A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT00730236
First received: August 6, 2008
Last updated: January 15, 2014
Last verified: January 2013
Results First Received: January 18, 2013  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Homozygous Familial Hypercholesterolemia
Intervention: Drug: AEGR-733

  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 18 Dec 2007 to 13 Oct 2011. A total of 11 medical clinics participated in the study.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
6-week Run-in Phase. Following screening, patients entered a 6-week Run-in Phase to stabilize their regimen of current lipid-lowering therapy(ies) and to be placed on a low-fat diet containing <20% energy from fat.

Reporting Groups
  Description
Lomitapide Escalated Lomitapide escalated with an initial oral dose of 5 mg/day for 2 weeks and then escalated at 4 week intervals to 60 mg/day. In rare situations (1 patient)who met strict safety and efficacy criteria could have their dose escalated to 80 mg/day.

Participant Flow:   Overall Study
    Lomitapide Escalated  
STARTED     29  
COMPLETED     23  
NOT COMPLETED     6  
Adverse Event                 4  
Withdrawal by Subject                 1  
Non-compliance                 1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Lomitapide Escalated Lomitapide escalated with an initial oral dose of 5 mg/day for 2 weeks and then escalated at 4 week intervals to 60 mg/day. In rare situations (1 patient)who met strict safety and efficacy criteria could have their dose escalated to 80 mg/day.

Baseline Measures
    Lomitapide Escalated  
Number of Participants  
[units: participants]
  29  
Age  
[units: years]
Mean ± Standard Deviation
  30.7  ± 10.64  
Gender  
[units: participants]
 
Female     13  
Male     16  
Race (NIH/OMB)  
[units: Participants]
 
American Indian or Alaska Native     0  
Asian     2  
Native Hawaiian or Other Pacific Islander     0  
Black or African American     1  
White     25  
More than one race     0  
Unknown or Not Reported     1  
Region of Enrollment  
[units: participants]
 
United States     7  
Canada     5  
South Africa     11  
Italy     6  



  Outcome Measures
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1.  Primary:   Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)   [ Time Frame: Baseline and Week 26 ]

2.  Secondary:   Percent Change From Baseline in Total Cholesterol (TC)   [ Time Frame: Baseline and Week 26 ]

3.  Secondary:   Percent Change From Baseline for Apolipoprotein B (Apo B)   [ Time Frame: Baseline and Week 26 ]

4.  Secondary:   Percent Change From Baseline in Triglycerides   [ Time Frame: Baseline and Week 26 ]

5.  Secondary:   Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C)   [ Time Frame: Baseline and Week 26 ]

6.  Secondary:   Percent Change From Baseline in Non-HDL-C   [ Time Frame: Baseline and Week 26 ]

7.  Secondary:   Percent Change From Baseline in Apolipoprotein AI (Apo AI)   [ Time Frame: Baseline and Week 26 ]

8.  Secondary:   Absolute Change From Baseline in Hepatic Fat Percent   [ Time Frame: Baseline and Week 78 ]

9.  Secondary:   Absolute Change From Baseline in Alanine Aminotransferase (ALT)   [ Time Frame: Baseline and Week 78 ]

10.  Secondary:   Absolute Change From Baseline in Aspartate Aminotransferase (AST)   [ Time Frame: Baseline and Week 78 ]

11.  Secondary:   Absolute Change From Baseline in Total Bilirubin   [ Time Frame: Baseline and Week 78 ]

12.  Secondary:   Absolute Change From Baseline in Weight   [ Time Frame: Baseline and Week 78 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
None


  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.


Results Point of Contact:  
Name/Title: Mark Sumeray, MD, Chief Medical Officer
Organization: Aegerion Pharmaceuticals
phone: 617-500-7867
e-mail: msumeray@aegerion.com


Publications of Results:
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

Other Publications:

Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT00730236     History of Changes
Obsolete Identifiers: NCT00603161
Other Study ID Numbers: 733-005 / UP1002
Study First Received: August 6, 2008
Results First Received: January 18, 2013
Last Updated: January 15, 2014
Health Authority: South Africa: Department of Health
Canada: Health Canada
United States: Food and Drug Administration