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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT00943306
First received: July 21, 2009
Last updated: January 15, 2014
Last verified: January 2014
  Purpose

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


Condition Intervention Phase
Familial Hypercholesterolemia
Drug: lomitapide
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor 'Lomitapide' (LOMITAPIDE) in Patients With Homozygous Familial Hypercholesterolemia

Resource links provided by NLM:


Further study details as provided by Aegerion Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Percent change in LDL-C [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Percent change in lipid parameters [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
  • Long term safety [ Time Frame: Every 12 weeks until the study is completed ] [ Designated as safety issue: Yes ]
    This is an open ended study that will continue until a local market authorization decision is made in each region

  • Percent hepatic fat by NMRS [ Time Frame: Every 24 weeks until the study is completed ] [ Designated as safety issue: Yes ]
    This is an open ended study that will remain open until a local decision regarding market authorization is made


Estimated Enrollment: 25
Study Start Date: September 2009
Estimated Study Completion Date: December 2014
Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: lomitapide
Maximum tolerated dose of lomitapide (up to 80mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Drug: lomitapide
5-60 mg po every day
Other Names:
  • AEGR-733
  • BMS-201038

Detailed Description:

This is 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 study 733-005/UP1002 who have not met any of the stopping criteria will be eligible to participate in 733-012. The treatment period will continue until a decision has been 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 is 80 mg/day. There is no reference therapy in this trial. The effects of the study drug will be compared to baseline data (from 733-005/UP1002). Concomitant lipid-lowering therapy including plasmapheresis or LDL apheresis is permitted.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00943306

Locations
United States, California
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
United States, Pennsylvania
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Canada, Ontario
Robarts Research Institute
London, Ontario, Canada, N6A 5K8
Canada, Quebec
Lipid Clinic and University of Montreal Community Genomic Medicine Center
Chicoutimi, Quebec, Canada, G7H 5H6
Italy
Medicina Interna Universitaria
Ferrara, Sicily, Italy
Dipartimento di Medicina Clinica e Delle Patologie Emergenti
Palermo, Sicily, Italy
Centro Universitario Dislipidemie
Milano, Italy
South Africa
Cardiology Research
Bloemfontein, South Africa, 9300
University of Capetown
Cape town, South Africa, 7925
Sponsors and Collaborators
Aegerion Pharmaceuticals, Inc.
Investigators
Principal Investigator: Marina Cuchel, MD, PhD University of Pennsylvania
Study Chair: Mark Sumeray, MD Aegerion Pharmaceuticals, Inc.
  More Information

Publications:
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

Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT00943306     History of Changes
Other Study ID Numbers: 733-012
Study First Received: July 21, 2009
Last Updated: January 15, 2014
Health Authority: United States: Food and Drug Administration
South Africa: Medicines Control Council
Canada: Health Canada
Italy: Ministry of Health

Keywords provided by Aegerion Pharmaceuticals, Inc.:
Homozygous
Familial
Hypercholesterolemia
MTP
Apheresis
lomitapide
Homozygous Familial Hypercholesterolemia (HoFH)

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Dyslipidemias
Genetic Diseases, Inborn
Hyperlipidemias
Hyperlipoproteinemias
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Metabolic Diseases
Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on October 22, 2014