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Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy

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ClinicalTrials.gov Identifier: NCT02173158
Recruitment Status : Completed
First Posted : June 24, 2014
Results First Posted : October 10, 2018
Last Update Posted : October 10, 2018
Sponsor:
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 Familial Hypercholesterolemia - Homozygous
Intervention Drug: lomitapide
Enrollment 9
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Lomitapide
Hide Arm/Group Description Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Period Title: Efficacy Phase
Started 9
Completed 8
Not Completed 1
Reason Not Completed
Adverse Event             1
Period Title: Safety Phase
Started 8
Completed 8
Not Completed 0
Arm/Group Title Lomitapide
Hide Arm/Group Description Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 9 participants
50.3  (14.71)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
4
  44.4%
Male
5
  55.6%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
American Indian or Alaska Native
0
   0.0%
Asian
9
 100.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
0
   0.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
1.Primary Outcome
Title Percent Change in LDL-C
Hide Description Mean percent change from baseline
Time Frame Baseline to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set for the efficacy phase, included all subjects who received study drug and had a baseline and at least one post-baseline assessment
Arm/Group Title Lomitapide
Hide Arm/Group Description:
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: Percent change
-42.2  (18.16)
2.Secondary Outcome
Title Change in Total Cholesterol
Hide Description Mean percent change from baseline
Time Frame Baseline to Week 56
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Arm/Group Title Lomitapide
Hide Arm/Group Description:
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: Percent change
-25.9  (18.57)
3.Secondary Outcome
Title Change in Apo B
Hide Description Mean percent change from baseline
Time Frame Baseline to Week 56
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Arm/Group Title Lomitapide
Hide Arm/Group Description:
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: Percent change
-41.4  (21.90)
4.Secondary Outcome
Title Change in Triglycerides
Hide Description Mean percent change from baseline
Time Frame Baseline to Week 56
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Arm/Group Title Lomitapide
Hide Arm/Group Description:
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: Percent change
-44.4  (12.70)
5.Secondary Outcome
Title Change in Non-HDL-C
Hide Description Mean percent change from baseline
Time Frame Baseline to Week 56
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Arm/Group Title Lomitapide
Hide Arm/Group Description:
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: Percent change
-34.6  (22.50)
6.Secondary Outcome
Title Change in VLDL-C
Hide Description Mean percent change from baseline
Time Frame Baseline to Week 56
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Arm/Group Title Lomitapide
Hide Arm/Group Description:
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: Percent change
-44.8  (12.05)
7.Secondary Outcome
Title Change in Lp(a)
Hide Description Mean percent change from baseline
Time Frame Baseline to Week 56
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Arm/Group Title Lomitapide
Hide Arm/Group Description:
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: Percent change
-27.2  (23.50)
8.Secondary Outcome
Title Change in HDL-C
Hide Description Mean percent change from baseline
Time Frame Baseline to Week 56
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Arm/Group Title Lomitapide
Hide Arm/Group Description:
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: Percent change
5.9  (19.64)
9.Secondary Outcome
Title Change in Apo AI
Hide Description Mean percent change from baseline
Time Frame Baseline to Week 56
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Arm/Group Title Lomitapide
Hide Arm/Group Description:
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: Percent change
-2.8  (11.61)
10.Secondary Outcome
Title Change in LDL-C
Hide Description Mean percent change from baseline
Time Frame Baseline to Week 56
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Arm/Group Title Lomitapide
Hide Arm/Group Description:
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: Percent change
-37.5  (24.21)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Efficacy Phase Safety Phase
Hide Arm/Group Description Baseline to Week 26 Week 26 to Week 56
All-Cause Mortality
Efficacy Phase Safety Phase
Affected / at Risk (%) Affected / at Risk (%)
Total   0/9 (0.00%)      0/8 (0.00%)    
Hide Serious Adverse Events
Efficacy Phase Safety Phase
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/9 (0.00%)      1/8 (12.50%)    
General disorders     
Chest pain  1  0/9 (0.00%)  0 1/8 (12.50%)  1
1
Term from vocabulary, MedDRA 17.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Efficacy Phase Safety Phase
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   9/9 (100.00%)      7/8 (87.50%)    
Blood and lymphatic system disorders     
Anaemia  1  2/9 (22.22%)  2/8 (25.00%) 
Iron deficiency anaemia  1  0/9 (0.00%)  1/8 (12.50%) 
Cardiac disorders     
Palpitations  1  1/9 (11.11%)  0/8 (0.00%) 
Gastrointestinal disorders     
Diarrhoea  1  7/9 (77.78%)  4/8 (50.00%) 
Nausea  1  2/9 (22.22%)  1/8 (12.50%) 
Abdominal distension  1  1/9 (11.11%)  0/8 (0.00%) 
Abdominal pain lower  1  1/9 (11.11%)  0/8 (0.00%) 
Dental caries  1  1/9 (11.11%)  0/8 (0.00%) 
Faeces soft  1  1/9 (11.11%)  0/8 (0.00%) 
Flatulence  1  1/9 (11.11%)  0/8 (0.00%) 
Haemorrhoids  1  1/9 (11.11%)  1/8 (12.50%) 
General disorders     
Chest pain  1  0/9 (0.00%)  1/8 (12.50%) 
Device breakage  1  0/9 (0.00%)  1/8 (12.50%) 
Feeling abnormal  1  1/9 (11.11%)  1/8 (12.50%) 
Malaise  1  1/9 (11.11%)  0/8 (0.00%) 
Hepatobiliary disorders     
Hepatic function abnormal  1  1/9 (11.11%)  1/8 (12.50%) 
Infections and infestations     
Nasopharyngitis  1  4/9 (44.44%)  3/8 (37.50%) 
Gastroenteritis  1  2/9 (22.22%)  1/8 (12.50%) 
Influenza  1  1/9 (11.11%)  1/8 (12.50%) 
Periodontitis  1  1/9 (11.11%)  1/8 (12.50%) 
Pneumonia  1  1/9 (11.11%)  0/8 (0.00%) 
Injury, poisoning and procedural complications     
Chemical burn of skin  1  0/9 (0.00%)  1/8 (12.50%) 
Heat stroke  1  0/9 (0.00%)  1/8 (12.50%) 
Contusion  1  1/9 (11.11%)  0/8 (0.00%) 
Subcutaneous haematoma  1  1/9 (11.11%)  0/8 (0.00%) 
Tooth fracture  1  1/9 (11.11%)  0/8 (0.00%) 
Investigations     
Liver function test abnormal  1  3/9 (33.33%)  1/8 (12.50%) 
Blood creatine phosphokinase increased  1  1/9 (11.11%)  1/8 (12.50%) 
White blood cell count decreased  1  0/9 (0.00%)  1/8 (12.50%) 
Metabolism and nutrition disorders     
Decreased appetite  1  1/9 (11.11%)  0/8 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back pain  1  1/9 (11.11%)  0/8 (0.00%) 
Mylagia  1  1/9 (11.11%)  0/8 (0.00%) 
Nervous system disorders     
Headache  1  2/9 (22.22%)  0/8 (0.00%) 
Skin and subcutaneous tissue disorders     
Drug eruption  1  1/9 (11.11%)  0/8 (0.00%) 
Eczema  1  1/9 (11.11%)  0/8 (0.00%) 
Pruritus allergic  1  1/9 (11.11%)  0/8 (0.00%) 
Rash  1  1/9 (11.11%)  1/8 (12.50%) 
1
Term from vocabulary, MedDRA 17.0
Indicates events were collected by systematic assessment
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.
Described in site contract
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Alison Long, MD - VP Clinical
Organization: Aegerion Pharmaceuticals, Inc.
Phone: 857-242-5142
EMail: alison.long@aegerion.com
Layout table for additonal information
Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02173158    
Other Study ID Numbers: AEGR-733-030
First Submitted: June 23, 2014
First Posted: June 24, 2014
Results First Submitted: February 9, 2018
Results First Posted: October 10, 2018
Last Update Posted: October 10, 2018