansky DM, Wolfe ML, Sarkis A, Millar JS, Ikewaki K, Siegelman ES, Gregg RE, Rader DJ. Inhibition of microsomal triglyceride transfer protein in familial hypercholesterolemia. N Engl J Med. 2007 Jan 11;356(2):148-56. doi: 10.1056/NEJMoa061189.

Reference Type BACKGROUND
PMID: 17215532 (View on PubMed)

Cuchel M, Meagher E, Marais AD, et.al. Abstract 1077: A phase III study of microsomal triglyceride transfer protein inhibitor lomitapide (AEGR-733) in patients with homozygous familial hypercholesterolemia: interim results at 6 months. Circulation, Nov 2009; 120: S441

Reference Type BACKGROUND

Larrey D, D'Erasmo L, O'Brien S, Arca M; Italian Working Group on Lomitapide. Long-term hepatic safety of lomitapide in homozygous familial hypercholesterolaemia. Liver Int. 2023 Feb;43(2):413-423. doi: 10.1111/liv.15497. Epub 2022 Dec 30.

Reference Type DERIVED
PMID: 36520008 (View on PubMed)

Other Identifiers

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AEGR-733-012

Identifier Type: -

Identifier Source: org_study_id

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