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

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ClinicalTrials.gov Identifier: NCT00943306
Recruitment Status : Completed
First Posted : July 22, 2009
Results First Posted : May 10, 2018
Last Update Posted : May 10, 2018
Sponsor:
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Familial Hypercholesterolemia
Intervention: Drug: lomitapide

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Lomitapide

Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.

lomitapide: 5-60 mg po every day


Participant Flow:   Overall Study
    Lomitapide
STARTED   19 
COMPLETED   16 
NOT COMPLETED   3 
Physician Decision                1 
Death                1 
Sponsor decision due to non-compliance                1 



  Baseline Characteristics

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.
Baseline from initiation of treatment in Study 733-005/UP1002 (Safety Population)

Reporting Groups
  Description
Lomitapide

Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.

lomitapide: 5-60 mg po every day


Baseline Measures
   Lomitapide 
Overall Participants Analyzed 
[Units: Participants]
 19 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      0   0.0% 
Between 18 and 65 years      19 100.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 30.4  (11.74) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      9  47.4% 
Male      10  52.6% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      0   0.0% 
Asian      1   5.3% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      0   0.0% 
White      17  89.5% 
More than one race      0   0.0% 
Unknown or Not Reported      1   5.3% 
Region of Enrollment 
[Units: Participants]
 
Canada   2 
United States   3 
South Africa   9 
Italy   5 


  Outcome Measures

1.  Primary:   Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)   [ Time Frame: Baseline and Week 126 ]

2.  Secondary:   Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)   [ Time Frame: Baseline and Week 174 ]

3.  Secondary:   Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)   [ Time Frame: Baseline and Week 222 ]

4.  Secondary:   Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)   [ Time Frame: Baseline and Week 246 ]

5.  Secondary:   Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)   [ Time Frame: Baseline and Week 270 ]

6.  Secondary:   Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)   [ Time Frame: Baseline and Week 294 ]

7.  Secondary:   Percent Change in Total Cholesterol   [ Time Frame: Baseline and Week 126 ]

8.  Secondary:   Percent Change in Total Cholesterol   [ Time Frame: Baseline and Week 174 ]

9.  Secondary:   Percent Change in Total Cholesterol   [ Time Frame: Baseline and Week 222 ]

10.  Secondary:   Percent Change in Total Cholesterol   [ Time Frame: Baseline and Week 246 ]

11.  Secondary:   Percent Change in Total Cholesterol   [ Time Frame: Baseline and Week 270 ]

12.  Secondary:   Percent Change in Total Cholesterol   [ Time Frame: Baseline and Week 294 ]

13.  Secondary:   Percent Change in Apolipoprotein B (Apo B)   [ Time Frame: Baseline and Week 126 ]

14.  Secondary:   Percent Change in Apolipoprotein B (Apo B)   [ Time Frame: Baseline and Week 174 ]

15.  Secondary:   Percent Change in Apolipoprotein B (Apo B)   [ Time Frame: Baseline and Week 222 ]

16.  Secondary:   Percent Change in Apolipoprotein B (Apo B)   [ Time Frame: Baseline and Week 246 ]

17.  Secondary:   Percent Change in Apolipoprotein B (Apo B)   [ Time Frame: Baseline and Week 270 ]

18.  Secondary:   Percent Change in Apolipoprotein B (Apo B)   [ Time Frame: Baseline and Week 294 ]

19.  Secondary:   Percent Change in Triglycerides   [ Time Frame: Baseline and Week 126 ]

20.  Secondary:   Percent Change in Triglycerides   [ Time Frame: Baseline and Week 174 ]

21.  Secondary:   Percent Change in Triglycerides   [ Time Frame: Baseline and Week 222 ]

22.  Secondary:   Percent Change in Triglycerides   [ Time Frame: Baseline and Week 246 ]

23.  Secondary:   Percent Change in Triglycerides   [ Time Frame: Baseline and Week 270 ]

24.  Secondary:   Percent Change in Triglycerides   [ Time Frame: Baseline and Week 294 ]

25.  Secondary:   Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)   [ Time Frame: Baseline and Week 126 ]

26.  Secondary:   Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)   [ Time Frame: Baseline and Week 174 ]

27.  Secondary:   Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)   [ Time Frame: Baseline and Week 222 ]

28.  Secondary:   Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)   [ Time Frame: Baseline and Week 246 ]

29.  Secondary:   Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)   [ Time Frame: Baseline and Week 270 ]

30.  Secondary:   Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)   [ Time Frame: Baseline and Week 294 ]

31.  Secondary:   Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)   [ Time Frame: Baseline and Week 126 ]

32.  Secondary:   Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)   [ Time Frame: Baseline and Week 174 ]

33.  Secondary:   Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)   [ Time Frame: Baseline and Week 222 ]

34.  Secondary:   Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)   [ Time Frame: Baseline and Week 246 ]

35.  Secondary:   Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)   [ Time Frame: Baseline and Week 270 ]

36.  Secondary:   Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)   [ Time Frame: Baseline and Week 294 ]

37.  Secondary:   Percent Change in Lp(a)   [ Time Frame: Baseline and Week 126 ]

38.  Secondary:   Percent Change in Lp(a)   [ Time Frame: Baseline and Week 174 ]

39.  Secondary:   Percent Change in Lp(a)   [ Time Frame: Baseline and Week 222 ]

40.  Secondary:   Percent Change in Lp(a)   [ Time Frame: Baseline and Week 246 ]

41.  Secondary:   Percent Change in Lp(a)   [ Time Frame: Baseline and Week 270 ]

42.  Secondary:   Percent Change in Lp(a)   [ Time Frame: Baseline and Week 294 ]

43.  Secondary:   Percent Change in High Density Lipoprotein Cholesterol (HDL-C)   [ Time Frame: Baseline and Week 126 ]

44.  Secondary:   Percent Change in High Density Lipoprotein Cholesterol (HDL-C)   [ Time Frame: Baseline and Week 174 ]

45.  Secondary:   Percent Change in High Density Lipoprotein Cholesterol (HDL-C)   [ Time Frame: Baseline and Week 222 ]

46.  Secondary:   Percent Change in High Density Lipoprotein Cholesterol (HDL-C)   [ Time Frame: Baseline and Week 246 ]

47.  Secondary:   Percent Change in High Density Lipoprotein Cholesterol (HDL-C)   [ Time Frame: Baseline and Week 270 ]

48.  Secondary:   Percent Change in High Density Lipoprotein Cholesterol (HDL-C)   [ Time Frame: Baseline and Week 294 ]

49.  Secondary:   Percent Change in Apolipoprotein AI (Apo AI)   [ Time Frame: Baseline and Week 126 ]

50.  Secondary:   Percent Change in Apolipoprotein AI (Apo AI)   [ Time Frame: Baseline and Week 174 ]

51.  Secondary:   Percent Change in Apolipoprotein AI (Apo AI)   [ Time Frame: Baseline and Week 222 ]

52.  Secondary:   Percent Change in Apolipoprotein AI (Apo AI)   [ Time Frame: Baseline and Week 246 ]

53.  Secondary:   Percent Change in Apolipoprotein AI (Apo AI)   [ Time Frame: Baseline and Week 270 ]

54.  Secondary:   Percent Change in Apolipoprotein AI (Apo AI)   [ Time Frame: Baseline and Week 294 ]


  Serious Adverse Events


  Other Adverse Events


  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.


  More Information

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: Alison Long, MD - VP Clinical
Organization: Aegerion Pharmaceuticals, Inc.
phone: 857-242-5142
e-mail: alison.long@aegerion.com


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: AEGR-733-012
First Submitted: July 21, 2009
First Posted: July 22, 2009
Results First Submitted: February 29, 2016
Results First Posted: May 10, 2018
Last Update Posted: May 10, 2018