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Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor

This study has been completed.
Sponsor:
Collaborators:
University of Pennsylvania
Doris Duke Charitable Foundation
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT01556906
First received: March 7, 2012
Last updated: April 4, 2013
Last verified: April 2013
History of Changes
Results First Received: January 18, 2013  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Homozygous Familial Hypercholesterolemia
Intervention: Drug: Lomitapide

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
The study was performed from 05 Jun 2003 to 16 Feb 2004. The study was performed at a single medical clinic.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
None

Reporting Groups
  Description
Lomitapide Escalated Lomitapide initiated with an oral dose of 0.03 mg/kg/day for 4 weeks and then escalated through an additional 3 dose levels (0.1, 0.3, and 1.0 mg/kg/day) every 4 weeks over a 16-week period.

Participant Flow:   Overall Study
    Lomitapide Escalated  
STARTED     6  
COMPLETED     6  
NOT COMPLETED     0  



  Baseline Characteristics
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Reporting Groups
  Description
Lomitapide Escalated Lomitapide initiated with an oral dose of 0.03 mg/kg/day for 4 weeks and then escalated through an additional 3 dose levels (0.1, 0.3, and 1.0 mg/kg/day) every 4 weeks over a 16-week period.

Baseline Measures
    Lomitapide Escalated  
Number of Participants  
[units: participants]
 		  6  
Age  
[units: years]
Mean ± Standard Deviation 		  25.7  ± 9.43  
Gender  
[units: participants]
 		 
Female     3  
Male     3  
Race (NIH/OMB)  
[units: participants]
 		 
American Indian or Alaska Native     0  
Asian     1  
Native Hawaiian or Other Pacific Islander     0  
Black or African American     0  
White     3  
More than one race     0  
Unknown or Not Reported     2  
Region of Enrollment  
[units: participants]
 		 
United States     6  



  Outcome Measures
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1.  Primary:   LDL-C   [ Time Frame: Up to 16 weeks of treatment comapred to Baseline ]
  Show Outcome Measure 1

2.  Secondary:   Absolute Change From Baseline in Alanine Aminotransferase (ALT)   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 2

3.  Secondary:   Absolute Change From Baseline in Aspartate Aminotransferase (AST)   [ Time Frame: Baseline and 16 weeks of treatment ]
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4.  Secondary:   Absolute Change From Baseline in Total Bilirubin   [ Time Frame: Baseline and 16 weeks of treatment ]
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5.  Secondary:   Absolute Change From Baseline in Hepatic Fat Percent   [ Time Frame: Baseline and 16 weeks of treatment ]
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6.  Secondary:   Absolute Change From Baseline in Forced Expiratory Volume During 1 Second (FEV1)   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 6

7.  Secondary:   Absolute Change From Baseline in Carbon Monoxide Lung Diffusing Capacity (DLCO)(a Pulmonary Function Test)   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 7

8.  Secondary:   Absolute Change From Baseline in Vitamin A   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 8

9.  Secondary:   Absolute Change From Baseline in Vitamin E   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 9

10.  Secondary:   Absolute Change From Baseline in Vitamin D   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 10

11.  Secondary:   Absolute Change From Baseline in Ratio of Vitamin E to Total Lipids   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 11

12.  Secondary:   Absolute Change From Baseline in Alpha Linoleic Acid (ALA)   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 12

13.  Secondary:   Absolute Change From Baseline in Eicosapentaenoic Acid (EPA)   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 13

14.  Secondary:   Absolute Change From Baseline in Docosahexaenoic Acid (DHA)   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 14

15.  Secondary:   Absolute Change From Baseline in Linoleic Acid (LA)   [ Time Frame: Baseline and 16 weeks of treatment ]
  Show Outcome Measure 15


  Serious Adverse Events
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  Other Adverse Events
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.  


Results Point of Contact:  
Name/Title: Chief Medical Officer
Organization: Aegerion Pharmaceutical
phone: 617-500-7867


Publications of Results:


Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01556906     History of Changes
Other Study ID Numbers: UP1001
Study First Received: March 7, 2012
Results First Received: January 18, 2013
Last Updated: April 4, 2013
Health Authority: United States: Food and Drug Administration