Trial Outcomes & Findings for Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor (NCT NCT01556906)
NCT ID: NCT01556906
Last Updated: 2013-04-10
Results Overview
Percent change in LDL-C compared to Baseline.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
Up to 16 weeks of treatment comapred to Baseline
Results posted on
2013-04-10
Participant Flow
The study was performed from 05 Jun 2003 to 16 Feb 2004. The study was performed at a single medical clinic.
Participant milestones
| Measure |
Lomitapide Escalated
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 Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor
Baseline characteristics by cohort
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Age Continuous
|
25.7 years
STANDARD_DEVIATION 9.43 • n=99 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Up to 16 weeks of treatment comapred to BaselinePopulation: All patients treated
Percent change in LDL-C compared to Baseline.
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
LDL-C
|
-50.94 percentage change in LDL-C
Standard Deviation 9.311
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute change from Baseline in ALT
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Alanine Aminotransferase (ALT)
|
91.2 U/L
Standard Deviation 85.53
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute change from Baseline in AST
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Aspartate Aminotransferase (AST)
|
37.5 U/L
Standard Deviation 32.51
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute change from Baseline in total bilirubin
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Total Bilirubin
|
-0.25 mg/dL
Standard Deviation 0.274
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute change from Baseline in hepatic fat percent
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Hepatic Fat Percent
|
19.3 percent of hapatic fat
Standard Deviation 12.92
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute change from Baseline in FEV1
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Forced Expiratory Volume During 1 Second (FEV1)
|
0.070 Liters
Standard Deviation 0.2406
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute change from Baseline in DLCO
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Carbon Monoxide Lung Diffusing Capacity (DLCO)(a Pulmonary Function Test)
|
-3.020 mL CO/min/mm Hg
Standard Deviation 5.3246
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute change from Baseline in vitamin A
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Vitamin A
|
-0.35 µmol/L
Standard Deviation 0.847
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute change from Baseline in vitamin E
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Vitamin E
|
-94.35 umol/L
Standard Deviation 130.797
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute Change From Baseline in Vitamin D
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Vitamin D
|
-6.57 nmol/L
Standard Deviation 10.712
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute Change From Baseline in ratio of vitamin E to total lipids
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Ratio of Vitamin E to Total Lipids
|
-0.0 ratio
Standard Deviation 1.71
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute Change From Baseline in ALA
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Alpha Linoleic Acid (ALA)
|
0.06 mg/mL
Standard Deviation 0.045
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute Change From Baseline in EPA
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Eicosapentaenoic Acid (EPA)
|
-0.08 mg/mL
Standard Deviation 0.079
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute Change From Baseline in DHA
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Docosahexaenoic Acid (DHA)
|
-0.08 mg/mL
Standard Deviation 0.045
|
SECONDARY outcome
Timeframe: Baseline and 16 weeks of treatmentPopulation: All patients treated
Absolute Change From Baseline in LA
Outcome measures
| Measure |
Lomitapide Escalated
n=6 Participants
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.
|
|---|---|
|
Absolute Change From Baseline in Linoleic Acid (LA)
|
-2.9 mg/mL
Standard Deviation 1.51
|
Adverse Events
Lomitapide Escalated
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Lomitapide Escalated
n=6 participants at risk
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.
|
|---|---|
|
Reproductive system and breast disorders
Breast mass
|
16.7%
1/6 • Number of events 1 • First dose through 28 days post-treatment
|
Other adverse events
| Measure |
Lomitapide Escalated
n=6 participants at risk
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.
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
83.3%
5/6 • First dose through 28 days post-treatment
|
|
Gastrointestinal disorders
Nausea
|
33.3%
2/6 • First dose through 28 days post-treatment
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
2/6 • First dose through 28 days post-treatment
|
|
Gastrointestinal disorders
Dyspepsia
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
66.7%
4/6 • First dose through 28 days post-treatment
|
|
Investigations
Alinine aminotransferase increased
|
50.0%
3/6 • First dose through 28 days post-treatment
|
|
Gastrointestinal disorders
Abdominal pain upper
|
33.3%
2/6 • First dose through 28 days post-treatment
|
|
General disorders
Fatigue
|
33.3%
2/6 • First dose through 28 days post-treatment
|
|
Gastrointestinal disorders
Constipation
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Nervous system disorders
Headache
|
50.0%
3/6 • First dose through 28 days post-treatment
|
|
Investigations
Aspartate aminotransferase increased
|
50.0%
3/6 • First dose through 28 days post-treatment
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
33.3%
2/6 • First dose through 28 days post-treatment
|
|
Respiratory, thoracic and mediastinal disorders
Lung disorder
|
66.7%
4/6 • First dose through 28 days post-treatment
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
33.3%
2/6 • First dose through 28 days post-treatment
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
2/6 • First dose through 28 days post-treatment
|
|
Nervous system disorders
Dizziness
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Metabolism and nutrition disorders
Decreased appetite
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Vascular disorders
Hot flush
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Investigations
International normalized ratio increased
|
33.3%
2/6 • First dose through 28 days post-treatment
|
|
General disorders
Asthenia
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Injury, poisoning and procedural complications
Contusion
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Injury, poisoning and procedural complications
Scratch
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Skin and subcutaneous tissue disorders
Xanthoma
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Eye disorders
Eye disorders
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Infections and infestations
Rhinitis
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Investigations
Prothrombin time prolonged
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Reproductive system and breast disorders
Breast mass
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Reproductive system and breast disorders
Ovarian cyst
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Skin and subcutaneous tissue disorders
Acne
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Skin and subcutaneous tissue disorders
Rash
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
General disorders
Chest pain
|
16.7%
1/6 • First dose through 28 days post-treatment
|
|
Renal and urinary disorders
Haemoglobinuria
|
16.7%
1/6 • First dose through 28 days post-treatment
|
Additional Information
Chief Medical Officer
Aegerion Pharmaceutical
Phone: 617-500-7867
Results disclosure agreements
- Principal investigator is a sponsor employee Information is unavailable.
- Publication restrictions are in place
Restriction type: OTHER