Trial Outcomes & Findings for A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH) (NCT NCT00730236)
NCT ID: NCT00730236
Last Updated: 2018-03-20
Results Overview
Percent change from Baseline in LDL-C
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
29 participants
Primary outcome timeframe
Baseline and Week 26
Results posted on
2018-03-20
Participant Flow
The study was performed from 18 Dec 2007 to 13 Oct 2011. A total of 11 medical clinics participated in the study.
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.
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
29
|
|
Overall Study
COMPLETED
|
23
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
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.
|
|---|---|
|
Overall Study
Adverse Event
|
4
|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Non-compliance
|
1
|
Baseline Characteristics
A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)
Baseline characteristics by cohort
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Age, Continuous
|
30.7 years
STANDARD_DEVIATION 10.64 • n=93 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
25 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=93 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=93 Participants
|
|
Region of Enrollment
Canada
|
5 participants
n=93 Participants
|
|
Region of Enrollment
South Africa
|
11 participants
n=93 Participants
|
|
Region of Enrollment
Italy
|
6 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 26Population: Intention To Treat (ITT) Population
Percent change from Baseline in LDL-C
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)
|
-40.1 Percent Change
Standard Deviation 31.25
|
SECONDARY outcome
Timeframe: Baseline and Week 26Population: ITT Population
Percent change from Baseline in TC
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Total Cholesterol (TC)
|
-36.4 Percent Change
Standard Deviation 28.2
|
SECONDARY outcome
Timeframe: Baseline and Week 26Population: ITT Population
Percent change from Baseline for Apo B
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Percent Change From Baseline for Apolipoprotein B (Apo B)
|
-39.4 Percent Change
Standard Deviation 30.01
|
SECONDARY outcome
Timeframe: Baseline and Week 26Population: ITT Population
Percent change from Baseline in triglycerides
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Triglycerides
|
-29.0 Percent Change
Standard Deviation 55.72
|
SECONDARY outcome
Timeframe: Baseline and Week 26Population: ITT Population
Percent change from Baseline in HDL-C
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C)
|
-6.9 Percent Change
Standard Deviation 19.76
|
SECONDARY outcome
Timeframe: Baseline and Week 26Population: ITT Population
Percent change from Baseline in non-HDL-C
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Non-HDL-C
|
-40.0 Percent Change
Standard Deviation 29.66
|
SECONDARY outcome
Timeframe: Baseline and Week 26Population: ITT Population
Percent change from Baseline in Apo AI
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Apolipoprotein AI (Apo AI)
|
-6.5 Percent Change
Standard Deviation 16.12
|
SECONDARY outcome
Timeframe: Baseline and Week 78Population: All patients treated
Absolute change from Baseline in hepatic fat percent
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Hepatic Fat Percent
|
6.9 Percent Hepatic Fat
Standard Deviation 5.03
|
SECONDARY outcome
Timeframe: Baseline and Week 78Population: All patients treated
Absolute change from Baseline in ALT
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Alanine Aminotransferase (ALT)
|
15.0 U/L
Standard Deviation 29.05
|
SECONDARY outcome
Timeframe: Baseline and Week 78Population: All patients treated
Absolute change from Baseline in AST
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Aspartate Aminotransferase (AST)
|
8.9 U/L
Standard Deviation 20.22
|
SECONDARY outcome
Timeframe: Baseline and Week 78Population: All patients treated
Absolute change from Baseline in total bilirubin
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Total Bilirubin
|
0.1 mg/dL
Standard Deviation 0.30
|
SECONDARY outcome
Timeframe: Baseline and Week 78Population: All patients treated
Absolute change from Baseline in weight
Outcome measures
| Measure |
Lomitapide Escalated
n=29 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Weight
|
-2.3 kg
Standard Deviation 3.46
|
Adverse Events
Lomitapide Escalated
Serious events: 3 serious events
Other events: 23 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Lomitapide Escalated
n=29 participants at risk
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.
|
|---|---|
|
Cardiac disorders
Angina pectoris
|
3.4%
1/29 • First dose to 28 days post treatment
|
|
Infections and infestations
Lower respiratory tract infection
|
3.4%
1/29 • First dose to 28 days post treatment
|
|
Cardiac disorders
Coronary artery arteriosclerosis
|
3.4%
1/29 • First dose to 28 days post treatment
|
|
Reproductive system and breast disorders
Menorrhagia
|
3.4%
1/29 • First dose to 28 days post treatment
|
|
Cardiac disorders
Acute coronary syndrome
|
3.4%
1/29 • First dose to 28 days post treatment
|
Other adverse events
| Measure |
Lomitapide Escalated
n=29 participants at risk
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.
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
79.3%
23/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Nausea
|
65.5%
19/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Vomiting
|
34.5%
10/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Dyspepsia
|
34.5%
10/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Abdominal discomfort
|
31.0%
9/29 • First dose to 28 days post treatment
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
13.8%
4/29 • First dose to 28 days post treatment
|
|
Investigations
Alanine aminotransferase increased
|
17.2%
5/29 • First dose to 28 days post treatment
|
|
General disorders
Chest pain
|
24.1%
7/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Abdominal pain
|
27.6%
8/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Abdominal pain upper
|
17.2%
5/29 • First dose to 28 days post treatment
|
|
General disorders
Fatigue
|
17.2%
5/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Constipation
|
20.7%
6/29 • First dose to 28 days post treatment
|
|
Investigations
Weight decreased
|
24.1%
7/29 • First dose to 28 days post treatment
|
|
Nervous system disorders
Headache
|
10.3%
3/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Flatulence
|
20.7%
6/29 • First dose to 28 days post treatment
|
|
Infections and infestations
Influenza
|
20.7%
6/29 • First dose to 28 days post treatment
|
|
Investigations
Aspartate aminotransferase increased
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.3%
3/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Abdominal distension
|
17.2%
5/29 • First dose to 28 days post treatment
|
|
Infections and infestations
Nasopharyngitis
|
17.2%
5/29 • First dose to 28 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Nervous system disorders
Dizziness
|
10.3%
3/29 • First dose to 28 days post treatment
|
|
Infections and infestations
Gastroenteritis
|
13.8%
4/29 • First dose to 28 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
13.8%
4/29 • First dose to 28 days post treatment
|
|
Metabolism and nutrition disorders
Decreased appetite
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Vascular disorders
Hot flush
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Cardiac disorders
Palpitations
|
10.3%
3/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Rectal tenesmus
|
10.3%
3/29 • First dose to 28 days post treatment
|
|
General disorders
Pyrexia
|
10.3%
3/29 • First dose to 28 days post treatment
|
|
Blood and lymphatic system disorders
Anaemia
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Cardiac disorders
Angina pectoris
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Eye disorders
Conjunctivitis
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Defaecation urgency
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Eructation
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Gastritis
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Gastrointestinal disorders
Gingivitis
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Hepatobiliary disorders
Hepatic steatosis
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Infections and infestations
Bronchitis
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Infections and infestations
Upper respiratory tract infection
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Injury, poisoning and procedural complications
Laceration
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Injury, poisoning and procedural complications
Limb injury
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Injury, poisoning and procedural complications
Thermal burn
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Investigations
Transaminases increased
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Nervous system disorders
Paraesthesia
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Psychiatric disorders
Anxiety
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Psychiatric disorders
Depression
|
6.9%
2/29 • First dose to 28 days post treatment
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
6.9%
2/29 • First dose to 28 days post treatment
|
Additional Information
Mark Sumeray, MD, Chief Medical Officer
Aegerion Pharmaceuticals
Phone: 617-500-7867
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER