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

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ClinicalTrials.gov Identifier: NCT01556906
Recruitment Status : Completed
First Posted : March 19, 2012
Results First Posted : February 22, 2013
Last Update Posted : April 10, 2013
Sponsor:
Collaborators:
University of Pennsylvania
Doris Duke Charitable Foundation
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Homozygous Familial Hypercholesterolemia
Intervention Drug: Lomitapide
Enrollment 6
Recruitment Details The study was performed from 05 Jun 2003 to 16 Feb 2004. The study was performed at a single medical clinic.
Pre-assignment Details  
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 6
Completed 6
Not Completed 0
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants
25.7  (9.43)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
3
  50.0%
Male
3
  50.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
  16.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
3
  50.0%
More than one race
0
   0.0%
Unknown or Not Reported
2
  33.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 6 participants
6
1.Primary Outcome
Title LDL-C
Hide Description Percent change in LDL-C compared to Baseline.
Time Frame Up to 16 weeks of treatment comapred to Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: percentage change in LDL-C
-50.94  (9.311)
2.Secondary Outcome
Title Absolute Change From Baseline in Alanine Aminotransferase (ALT)
Hide Description Absolute change from Baseline in ALT
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: U/L
91.2  (85.53)
3.Secondary Outcome
Title Absolute Change From Baseline in Aspartate Aminotransferase (AST)
Hide Description Absolute change from Baseline in AST
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: U/L
37.5  (32.51)
4.Secondary Outcome
Title Absolute Change From Baseline in Total Bilirubin
Hide Description Absolute change from Baseline in total bilirubin
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mg/dL
-0.25  (0.274)
5.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 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: percent of hapatic fat
19.3  (12.92)
6.Secondary Outcome
Title Absolute Change From Baseline in Forced Expiratory Volume During 1 Second (FEV1)
Hide Description Absolute change from Baseline in FEV1
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: Liters
0.070  (0.2406)
7.Secondary Outcome
Title Absolute Change From Baseline in Carbon Monoxide Lung Diffusing Capacity (DLCO)(a Pulmonary Function Test)
Hide Description Absolute change from Baseline in DLCO
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mL CO/min/mm Hg
-3.020  (5.3246)
8.Secondary Outcome
Title Absolute Change From Baseline in Vitamin A
Hide Description Absolute change from Baseline in vitamin A
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: µmol/L
-0.35  (0.847)
9.Secondary Outcome
Title Absolute Change From Baseline in Vitamin E
Hide Description Absolute change from Baseline in vitamin E
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: umol/L
-94.35  (130.797)
10.Secondary Outcome
Title Absolute Change From Baseline in Vitamin D
Hide Description Absolute Change From Baseline in Vitamin D
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: nmol/L
-6.57  (10.712)
11.Secondary Outcome
Title Absolute Change From Baseline in Ratio of Vitamin E to Total Lipids
Hide Description Absolute Change From Baseline in ratio of vitamin E to total lipids
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: ratio
-0.0  (1.71)
12.Secondary Outcome
Title Absolute Change From Baseline in Alpha Linoleic Acid (ALA)
Hide Description Absolute Change From Baseline in ALA
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mg/mL
0.06  (0.045)
13.Secondary Outcome
Title Absolute Change From Baseline in Eicosapentaenoic Acid (EPA)
Hide Description Absolute Change From Baseline in EPA
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mg/mL
-0.08  (0.079)
14.Secondary Outcome
Title Absolute Change From Baseline in Docosahexaenoic Acid (DHA)
Hide Description Absolute Change From Baseline in DHA
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mg/mL
-0.08  (0.045)
15.Secondary Outcome
Title Absolute Change From Baseline in Linoleic Acid (LA)
Hide Description Absolute Change From Baseline in LA
Time Frame Baseline and 16 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All patients treated
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mg/mL
-2.9  (1.51)
Time Frame First dose through 28 days post-treatment
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Lomitapide Escalated
Hide Arm/Group Description 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.
All-Cause Mortality
Lomitapide Escalated
Affected / at Risk (%)
Total   --/--    
Hide Serious Adverse Events
Lomitapide Escalated
Affected / at Risk (%) # Events
Total   1/6 (16.67%)    
Reproductive system and breast disorders   
Breast mass * 1  1/6 (16.67%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 14.0
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Lomitapide Escalated
Affected / at Risk (%) # Events
Total   6/6 (100.00%)    
Eye disorders   
Eye disorders * 1  1/6 (16.67%) 
Gastrointestinal disorders   
Diarrhoea * 1  5/6 (83.33%) 
Nausea * 1  2/6 (33.33%) 
Vomiting * 1  2/6 (33.33%) 
Dyspepsia * 1  1/6 (16.67%) 
Abdominal pain upper * 1  2/6 (33.33%) 
Constipation * 1  1/6 (16.67%) 
General disorders   
Fatigue * 1  2/6 (33.33%) 
Asthenia * 1  1/6 (16.67%) 
Chest pain * 1  1/6 (16.67%) 
Infections and infestations   
Rhinitis * 1  1/6 (16.67%) 
Injury, poisoning and procedural complications   
Contusion * 1  1/6 (16.67%) 
Scratch * 1  1/6 (16.67%) 
Investigations   
Alinine aminotransferase increased * 1  3/6 (50.00%) 
Aspartate aminotransferase increased * 1  3/6 (50.00%) 
International normalized ratio increased * 1  2/6 (33.33%) 
Prothrombin time prolonged * 1  1/6 (16.67%) 
Metabolism and nutrition disorders   
Decreased appetite * 1  1/6 (16.67%) 
Musculoskeletal and connective tissue disorders   
Arthralgia * 1  2/6 (33.33%) 
Myalgia * 1  1/6 (16.67%) 
Neck pain * 1  1/6 (16.67%) 
Pain in extremity * 1  1/6 (16.67%) 
Nervous system disorders   
Headache * 1  3/6 (50.00%) 
Dizziness * 1  1/6 (16.67%) 
Renal and urinary disorders   
Haemoglobinuria * 1  1/6 (16.67%) 
Reproductive system and breast disorders   
Breast mass * 1  1/6 (16.67%) 
Ovarian cyst * 1  1/6 (16.67%) 
Respiratory, thoracic and mediastinal disorders   
Oropharyngeal pain * 1  4/6 (66.67%) 
Nasal congestion * 1  2/6 (33.33%) 
Lung disorder * 1  4/6 (66.67%) 
Cough * 1  2/6 (33.33%) 
Dyspnoea * 1  1/6 (16.67%) 
Sneezing * 1  1/6 (16.67%) 
Skin and subcutaneous tissue disorders   
Xanthoma * 1  1/6 (16.67%) 
Acne * 1  1/6 (16.67%) 
Hyperhidrosis * 1  1/6 (16.67%) 
Rash * 1  1/6 (16.67%) 
Vascular disorders   
Hot flush * 1  1/6 (16.67%) 
*
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.
Information is unavailable.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Chief Medical Officer
Organization: Aegerion Pharmaceutical
Phone: 617-500-7867
Layout table for additonal information
Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01556906    
Other Study ID Numbers: UP1001
First Submitted: March 7, 2012
First Posted: March 19, 2012
Results First Submitted: January 18, 2013
Results First Posted: February 22, 2013
Last Update Posted: April 10, 2013