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Trial record 43 of 153 for:    "familial hypercholesterolemia"

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

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ClinicalTrials.gov Identifier: NCT00730236
Recruitment Status : Completed
First Posted : August 8, 2008
Results First Posted : February 22, 2013
Last Update Posted : March 20, 2018
Sponsor:
Collaborator:
FDA Office of Orphan Products Development
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Homozygous Familial Hypercholesterolemia
Intervention Drug: AEGR-733
Enrollment 29

Recruitment Details 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 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.
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 29
Completed 23
Not Completed 6
Reason Not Completed
Adverse Event             4
Withdrawal by Subject             1
Non-compliance             1
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 29
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 29 participants
30.7  (10.64)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
Female
13
  44.8%
Male
16
  55.2%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
   6.9%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
   3.4%
White
25
  86.2%
More than one race
0
   0.0%
Unknown or Not Reported
1
   3.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 29 participants
United States 7
Canada 5
South Africa 11
Italy 6
1.Primary Outcome
Title Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)
Hide Description Percent change from Baseline in LDL-C
Time Frame Baseline and Week 26
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intention To Treat (ITT) Population
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: Percent Change
-40.1  (31.25)
2.Secondary Outcome
Title Percent Change From Baseline in Total Cholesterol (TC)
Hide Description Percent change from Baseline in TC
Time Frame Baseline and Week 26
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: Percent Change
-36.4  (28.2)
3.Secondary Outcome
Title Percent Change From Baseline for Apolipoprotein B (Apo B)
Hide Description Percent change from Baseline for Apo B
Time Frame Baseline and Week 26
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: Percent Change
-39.4  (30.01)
4.Secondary Outcome
Title Percent Change From Baseline in Triglycerides
Hide Description Percent change from Baseline in triglycerides
Time Frame Baseline and Week 26
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: Percent Change
-29.0  (55.72)
5.Secondary Outcome
Title Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C)
Hide Description Percent change from Baseline in HDL-C
Time Frame Baseline and Week 26
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Median (Standard Deviation)
Unit of Measure: Percent Change
-6.9  (19.76)
6.Secondary Outcome
Title Percent Change From Baseline in Non-HDL-C
Hide Description Percent change from Baseline in non-HDL-C
Time Frame Baseline and Week 26
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: Percent Change
-40.0  (29.66)
7.Secondary Outcome
Title Percent Change From Baseline in Apolipoprotein AI (Apo AI)
Hide Description Percent change from Baseline in Apo AI
Time Frame Baseline and Week 26
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: Percent Change
-6.5  (16.12)
8.Secondary Outcome
Title Absolute Change From Baseline in Hepatic Fat Percent
Hide Description Absolute change from Baseline in hepatic fat percent
Time Frame Baseline and Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: Percent Hepatic Fat
6.9  (5.03)
9.Secondary Outcome
Title Absolute Change From Baseline in Alanine Aminotransferase (ALT)
Hide Description Absolute change from Baseline in ALT
Time Frame Baseline and Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: U/L
15.0  (29.05)
10.Secondary Outcome
Title Absolute Change From Baseline in Aspartate Aminotransferase (AST)
Hide Description Absolute change from Baseline in AST
Time Frame Baseline and Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: U/L
8.9  (20.22)
11.Secondary Outcome
Title Absolute Change From Baseline in Total Bilirubin
Hide Description Absolute change from Baseline in total bilirubin
Time Frame Baseline and Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: mg/dL
0.1  (0.30)
12.Secondary Outcome
Title Absolute Change From Baseline in Weight
Hide Description Absolute change from Baseline in weight
Time Frame Baseline and Week 78
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 29
Mean (Standard Deviation)
Unit of Measure: kg
-2.3  (3.46)
Time Frame First dose to 28 days post treatment
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description 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.
All-Cause Mortality
Lomitapide Escalated
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Lomitapide Escalated
Affected / at Risk (%)
Total   3/29 (10.34%) 
Cardiac disorders   
Angina pectoris * 1  1/29 (3.45%) 
Coronary artery arteriosclerosis * 1  1/29 (3.45%) 
Acute coronary syndrome * 1  1/29 (3.45%) 
Infections and infestations   
Lower respiratory tract infection * 1  1/29 (3.45%) 
Reproductive system and breast disorders   
Menorrhagia * 1  1/29 (3.45%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 14.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Lomitapide Escalated
Affected / at Risk (%)
Total   23/29 (79.31%) 
Blood and lymphatic system disorders   
Anaemia * 1  2/29 (6.90%) 
Lymphadenopathy * 1  2/29 (6.90%) 
Cardiac disorders   
Palpitations * 1  3/29 (10.34%) 
Angina pectoris * 1  2/29 (6.90%) 
Eye disorders   
Conjunctivitis * 1  2/29 (6.90%) 
Gastrointestinal disorders   
Diarrhoea * 1  23/29 (79.31%) 
Nausea * 1  19/29 (65.52%) 
Vomiting * 1  10/29 (34.48%) 
Dyspepsia * 1  10/29 (34.48%) 
Abdominal discomfort * 1  9/29 (31.03%) 
Abdominal pain * 1  8/29 (27.59%) 
Abdominal pain upper * 1  5/29 (17.24%) 
Constipation * 1  6/29 (20.69%) 
Flatulence * 1  6/29 (20.69%) 
Abdominal distension * 1  5/29 (17.24%) 
Rectal tenesmus * 1  3/29 (10.34%) 
Defaecation urgency * 1  2/29 (6.90%) 
Eructation * 1  2/29 (6.90%) 
Gastritis * 1  2/29 (6.90%) 
Gastrooesophageal reflux disease * 1  2/29 (6.90%) 
Gingivitis * 1  2/29 (6.90%) 
General disorders   
Chest pain * 1  7/29 (24.14%) 
Fatigue * 1  5/29 (17.24%) 
Pyrexia * 1  3/29 (10.34%) 
Hepatobiliary disorders   
Hepatic steatosis * 1  2/29 (6.90%) 
Infections and infestations   
Influenza * 1  6/29 (20.69%) 
Nasopharyngitis * 1  5/29 (17.24%) 
Gastroenteritis * 1  4/29 (13.79%) 
Bronchitis * 1  2/29 (6.90%) 
Upper respiratory tract infection * 1  2/29 (6.90%) 
Injury, poisoning and procedural complications   
Laceration * 1  2/29 (6.90%) 
Limb injury * 1  2/29 (6.90%) 
Thermal burn * 1  2/29 (6.90%) 
Investigations   
Alanine aminotransferase increased * 1  5/29 (17.24%) 
Weight decreased * 1  7/29 (24.14%) 
Aspartate aminotransferase increased * 1  2/29 (6.90%) 
Transaminases increased * 1  2/29 (6.90%) 
Metabolism and nutrition disorders   
Decreased appetite * 1  2/29 (6.90%) 
Musculoskeletal and connective tissue disorders   
Arthralgia * 1  2/29 (6.90%) 
Back pain * 1  4/29 (13.79%) 
Myalgia * 1  2/29 (6.90%) 
Pain in extremity * 1  2/29 (6.90%) 
Musculoskeletal pain * 1  2/29 (6.90%) 
Nervous system disorders   
Headache * 1  3/29 (10.34%) 
Dizziness * 1  3/29 (10.34%) 
Paraesthesia * 1  2/29 (6.90%) 
Psychiatric disorders   
Anxiety * 1  2/29 (6.90%) 
Depression * 1  2/29 (6.90%) 
Respiratory, thoracic and mediastinal disorders   
Oropharyngeal pain * 1  4/29 (13.79%) 
Nasal congestion * 1  3/29 (10.34%) 
Cough * 1  2/29 (6.90%) 
Epistaxis * 1  2/29 (6.90%) 
Vascular disorders   
Hot flush * 1  2/29 (6.90%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 14.0
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 CTAs generally envision a multisite publication, with the PI’s right to publish individually if the pooled publication does not occur within 12 months of study completion. Sponsor has a 45 to 60 day review/approval period to request deletion of confidential information or to request limited deferral to protect its proprietary technology. In one case, the publication provision is more general, specifying that the Sponsor and clinical site will agree on the manner/terms of publication.
Results Point of Contact
Name/Title: Mark Sumeray, MD, Chief Medical Officer
Organization: Aegerion Pharmaceuticals
Phone: 617-500-7867
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: