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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| First Received Date ICMJE | July 21, 2009 | ||||||||
| Last Updated Date | January 3, 2013 | ||||||||
| Start Date ICMJE | September 2009 | ||||||||
| Estimated Primary Completion Date | December 2013 (final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures ICMJE |
Percent change in LDL-C [ Time Frame: 48 weeks ] [ Designated as safety issue: No ] | ||||||||
| Original Primary Outcome Measures ICMJE | Same as current | ||||||||
| Change History | Complete list of historical versions of study NCT00943306 on ClinicalTrials.gov Archive Site | ||||||||
| Current Secondary Outcome Measures ICMJE |
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| Original Secondary Outcome Measures ICMJE |
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| Current Other Outcome Measures ICMJE | Not Provided | ||||||||
| Original Other Outcome Measures ICMJE | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title ICMJE | Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia | ||||||||
| Official Title ICMJE | 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 | ||||||||
| Brief Summary | 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. |
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| 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. |
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| Study Type ICMJE | Interventional | ||||||||
| Study Phase | Phase 3 | ||||||||
| Study Design ICMJE | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
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| Condition ICMJE | Familial Hypercholesterolemia | ||||||||
| Intervention ICMJE | Drug: lomitapide
5-60 mg po every day
Other Names:
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| Study Arm (s) | Experimental: lomitapide
Maximum tolerated dose of lomitapide (up to 80mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Intervention: Drug: lomitapide |
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| Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status ICMJE | Active, not recruiting | ||||||||
| Estimated Enrollment ICMJE | 25 | ||||||||
| Estimated Completion Date | December 2014 | ||||||||
| Estimated Primary Completion Date | December 2013 (final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Both | ||||||||
| Ages | 18 Years and older | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||||
| Location Countries ICMJE | United States, Canada, Italy, South Africa | ||||||||
| Administrative Information | |||||||||
| NCT Number ICMJE | NCT00943306 | ||||||||
| Other Study ID Numbers ICMJE | 733-012 | ||||||||
| Has Data Monitoring Committee | No | ||||||||
| Responsible Party | Aegerion Pharmaceuticals, Inc. | ||||||||
| Study Sponsor ICMJE | Aegerion Pharmaceuticals, Inc. | ||||||||
| Collaborators ICMJE | Not Provided | ||||||||
| Investigators ICMJE |
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| Information Provided By | Aegerion Pharmaceuticals, Inc. | ||||||||
| Verification Date | March 2012 | ||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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