Trial record 1 of 72 for:
"Familial hypercholesterolemia"
Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
This study is ongoing, but not recruiting participants.
Sponsor:
Aegerion Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT00943306
First received: July 21, 2009
Last updated: January 3, 2013
Last verified: March 2012
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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:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
Farber lipogranulomatosis
hypercholesterolemia
MedlinePlus related topics:
Cholesterol
Drug Information available for:
Lomitapide
U.S. FDA Resources
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 |
| Estimated 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:
|
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:
- Completed UP1002 or 733-005.
- Willing and able to provide consent and comply with the requirements of the study protocol.
Exclusion Criteria:
- Met any of the stopping rules for study discontinuation at the final visit of study UP1002 or 733-005.
Contacts and Locations
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 3, 2013 |
| 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 Hyperlipidemias Dyslipidemias Lipid Metabolism Disorders |
Metabolic Diseases Lipid Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Hyperlipoproteinemias |
ClinicalTrials.gov processed this record on May 23, 2013