Homozygous Familial Hypercholesterolaemia (HoFH)
Conditions
Brief summary
This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable lipid lowering therapy (LLT) (including lipoprotein apheresis (LA), when applicable) comprising of the following phases: * Screening Period (starting at Week 12, i.e. ≤12 weeks prior to Baseline for up to 6 weeks) * Stratified Enrolment and Start of Run in Period (starting at minimum at Week 6, i.e., 6 weeks prior to Baseline for a minimum of 6 weeks): * Efficacy Phase (starting at Baseline, i.e. Day \[D\] 0 for 24 weeks±3 days * Safety Phase (starting at Week 24±3 days for 80±1 weeks)
Detailed description
Lomitapide has been approved for use in adult patients with HoFH in the European Union (EU) and European Economic Area (EEA), United States of America (USA), Israel, Argentina, Canada, Colombia, and Japan. This study is designed to determine if lomitapide is effective and can be safely administered to paediatric patients with HoFH. If the efficacy and safety so far observed in adults is confirmed in paediatric patients, the potential exists to significantly lower low-density lipoprotein cholesterol (LDL-C) levels in paediatric patients with HoFH. Furthermore, the lower LDL-C levels may reduce atherosclerosis progression and would be expected to benefit these paediatric patients with HoFH. A single arm, non comparator design has been selected due to the rarity of the disease and because the evaluation of safety variables such as growth and sexual maturation requires longer term observation than would be feasible in the context of a placebo controlled study. To mitigate the disadvantages of a single arm design, the study includes a Run in Period of at least 6 weeks during which current lipid lowering therapy (LLT) (including lipoprotein apheresis (LA), when applicable) will be stabilised to establish baseline levels allowing each patient to serve as his/her own control. Patients will also remain on stable LLT (including LA, when applicable) during the Efficacy Phase of the study through Week 24±3 days.
Interventions
DRUGLomitapide
2mg,5mg, 10mg and 20mg capsules
Sponsors
Amryt Pharma
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* Male and female patients aged 5 to ≤17 years with HoFH as defined by any of the following criteria recommended by the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society (EAS) (Cuchel, Bruckert et al. 2014): 1. Genetic confirmation of 2 mutant alleles at the LDL receptor (LDLR), apo B, Proprotein convertase subtilisin/kexin type 9 (PCSK9), or LDL receptor adapter protein 1 (LDLRAP1) gene locus OR 2. An untreated LDL C \>500 mg/dL (13 mmol/L) or treated LDL C ≥300 mg/dL (8 mmol/L ) together with either Cutaneous or tendon xanthoma before age 10 years or Untreated LDL C levels consistent with heterozygous FH in both parents * Baseline LDL C on LLT (maximum concentration \[Cmax\] immediately prior to LA, if applicable) 1. \>160 mg/dL (4.1 mmol/L, no documented cardiovascular disease \[CVD\]) or 2. \>130 mg/dL (3.4 mmol/L, established CVD defined as aortic valve disease and/or coronary atherosclerosis) * Body weight ≥15 kg or body mass index (BMI) and height both \>10th percentile according to World Health Organization (WHO) Growth Charts for Boys and Girls 5 to 19 Years of Age * Patient and/or his/her legal representative has/have been informed, has/have read and understood the patient information/informed consent form, and has/have given written informed assent/consent * Patient and/or his/her legal representative must be able and willing to follow study procedures and instructions, particularly that 1. LLT (including LA, when applicable) must be stable for at least 6 weeks prior to Baseline (Run in Period) and remain stable through Week 24±3 days (end of Efficacy Phase) 2. The patient must be compliant with both the low fat diet supplying \<20% of energy (calories) from fat or \<30 g fat, whichever is the lesser amount starting at the beginning of the Run in Period and the dietary supplement regimen starting at Week 2 of the Run in Period, both continuing until completion of the study * Postmenarchal female adolescents must be willing to use an effective form of birth control with failure rates \<1% per year (e.g., implant, injectable, combined oral contraceptive, intrauterine contraceptive device, sexual abstinence, vasectomy or vasectomised partner) during participation in the study (and at least 4 weeks thereafter). Patients taking oestrogen based oral contraceptives should be advised about possible loss of effectiveness due to diarrhea and/or vomiting. Additional contraceptive measures should be used for 7 days after resolution of symptoms. * Patient must be in stable physical and mental health at screening
Exclusion criteria
* Other forms of primary hyperlipoproteinaemia and secondary causes of hypercholesterolaemia (e.g., nephrotic syndrome, hypothyroidism) * Contraindications for the use of lomitapide according to section 4.3 of the European Medicines Agency (EMA) Summary of Product Characteristics (SmPC), such as hypersensitivity to the active substance or to any of the excipients listed in Section 6.1 of the SmPC, known significant or chronic inflammatory bowel disease or malabsorption * Moderate (Child Pugh B) or severe hepatic impairment (Child Pugh C), active liver disease and/or abnormal liver function tests at screening (AST or ALT \>1.5 x upper limit of normal (ULN) and/or total bilirubin \>1.5 x ULN in the absence of Gilbert's syndrome or AP \>1.5 x ULN \[based on appropriate age and gender normal values\]) * Serum CK \>2 x ULN * Chronic renal insufficiency with glomerular filtration rate (GFR) \<70 mL/min/1.73 m2 calculated using the Schwartz formula * Uncontrolled hypertension (defined as mean systolic and/or diastolic blood pressure ≥95% of normal for age and sex) despite medical therapy * New York Heart Association (NYHA) Class III or IV congestive heart failure * Precocious/delayed puberty or endocrine disorder affecting growth (e.g., hypothyroidism, premature adrenarche) * History of drug abuse within the last 3 years or habitual alcohol consumption (defined as \>1 ounce \[28 g\] of liquor or 4 ounce glass \[113 g\] of wine, or the equivalent, ≥3 times per week) * Life expectancy predicted to be \<5 years * History of a non skin malignancy (with the exception of cervical cancer in situ) within 3 years prior to enrolment * Treatment with any Investigational Medicinal Product (IMP) within 6 months or 5 times the terminal half life of the corresponding IMP, whichever is longer, before the screening visit * Patient is a dependent of the sponsor, of the investigational team or his/her immediate family * Pregnant or nursing women
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline | Baseline through Week 24 | To evaluate the efficacy of lomitapide, as defined by the percent change in low density lipoprotein cholesterol (LDL C) at the maximum tolerated dose (MTD) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in apo B at the maximum tolerated dose (MTD) |
| Change in LA From Week 28 Through Week 104 | Week 28 through Week 104 | To evaluate the efficacy of lomitapide, as defined by the change in lipoprotein apheresis |
| Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24 | Baseline through Week 24 | To evaluate the efficacy of lomitapide, as defined by the number and percentage of patients achieving EAS recommended target (2013) of LDL-C |
| Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the number and percentage of patients achieving EAS recommended target (2013) of LDL-C |
| Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Baseline through Week 24 | To evaluate the efficacy of lomitapide, as defined by the percent change of the following lipid parameters at the maximum tolerated dose (MTD): * Non-high-density lipoprotein cholesterol (Non-HDL-C) * Total cholesterol (TC) * Very-low-density lipoprotein cholesterol (VLDL-C) * Apolipoprotein B (Apo B) * TG (Triglycerides) |
| Efficacy Endpoint: Percent Change From Baseline at Week 24 for Lp(a) | Baseline through Week 24 | To evaluate the efficacy of lomitapide, as defined by the percent change in Lp(a) at the maximum tolerated dose (MTD) |
| Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in LDL-C at the maximum tolerated dose (MTD) |
| Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in Non-HDL-C at the maximum tolerated dose (MTD) |
| Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in TC at the maximum tolerated dose (MTD) |
| Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in VLDL-C at the maximum tolerated dose (MTD) |
| Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in TG at the maximum tolerated dose (MTD) |
| Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in Lp(a) at the maximum tolerated dose (MTD) |
| Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in TC/HDL-C ratio at the maximum tolerated dose (MTD) |
| Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Baseline through Week 104 week | To evaluate the efficacy of lomitapide, as defined by the percent change in HDL-C at the maximum tolerated dose (MTD) |
| Change in LLT From Week 28 Through Week 104 | Week 28 through Week 104 | To evaluate the efficacy of lomitapide, as defined by the change in background lipid lowering therapy |
Other
| Measure | Time frame | Description |
|---|---|---|
| Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Baseline through Week 104 | To evaluate the safety of lomitapide, as defined by lipid accumulation in the liver as measured by ultrasound |
| Percent Change of Body Mass Index (BMI) | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change of BMI |
| Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Baseline through Week 104 | To evaluate the safety of lomitapide, as defined by lipid accumulation in the liver as measured by nuclear magnetic resonance (NMR) |
Countries
Germany, Israel, Italy, Saudi Arabia, Spain, Tunisia
Participant flow
Recruitment details
Male and female subjects aged 5 to ≤17 years with HoFH were enrolled in the trial at 12 study centres in various countries (3 in Germany, 1 in Israel, 2 in Italy, 2 in Saudi Arabia, 3 in Spain and 1 in Tunisia). The first patient first visit was 14 Dec 2020 and the last patient last visit was 06 Jun 2024.
Pre-assignment details
Subjects underwent assessments to determine eligibility at the Initial Screening Visit (up to 12 weeks prior to Day 0). A total of 46 subjects were enrolled in this study. Of these, 3 subjects were 'Run in' failures and did not complete the Run in Period.). Therefore, a total of 43 (93.5%) subjects entered the Efficacy Phase at Visit 4.
Participants by arm
| Arm | Count |
|---|---|
| Age 5-10 Years Lomitapide dosing commenced with 2mg at week 1 for 8 Weeks, then increased to 5mg Week 8±3 days, 10mg at Week 12±3 days to the maximum allowable dose of 20mg by Week 16±3 days or the MTD by Week 20±3 days based upon acceptable safety and tolerability criteria in addition to LDL C values.
Lomitapide: 2mg, 5mg, 10mg and 20mg capsules | 20 |
| Age 11-17 Years 11-15 years: Lomitapide dosing commenced with 2mg at week 1 for 4 Weeks, then increased to 5mg Week 4±3 days, 10mg at Week 8±3 days, 20mg at week 12±3 days to the maximum allowable dose of 40mg by Week 16±3 days or the MTD by Week 20±3 days based upon acceptable safety and tolerability criteria in addition to LDL C values.
16-17 years: Lomitapide dosing commenced with 5mg at week 1 for 4 Weeks, then increase to 10mg Week 4±3 days, 20 mg at Week 8±3 days, 40mgs at week 12±3 days to the maximum allowable dose of 60mg by Week 16±3 days or the MTD by Week 20±3 days based upon acceptable safety and tolerability criteria in addition to LDL C values.
Lomitapide: 2mg, 5mg, 10mg and 20mg capsules | 23 |
| Total | 43 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Open Label Efficacy Phase | Adverse Event | 0 | 2 |
| Open Label Safety Phase | AEs and none compliance | 0 | 1 |
| Open Label Safety Phase | Withdrawal by Subject | 0 | 1 |
| Stratified Enrolment & run-in | Death | 1 | 0 |
| Stratified Enrolment & run-in | Protocol Violation | 0 | 1 |
| Stratified Enrolment & run-in | Withdrawal by Subject | 0 | 1 |
Baseline characteristics
| Characteristic | Age 5-10 Years | Age 11-17 Years | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 20 Participants | 23 Participants | 43 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Continuous | 7.0 years STANDARD_DEVIATION 1.54 | 14.0 years STANDARD_DEVIATION 1.97 | 10.7 years STANDARD_DEVIATION 3.99 |
| Body mass index | 15.78 kg/m2 STANDARD_DEVIATION 3.338 | 21.52 kg/m2 STANDARD_DEVIATION 4.84 | 18.85 kg/m2 STANDARD_DEVIATION 5.071 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 0 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 19 Participants | 23 Participants | 42 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 19 Participants | 23 Participants | 42 Participants |
| Region of Enrollment Germany | 1 participants | 6 participants | 7 participants |
| Region of Enrollment Israel | 3 participants | 0 participants | 3 participants |
| Region of Enrollment Italy | 5 participants | 2 participants | 7 participants |
| Region of Enrollment Saudi Arabia | 3 participants | 10 participants | 13 participants |
| Region of Enrollment Spain | 3 participants | 4 participants | 7 participants |
| Region of Enrollment Tunisia | 5 participants | 1 participants | 6 participants |
| Sex: Female, Male Female | 10 Participants | 14 Participants | 24 Participants |
| Sex: Female, Male Male | 10 Participants | 9 Participants | 19 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 1 / 20 | 1 / 23 |
| other Total, other adverse events | 19 / 20 | 23 / 23 |
| serious Total, serious adverse events | 4 / 20 | 7 / 23 |
Outcome results
Primary
Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline
To evaluate the efficacy of lomitapide, as defined by the percent change in low density lipoprotein cholesterol (LDL C) at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 24
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| All Age Groups | Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline | -53.910 Percent change from Baseline | Standard Deviation 25.8287 |
| Age 5-10 Years | Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline | -56.529 Percent change from Baseline | Standard Deviation 26.5401 |
| Age 11-17 Years | Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline | -51.633 Percent change from Baseline | Standard Deviation 25.5658 |
Comparison: This was a single-arm study, no comparative group can be assigned; the comparison made for the primary efficacy endpoint is done only between baseline and Week 24 in paediatric subjects (5 to ≤17 years of age). Analysis was made using the one-sample t-test to test the null hypothesis that the percentage change from Baseline was equal to zero against the alternative hypothesis that the percentage change from Baseline was not equal to zero.p-value: <0.000195% CI: [-61.859, -45.9612]t-test, 1 sided
Secondary
Change in LA From Week 28 Through Week 104
To evaluate the efficacy of lomitapide, as defined by the change in lipoprotein apheresis
Time frame: Week 28 through Week 104
Population: Patients have been evaluated both, as individual age groups and all together.~Number of subjects treated with LLT (including LA) at Week 24:~* All age groups: 17 patients~* Age 5-10 Years: 6 patients~* Age 11-17 Years: 11 patients
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| All Age Groups | Change in LA From Week 28 Through Week 104 | Discontinuation due to low LDL-C | 1 Participants |
| All Age Groups | Change in LA From Week 28 Through Week 104 | Reduction due to other reason | 4 Participants |
| All Age Groups | Change in LA From Week 28 Through Week 104 | Reduction due to Adverse Event (AE) | 1 Participants |
| All Age Groups | Change in LA From Week 28 Through Week 104 | Reduction due to low LDL-C | 3 Participants |
| All Age Groups | Change in LA From Week 28 Through Week 104 | Discontinuation due to other reason | 1 Participants |
| Age 5-10 Years | Change in LA From Week 28 Through Week 104 | Reduction due to other reason | 3 Participants |
| Age 5-10 Years | Change in LA From Week 28 Through Week 104 | Reduction due to Adverse Event (AE) | 1 Participants |
| Age 5-10 Years | Change in LA From Week 28 Through Week 104 | Reduction due to low LDL-C | 1 Participants |
| Age 5-10 Years | Change in LA From Week 28 Through Week 104 | Discontinuation due to low LDL-C | 1 Participants |
| Age 5-10 Years | Change in LA From Week 28 Through Week 104 | Discontinuation due to other reason | 1 Participants |
| Age 11-17 Years | Change in LA From Week 28 Through Week 104 | Discontinuation due to other reason | 0 Participants |
| Age 11-17 Years | Change in LA From Week 28 Through Week 104 | Discontinuation due to low LDL-C | 0 Participants |
| Age 11-17 Years | Change in LA From Week 28 Through Week 104 | Reduction due to Adverse Event (AE) | 0 Participants |
| Age 11-17 Years | Change in LA From Week 28 Through Week 104 | Reduction due to other reason | 1 Participants |
| Age 11-17 Years | Change in LA From Week 28 Through Week 104 | Reduction due to low LDL-C | 2 Participants |
Secondary
Change in LLT From Week 28 Through Week 104
To evaluate the efficacy of lomitapide, as defined by the change in background lipid lowering therapy
Time frame: Week 28 through Week 104
Population: Patients have been evaluated both, as individual age groups and all together.~Number of subjects treated with LLT at Week 24:~* All age groups: 41 patients~* Age 5-10 Years: 20 patients~* Age 11-17 Years: 21 patients
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| All Age Groups | Change in LLT From Week 28 Through Week 104 | Increase due to high LDL-C | 3 Participants |
| All Age Groups | Change in LLT From Week 28 Through Week 104 | Discontinuation due to other reason | 2 Participants |
| All Age Groups | Change in LLT From Week 28 Through Week 104 | Reduction due to low LDL-C | 2 Participants |
| All Age Groups | Change in LLT From Week 28 Through Week 104 | Discontinuation due to lomitapide increase | 1 Participants |
| All Age Groups | Change in LLT From Week 28 Through Week 104 | Increase due to other reason | 1 Participants |
| Age 5-10 Years | Change in LLT From Week 28 Through Week 104 | Discontinuation due to other reason | 1 Participants |
| Age 5-10 Years | Change in LLT From Week 28 Through Week 104 | Reduction due to low LDL-C | 1 Participants |
| Age 5-10 Years | Change in LLT From Week 28 Through Week 104 | Discontinuation due to lomitapide increase | 1 Participants |
| Age 5-10 Years | Change in LLT From Week 28 Through Week 104 | Increase due to high LDL-C | 3 Participants |
| Age 5-10 Years | Change in LLT From Week 28 Through Week 104 | Increase due to other reason | 1 Participants |
| Age 11-17 Years | Change in LLT From Week 28 Through Week 104 | Increase due to other reason | 0 Participants |
| Age 11-17 Years | Change in LLT From Week 28 Through Week 104 | Increase due to high LDL-C | 0 Participants |
| Age 11-17 Years | Change in LLT From Week 28 Through Week 104 | Reduction due to low LDL-C | 1 Participants |
| Age 11-17 Years | Change in LLT From Week 28 Through Week 104 | Discontinuation due to other reason | 1 Participants |
| Age 11-17 Years | Change in LLT From Week 28 Through Week 104 | Discontinuation due to lomitapide increase | 0 Participants |
Secondary
Efficacy Endpoint: Percent Change From Baseline at Week 24 for Lp(a)
To evaluate the efficacy of lomitapide, as defined by the percent change in Lp(a) at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 24
Population: Patients have been evaluated both, as individual age groups and all together. Note: Lp(a) assessment was inadvertently removed from the central laboratory lipid panel during the laboratory set up phase. However, Lp(a) has also been assessed locally, but in various units (mg/dL, g/L, and nmol/L). Sicne Lp(a) concentrations should not be converted from 'nmol/L' to 'mg/dL', or vice versa, and more subjects had values reported in nmol/L, results are presented for the nmol/l only.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| All Age Groups | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Lp(a) | -26.757 Percent change from Baseline | Standard Deviation 32.5675 |
| Age 5-10 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Lp(a) | -17.248 Percent change from Baseline | Standard Deviation 36.4246 |
| Age 11-17 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Lp(a) | -36.996 Percent change from Baseline | Standard Deviation 25.3314 |
Secondary
Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters
To evaluate the efficacy of lomitapide, as defined by the percent change of the following lipid parameters at the maximum tolerated dose (MTD): * Non-high-density lipoprotein cholesterol (Non-HDL-C) * Total cholesterol (TC) * Very-low-density lipoprotein cholesterol (VLDL-C) * Apolipoprotein B (Apo B) * TG (Triglycerides)
Time frame: Baseline through Week 24
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Apo B | -53.1 Percent change from Baseline | Standard Deviation 24.76 |
| All Age Groups | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | VLDL-C | -49.678 Percent change from Baseline | Standard Deviation 31.6126 |
| All Age Groups | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Non-HDL-C | -54.185 Percent change from Baseline | Standard Deviation 25.0256 |
| All Age Groups | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | TC | -50.176 Percent change from Baseline | Standard Deviation 24.5397 |
| All Age Groups | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | TG | -49.28 Percent change from Baseline | Standard Deviation 31.729 |
| Age 5-10 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | VLDL-C | -53.030 Percent change from Baseline | Standard Deviation 28.0698 |
| Age 5-10 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Non-HDL-C | -56.655 Percent change from Baseline | Standard Deviation 25.9804 |
| Age 5-10 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | TC | -52.883 Percent change from Baseline | Standard Deviation 25.626 |
| Age 5-10 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Apo B | -56.2 Percent change from Baseline | Standard Deviation 24.45 |
| Age 5-10 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | TG | -52.292 Percent change from Baseline | Standard Deviation 27.8956 |
| Age 11-17 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | TG | -46.674 Percent change from Baseline | Standard Deviation 35.1359 |
| Age 11-17 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Apo B | -50.4 Percent change from Baseline | Standard Deviation 25.26 |
| Age 11-17 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Non-HDL-C | -52.038 Percent change from Baseline | Standard Deviation 24.5423 |
| Age 11-17 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | VLDL-C | -46.763 Percent change from Baseline | Standard Deviation 34.7606 |
| Age 11-17 Years | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | TC | -47.822 Percent change from Baseline | Standard Deviation 23.8756 |
Secondary
Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study
To evaluate the efficacy of lomitapide, as defined by the number and percentage of patients achieving EAS recommended target (2013) of LDL-C
Time frame: Baseline through Week 104
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| All Age Groups | Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study | <115 mg/dL - anytime up to week 104 | 20 Participants |
| All Age Groups | Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study | <135 mg/dL - anytime up to week 104 | 23 Participants |
| All Age Groups | Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study | <110 mg/dL - anytime up to week 104 | 19 Participants |
| Age 5-10 Years | Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study | <115 mg/dL - anytime up to week 104 | 7 Participants |
| Age 5-10 Years | Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study | <135 mg/dL - anytime up to week 104 | 10 Participants |
| Age 5-10 Years | Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study | <110 mg/dL - anytime up to week 104 | 6 Participants |
| Age 11-17 Years | Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study | <135 mg/dL - anytime up to week 104 | 13 Participants |
| Age 11-17 Years | Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study | <110 mg/dL - anytime up to week 104 | 13 Participants |
| Age 11-17 Years | Number and Percentage of Patients Achieving EAS Recommended Target (2013) of LDL-C at Any Time in the Study | <115 mg/dL - anytime up to week 104 | 13 Participants |
Secondary
Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24
To evaluate the efficacy of lomitapide, as defined by the number and percentage of patients achieving EAS recommended target (2013) of LDL-C
Time frame: Baseline through Week 24
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| All Age Groups | Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24 | <115 mg/dL - anytime up to Week 24 | 16 Participants |
| All Age Groups | Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24 | <135 mg/dL - anytime up to Week 24 | 18 Participants |
| All Age Groups | Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24 | <110 mg/dL - anytime up to Week 24 | 14 Participants |
| Age 5-10 Years | Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24 | <115 mg/dL - anytime up to Week 24 | 5 Participants |
| Age 5-10 Years | Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24 | <135 mg/dL - anytime up to Week 24 | 7 Participants |
| Age 5-10 Years | Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24 | <110 mg/dL - anytime up to Week 24 | 4 Participants |
| Age 11-17 Years | Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24 | <135 mg/dL - anytime up to Week 24 | 11 Participants |
| Age 11-17 Years | Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24 | <110 mg/dL - anytime up to Week 24 | 10 Participants |
| Age 11-17 Years | Number and Percentage of Patients Achieving European Atherosclerosis Society (EAS) Recommended Target (2013) of LDL-C at Any Timepoint Between Baseline and Week 24 | <115 mg/dL - anytime up to Week 24 | 11 Participants |
Secondary
Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B
To evaluate the efficacy of lomitapide, as defined by the percent change in apo B at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 104
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 4 | 0.2 Percent change from Baseline | Standard Deviation 18.99 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 20 | -51.6 Percent change from Baseline | Standard Deviation 24.21 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 56 | -47.1 Percent change from Baseline | Standard Deviation 33.06 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 68 | -44.8 Percent change from Baseline | Standard Deviation 33.99 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 8 | -6.5 Percent change from Baseline | Standard Deviation 19.25 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 80 | -47.1 Percent change from Baseline | Standard Deviation 30.58 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 24 | -54.0 Percent change from Baseline | Standard Deviation 25.02 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 92 | -41.8 Percent change from Baseline | Standard Deviation 35.12 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 104 | -44.6 Percent change from Baseline | Standard Deviation 30.22 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 28 | -56.2 Percent change from Baseline | Standard Deviation 26.17 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 32 | -52.0 Percent change from Baseline | Standard Deviation 28.64 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 12 | -15.5 Percent change from Baseline | Standard Deviation 21.91 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 36 | -43.9 Percent change from Baseline | Standard Deviation 30.91 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 40 | -48.9 Percent change from Baseline | Standard Deviation 29.11 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 16 | -36.1 Percent change from Baseline | Standard Deviation 16.7 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 44 | -46.1 Percent change from Baseline | Standard Deviation 28.75 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 48 | -48.6 Percent change from Baseline | Standard Deviation 29.03 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 52 | -47.7 Percent change from Baseline | Standard Deviation 30.16 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 44 | -53.5 Percent change from Baseline | Standard Deviation 25.61 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 52 | -55.4 Percent change from Baseline | Standard Deviation 26.77 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 28 | -59.2 Percent change from Baseline | Standard Deviation 25.77 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 4 | -0.5 Percent change from Baseline | Standard Deviation 16.45 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 56 | -57.7 Percent change from Baseline | Standard Deviation 28.41 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 20 | -52.1 Percent change from Baseline | Standard Deviation 24.26 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 40 | -50.6 Percent change from Baseline | Standard Deviation 27.92 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 68 | -53.9 Percent change from Baseline | Standard Deviation 24.92 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 32 | -55.7 Percent change from Baseline | Standard Deviation 27.78 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 16 | -32.6 Percent change from Baseline | Standard Deviation 14.52 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 80 | -53.7 Percent change from Baseline | Standard Deviation 25.96 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 48 | -56.9 Percent change from Baseline | Standard Deviation 24.99 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 36 | -47.4 Percent change from Baseline | Standard Deviation 31.45 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 92 | -45.5 Percent change from Baseline | Standard Deviation 28.97 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 24 | -55.4 Percent change from Baseline | Standard Deviation 25.73 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 12 | -10.3 Percent change from Baseline | Standard Deviation 14.37 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 104 | -51.1 Percent change from Baseline | Standard Deviation 22.42 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 8 | -2.4 Percent change from Baseline | Standard Deviation 16.63 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 104 | -38.1 Percent change from Baseline | Standard Deviation 35.85 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 4 | 0.9 Percent change from Baseline | Standard Deviation 21.41 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 8 | -10.3 Percent change from Baseline | Standard Deviation 21.15 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 16 | -39.7 Percent change from Baseline | Standard Deviation 18.5 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 20 | -51.2 Percent change from Baseline | Standard Deviation 24.76 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 24 | -52.7 Percent change from Baseline | Standard Deviation 24.96 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 28 | -53.2 Percent change from Baseline | Standard Deviation 26.91 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 32 | -47.8 Percent change from Baseline | Standard Deviation 29.8 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 36 | -40.3 Percent change from Baseline | Standard Deviation 30.74 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 40 | -47.3 Percent change from Baseline | Standard Deviation 30.81 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 44 | -38.7 Percent change from Baseline | Standard Deviation 30.42 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 48 | -40.7 Percent change from Baseline | Standard Deviation 30.97 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 52 | -39.1 Percent change from Baseline | Standard Deviation 32.14 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 56 | -35.9 Percent change from Baseline | Standard Deviation 34.63 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 68 | -35.7 Percent change from Baseline | Standard Deviation 39.71 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 80 | -40.0 Percent change from Baseline | Standard Deviation 34.09 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 92 | -37.7 Percent change from Baseline | Standard Deviation 41.39 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Week 12 | -20.8 Percent change from Baseline | Standard Deviation 26.84 |
Secondary
Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C
To evaluate the efficacy of lomitapide, as defined by the percent change in LDL-C at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 104
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 20 | -50.730 Percent change from Baseline | Standard Deviation 24.8321 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 8 | -6.798 Percent change from Baseline | Standard Deviation 19.1524 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 12 | -16.638 Percent change from Baseline | Standard Deviation 23.1978 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 16 | -36.458 Percent change from Baseline | Standard Deviation 17.9894 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 4 | -1.962 Percent change from Baseline | Standard Deviation 20.7168 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 24 | -54.602 Percent change from Baseline | Standard Deviation 26.4557 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 28 | -55.518 Percent change from Baseline | Standard Deviation 28.5714 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 32 | -52.126 Percent change from Baseline | Standard Deviation 28.7496 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 36 | -39.823 Percent change from Baseline | Standard Deviation 32.6612 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 40 | -49.427 Percent change from Baseline | Standard Deviation 29.583 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 44 | -44.312 Percent change from Baseline | Standard Deviation 30.3775 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 48 | -45.315 Percent change from Baseline | Standard Deviation 29.6526 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 52 | -44.583 Percent change from Baseline | Standard Deviation 32.8128 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 56 | -45.994 Percent change from Baseline | Standard Deviation 34.3631 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 68 | -40.831 Percent change from Baseline | Standard Deviation 37.3821 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 80 | -42.319 Percent change from Baseline | Standard Deviation 34.94 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 92 | -37.199 Percent change from Baseline | Standard Deviation 39.6625 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 104 | -40.159 Percent change from Baseline | Standard Deviation 30.5026 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 104 | -43.168 Percent change from Baseline | Standard Deviation 25.4634 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 4 | -5.936 Percent change from Baseline | Standard Deviation 10.8438 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 40 | -52.511 Percent change from Baseline | Standard Deviation 27.4375 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 52 | -52.956 Percent change from Baseline | Standard Deviation 25.8671 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 8 | -2.037 Percent change from Baseline | Standard Deviation 11.8697 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 68 | -52.220 Percent change from Baseline | Standard Deviation 24.9512 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 80 | -49.410 Percent change from Baseline | Standard Deviation 28.8677 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 12 | -11.742 Percent change from Baseline | Standard Deviation 16.1686 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 44 | -53.502 Percent change from Baseline | Standard Deviation 25.8673 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 92 | -41.059 Percent change from Baseline | Standard Deviation 33.6178 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 16 | -32.971 Percent change from Baseline | Standard Deviation 14.6467 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 36 | -45.072 Percent change from Baseline | Standard Deviation 32.4809 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 56 | -57.616 Percent change from Baseline | Standard Deviation 28.1958 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 20 | -49.906 Percent change from Baseline | Standard Deviation 25.1366 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 32 | -55.561 Percent change from Baseline | Standard Deviation 29.7938 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 48 | -53.965 Percent change from Baseline | Standard Deviation 23.2741 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 24 | -55.825 Percent change from Baseline | Standard Deviation 27.9112 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 28 | -57.929 Percent change from Baseline | Standard Deviation 29.0704 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 24 | -53.554 Percent change from Baseline | Standard Deviation 25.7903 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 28 | -53.108 Percent change from Baseline | Standard Deviation 28.6469 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 32 | -48.086 Percent change from Baseline | Standard Deviation 27.8122 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 68 | -31.151 Percent change from Baseline | Standard Deviation 43.6898 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 36 | -34.574 Percent change from Baseline | Standard Deviation 32.9076 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 104 | -37.150 Percent change from Baseline | Standard Deviation 35.3743 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 40 | -46.637 Percent change from Baseline | Standard Deviation 31.8088 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 44 | -35.122 Percent change from Baseline | Standard Deviation 32.3611 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 80 | -35.227 Percent change from Baseline | Standard Deviation 39.6221 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 48 | -37.963 Percent change from Baseline | Standard Deviation 32.9544 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 4 | 1.651 Percent change from Baseline | Standard Deviation 26.5109 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 8 | -11.058 Percent change from Baseline | Standard Deviation 23.3978 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 52 | -35.718 Percent change from Baseline | Standard Deviation 37.6118 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 12 | -21.289 Percent change from Baseline | Standard Deviation 27.9618 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 16 | -40.163 Percent change from Baseline | Standard Deviation 20.812 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 20 | -51.476 Percent change from Baseline | Standard Deviation 25.1505 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 56 | -33.761 Percent change from Baseline | Standard Deviation 36.6832 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Week 92 | -32.910 Percent change from Baseline | Standard Deviation 46.0836 |
Secondary
Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a))
To evaluate the efficacy of lomitapide, as defined by the percent change in Lp(a) at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 104
Population: Patients have been evaluated both, as individual age groups and all together. Note: Lp(a) assessment was inadvertently removed from the central laboratory lipid panel during the laboratory set up phase. However, Lp(a) has also been assessed locally, but in various units (mg/dL, g/L, and nmol/L). Sicne Lp(a) concentrations should not be converted from 'nmol/L' to 'mg/dL', or vice versa, and more subjects had values reported in nmol/L, results are presented for the nmol/l only.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 20 | -16.602 Percent change from Baseline | Standard Deviation 47.9856 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 8 | 1.508 Percent change from Baseline | Standard Deviation 46.1156 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 12 | 0.592 Percent change from Baseline | Standard Deviation 32.5241 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 16 | -14.648 Percent change from Baseline | Standard Deviation 26.314 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 4 | 9.660 Percent change from Baseline | Standard Deviation 63.5768 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 24 | -28.874 Percent change from Baseline | Standard Deviation 33.5277 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 28 | -32.628 Percent change from Baseline | Standard Deviation 32.4047 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 32 | -20.594 Percent change from Baseline | Standard Deviation 50.116 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 36 | -25.278 Percent change from Baseline | Standard Deviation 32.4201 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 40 | -28.257 Percent change from Baseline | Standard Deviation 34.6849 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 44 | -30.124 Percent change from Baseline | Standard Deviation 34.9943 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 48 | -17.905 Percent change from Baseline | Standard Deviation 38.3429 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 52 | -25.635 Percent change from Baseline | Standard Deviation 42.3832 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 56 | -22.366 Percent change from Baseline | Standard Deviation 34.5809 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 68 | -20.634 Percent change from Baseline | Standard Deviation 42.9257 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 80 | -22.659 Percent change from Baseline | Standard Deviation 42.2271 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 92 | -22.235 Percent change from Baseline | Standard Deviation 41.1508 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 104 | -29.064 Percent change from Baseline | Standard Deviation 34.593 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 104 | -24.776 Percent change from Baseline | Standard Deviation 37.6534 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 4 | 24.269 Percent change from Baseline | Standard Deviation 84.1451 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 40 | -20.400 Percent change from Baseline | Standard Deviation 39.7347 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 52 | -17.270 Percent change from Baseline | Standard Deviation 47.3261 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 8 | 13.870 Percent change from Baseline | Standard Deviation 54.053 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 68 | -9.136 Percent change from Baseline | Standard Deviation 47.0506 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 80 | -19.135 Percent change from Baseline | Standard Deviation 48.3708 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 12 | 13.509 Percent change from Baseline | Standard Deviation 36.2732 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 44 | -25.915 Percent change from Baseline | Standard Deviation 38.3512 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 92 | -16.468 Percent change from Baseline | Standard Deviation 44.0841 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 16 | -6.564 Percent change from Baseline | Standard Deviation 29.3156 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 36 | -21.547 Percent change from Baseline | Standard Deviation 36.2351 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 56 | -14.691 Percent change from Baseline | Standard Deviation 33.904 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 20 | -3.961 Percent change from Baseline | Standard Deviation 58.6273 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 32 | -27.534 Percent change from Baseline | Standard Deviation 46.6328 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 48 | -8.621 Percent change from Baseline | Standard Deviation 43.0927 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 24 | -15.021 Percent change from Baseline | Standard Deviation 38.1874 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 28 | -23.314 Percent change from Baseline | Standard Deviation 36.5744 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 24 | -42.728 Percent change from Baseline | Standard Deviation 21.912 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 28 | -46.081 Percent change from Baseline | Standard Deviation 20.1331 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 32 | -10.877 Percent change from Baseline | Standard Deviation 55.6561 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 68 | -41.989 Percent change from Baseline | Standard Deviation 24.2691 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 36 | -32.741 Percent change from Baseline | Standard Deviation 23.6913 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 104 | -40.212 Percent change from Baseline | Standard Deviation 24.848 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 40 | -40.480 Percent change from Baseline | Standard Deviation 21.6021 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 44 | -37.940 Percent change from Baseline | Standard Deviation 28.751 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 80 | -30.295 Percent change from Baseline | Standard Deviation 26.4884 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 48 | -35.145 Percent change from Baseline | Standard Deviation 20.2017 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 4 | -6.072 Percent change from Baseline | Standard Deviation 23.8773 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 8 | -13.327 Percent change from Baseline | Standard Deviation 30.7067 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 52 | -41.170 Percent change from Baseline | Standard Deviation 27.8522 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 12 | -15.847 Percent change from Baseline | Standard Deviation 17.2462 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 16 | -26.775 Percent change from Baseline | Standard Deviation 15.7748 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 20 | -31.540 Percent change from Baseline | Standard Deviation 26.7998 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 56 | -36.621 Percent change from Baseline | Standard Deviation 33.5132 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Week 92 | -37.229 Percent change from Baseline | Standard Deviation 31.2661 |
Secondary
Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C
To evaluate the efficacy of lomitapide, as defined by the percent change in Non-HDL-C at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 104
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 20 | -50.928 Percent change from Baseline | Standard Deviation 24.3958 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 8 | -6.745 Percent change from Baseline | Standard Deviation 18.6776 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 12 | -16.497 Percent change from Baseline | Standard Deviation 22.985 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 16 | -36.610 Percent change from Baseline | Standard Deviation 17.4339 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 4 | -1.765 Percent change from Baseline | Standard Deviation 21.1682 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 24 | -55.033 Percent change from Baseline | Standard Deviation 25.4297 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 28 | -55.984 Percent change from Baseline | Standard Deviation 27.5559 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 32 | -52.629 Percent change from Baseline | Standard Deviation 27.8748 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 36 | -40.574 Percent change from Baseline | Standard Deviation 31.4293 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 40 | -50.013 Percent change from Baseline | Standard Deviation 28.4878 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 44 | -44.702 Percent change from Baseline | Standard Deviation 29.8885 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 48 | -46.770 Percent change from Baseline | Standard Deviation 29.3974 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 52 | -45.222 Percent change from Baseline | Standard Deviation 31.9577 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 56 | -46.558 Percent change from Baseline | Standard Deviation 33.4734 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 68 | -41.576 Percent change from Baseline | Standard Deviation 36.0854 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 80 | -42.388 Percent change from Baseline | Standard Deviation 34.3439 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 92 | -37.755 Percent change from Baseline | Standard Deviation 38.4243 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 104 | -40.511 Percent change from Baseline | Standard Deviation 30.1102 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 104 | -43.555 Percent change from Baseline | Standard Deviation 24.5843 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 4 | -5.950 Percent change from Baseline | Standard Deviation 10.5881 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 40 | -52.837 Percent change from Baseline | Standard Deviation 26.6409 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 52 | -53.190 Percent change from Baseline | Standard Deviation 25.4004 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 8 | -1.938 Percent change from Baseline | Standard Deviation 12.1118 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 68 | -52.331 Percent change from Baseline | Standard Deviation 24.4942 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 80 | -49.605 Percent change from Baseline | Standard Deviation 28.2105 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 12 | -12.039 Percent change from Baseline | Standard Deviation 15.5376 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 44 | -53.685 Percent change from Baseline | Standard Deviation 25.3674 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 92 | -41.420 Percent change from Baseline | Standard Deviation 32.768 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 16 | -33.214 Percent change from Baseline | Standard Deviation 14.689 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 36 | -45.464 Percent change from Baseline | Standard Deviation 31.6491 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 56 | -57.724 Percent change from Baseline | Standard Deviation 27.7033 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 20 | -50.150 Percent change from Baseline | Standard Deviation 24.7267 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 32 | -55.742 Percent change from Baseline | Standard Deviation 29.1688 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 48 | -55.873 Percent change from Baseline | Standard Deviation 23.6964 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 24 | -56.006 Percent change from Baseline | Standard Deviation 27.3439 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 28 | -58.201 Percent change from Baseline | Standard Deviation 28.4121 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 24 | -54.200 Percent change from Baseline | Standard Deviation 24.322 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 28 | -53.768 Percent change from Baseline | Standard Deviation 27.262 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 32 | -49.171 Percent change from Baseline | Standard Deviation 26.7611 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 68 | -32.433 Percent change from Baseline | Standard Deviation 42.0777 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 36 | -35.685 Percent change from Baseline | Standard Deviation 31.3277 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 104 | -37.468 Percent change from Baseline | Standard Deviation 35.2966 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 40 | -47.458 Percent change from Baseline | Standard Deviation 30.485 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 44 | -35.720 Percent change from Baseline | Standard Deviation 31.9421 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 80 | -35.171 Percent change from Baseline | Standard Deviation 38.9712 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 48 | -38.577 Percent change from Baseline | Standard Deviation 32.1148 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 4 | 2.039 Percent change from Baseline | Standard Deviation 27.2319 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 8 | -11.045 Percent change from Baseline | Standard Deviation 22.5075 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 52 | -36.785 Percent change from Baseline | Standard Deviation 36.5745 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 12 | -20.733 Percent change from Baseline | Standard Deviation 28.0917 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 16 | -40.218 Percent change from Baseline | Standard Deviation 19.7812 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 20 | -51.632 Percent change from Baseline | Standard Deviation 24.6817 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 56 | -34.805 Percent change from Baseline | Standard Deviation 35.651 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Week 92 | -33.682 Percent change from Baseline | Standard Deviation 44.497 |
Secondary
Percent Change From Baseline at All Other Time Points Through Week 104 for TC
To evaluate the efficacy of lomitapide, as defined by the percent change in TC at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 104
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 20 | -48.177 Percent change from Baseline | Standard Deviation 23.2987 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 8 | -6.970 Percent change from Baseline | Standard Deviation 17.6904 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 12 | -15.961 Percent change from Baseline | Standard Deviation 21.4313 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 16 | -33.976 Percent change from Baseline | Standard Deviation 15.7159 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 4 | -2.057 Percent change from Baseline | Standard Deviation 19.3772 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 24 | -50.922 Percent change from Baseline | Standard Deviation 24.9738 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 28 | -51.998 Percent change from Baseline | Standard Deviation 26.9668 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 32 | -48.945 Percent change from Baseline | Standard Deviation 26.2375 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 36 | -40.643 Percent change from Baseline | Standard Deviation 30.5125 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 40 | -45.402 Percent change from Baseline | Standard Deviation 27.5358 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 44 | -41.443 Percent change from Baseline | Standard Deviation 28.3745 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 48 | -44.164 Percent change from Baseline | Standard Deviation 27.6987 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 52 | -42.764 Percent change from Baseline | Standard Deviation 30.6617 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 56 | -43.234 Percent change from Baseline | Standard Deviation 31.3599 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 68 | -39.672 Percent change from Baseline | Standard Deviation 33.2025 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 80 | -40.291 Percent change from Baseline | Standard Deviation 31.9119 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 92 | -34.946 Percent change from Baseline | Standard Deviation 35.8485 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 104 | -36.017 Percent change from Baseline | Standard Deviation 32.2354 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 104 | -42.430 Percent change from Baseline | Standard Deviation 24.0856 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 4 | -5.487 Percent change from Baseline | Standard Deviation 10.2352 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 40 | -46.997 Percent change from Baseline | Standard Deviation 27.2264 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 52 | -51.169 Percent change from Baseline | Standard Deviation 24.8422 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 8 | -3.092 Percent change from Baseline | Standard Deviation 13.2354 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 68 | -49.108 Percent change from Baseline | Standard Deviation 24.7124 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 80 | -48.031 Percent change from Baseline | Standard Deviation 26.5964 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 12 | -12.881 Percent change from Baseline | Standard Deviation 15.4304 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 44 | -50.186 Percent change from Baseline | Standard Deviation 24.6881 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 92 | -38.474 Percent change from Baseline | Standard Deviation 31.6123 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 16 | -30.986 Percent change from Baseline | Standard Deviation 14.0719 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 36 | -45.656 Percent change from Baseline | Standard Deviation 29.7175 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 56 | -54.109 Percent change from Baseline | Standard Deviation 26.058 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 20 | -48.524 Percent change from Baseline | Standard Deviation 23.7013 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 32 | -52.642 Percent change from Baseline | Standard Deviation 27.5342 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 48 | -53.422 Percent change from Baseline | Standard Deviation 22.4426 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 24 | -52.132 Percent change from Baseline | Standard Deviation 26.9234 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 28 | -54.806 Percent change from Baseline | Standard Deviation 27.4139 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 24 | -49.885 Percent change from Baseline | Standard Deviation 23.7998 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 28 | -49.190 Percent change from Baseline | Standard Deviation 26.9564 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 32 | -44.838 Percent change from Baseline | Standard Deviation 24.8402 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 68 | -30.236 Percent change from Baseline | Standard Deviation 38.271 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 36 | -35.365 Percent change from Baseline | Standard Deviation 31.2402 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 104 | -29.605 Percent change from Baseline | Standard Deviation 38.3282 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 40 | -43.882 Percent change from Baseline | Standard Deviation 28.4123 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 44 | -32.699 Percent change from Baseline | Standard Deviation 29.7008 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 80 | -32.143 Percent change from Baseline | Standard Deviation 35.589 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 48 | -35.369 Percent change from Baseline | Standard Deviation 29.8353 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 4 | 1.062 Percent change from Baseline | Standard Deviation 24.8373 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 8 | -10.643 Percent change from Baseline | Standard Deviation 20.7648 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 52 | -33.371 Percent change from Baseline | Standard Deviation 34.4186 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 12 | -19.041 Percent change from Baseline | Standard Deviation 26.1667 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 16 | -37.154 Percent change from Baseline | Standard Deviation 17.1693 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 20 | -47.847 Percent change from Baseline | Standard Deviation 23.4897 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 56 | -31.787 Percent change from Baseline | Standard Deviation 33.0105 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Week 92 | -31.025 Percent change from Baseline | Standard Deviation 40.6102 |
Secondary
Percent Change From Baseline at All Other Time Points Through Week 104 for TG
To evaluate the efficacy of lomitapide, as defined by the percent change in TG at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 104
Population: Patients from all age groups have been evaluated together.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 80 | -38.312 Percent change from Baseline | Standard Deviation 44.1074 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 28 | -55.639 Percent change from Baseline | Standard Deviation 24.6317 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 20 | -48.989 Percent change from Baseline | Standard Deviation 25.8872 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 12 | -7.258 Percent change from Baseline | Standard Deviation 49.699 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 32 | -48.697 Percent change from Baseline | Standard Deviation 54.0396 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 24 | -52.878 Percent change from Baseline | Standard Deviation 26.8475 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 92 | -41.549 Percent change from Baseline | Standard Deviation 30.2027 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 36 | -46.823 Percent change from Baseline | Standard Deviation 25.4762 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 68 | -48.471 Percent change from Baseline | Standard Deviation 30.8061 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 4 | 9.387 Percent change from Baseline | Standard Deviation 41.7233 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 40 | -51.612 Percent change from Baseline | Standard Deviation 25.9533 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 56 | -47.707 Percent change from Baseline | Standard Deviation 32.015 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 16 | -34.970 Percent change from Baseline | Standard Deviation 30.2259 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 44 | -47.065 Percent change from Baseline | Standard Deviation 31.7596 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 104 | -39.739 Percent change from Baseline | Standard Deviation 39.8597 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 52 | -49.415 Percent change from Baseline | Standard Deviation 28.4096 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 48 | -39.769 Percent change from Baseline | Standard Deviation 48.3307 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 8 | 2.649 Percent change from Baseline | Standard Deviation 35.3115 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 48 | -51.803 Percent change from Baseline | Standard Deviation 35.2601 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 52 | -55.142 Percent change from Baseline | Standard Deviation 20.0234 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 56 | -55.648 Percent change from Baseline | Standard Deviation 26.762 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 20 | -51.346 Percent change from Baseline | Standard Deviation 27.1808 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 68 | -51.253 Percent change from Baseline | Standard Deviation 21.5583 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 8 | 5.577 Percent change from Baseline | Standard Deviation 36.3036 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 80 | -47.765 Percent change from Baseline | Standard Deviation 27.6927 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 92 | -45.140 Percent change from Baseline | Standard Deviation 23.5731 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 24 | -53.866 Percent change from Baseline | Standard Deviation 27.5741 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 104 | -45.352 Percent change from Baseline | Standard Deviation 25.8574 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 4 | 1.699 Percent change from Baseline | Standard Deviation 32.1342 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 28 | -57.852 Percent change from Baseline | Standard Deviation 24.1398 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 32 | -54.381 Percent change from Baseline | Standard Deviation 28.481 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 12 | -6.133 Percent change from Baseline | Standard Deviation 56.7548 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 36 | -48.286 Percent change from Baseline | Standard Deviation 23.9243 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 40 | -55.105 Percent change from Baseline | Standard Deviation 20.8662 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 44 | -54.885 Percent change from Baseline | Standard Deviation 18.5911 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 16 | -37.161 Percent change from Baseline | Standard Deviation 23.9242 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 44 | -39.245 Percent change from Baseline | Standard Deviation 39.9507 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 4 | 16.376 Percent change from Baseline | Standard Deviation 48.5494 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 8 | 0.029 Percent change from Baseline | Standard Deviation 35.1807 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 12 | -8.327 Percent change from Baseline | Standard Deviation 43.4273 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 16 | -32.643 Percent change from Baseline | Standard Deviation 36.4318 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 20 | -46.856 Percent change from Baseline | Standard Deviation 25.136 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 24 | -52.031 Percent change from Baseline | Standard Deviation 26.8624 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 28 | -53.426 Percent change from Baseline | Standard Deviation 25.5749 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 32 | -42.380 Percent change from Baseline | Standard Deviation 73.2737 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 36 | -45.361 Percent change from Baseline | Standard Deviation 27.5563 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 40 | -48.451 Percent change from Baseline | Standard Deviation 29.9921 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 104 | -34.126 Percent change from Baseline | Standard Deviation 50.4118 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 48 | -28.939 Percent change from Baseline | Standard Deviation 56.3536 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 52 | -43.352 Percent change from Baseline | Standard Deviation 34.8302 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 56 | -39.349 Percent change from Baseline | Standard Deviation 35.5528 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 68 | -46.107 Percent change from Baseline | Standard Deviation 37.3356 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 80 | -28.860 Percent change from Baseline | Standard Deviation 55.1679 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Week 92 | -37.558 Percent change from Baseline | Standard Deviation 36.5005 |
Secondary
Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C
To evaluate the efficacy of lomitapide, as defined by the percent change in VLDL-C at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 104
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 4 | 9.770 Percent change from Baseline | Standard Deviation 42.2029 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 8 | 2.857 Percent change from Baseline | Standard Deviation 34.5887 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 12 | -7.405 Percent change from Baseline | Standard Deviation 48.8428 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 36 | -46.596 Percent change from Baseline | Standard Deviation 25.9994 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 16 | -34.706 Percent change from Baseline | Standard Deviation 31.659 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 20 | -49.068 Percent change from Baseline | Standard Deviation 25.6958 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 24 | -53.050 Percent change from Baseline | Standard Deviation 27.2118 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 28 | -55.394 Percent change from Baseline | Standard Deviation 25.0601 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 32 | -47.899 Percent change from Baseline | Standard Deviation 55.7396 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 40 | -51.167 Percent change from Baseline | Standard Deviation 26.7498 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 44 | -46.750 Percent change from Baseline | Standard Deviation 33.3163 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 48 | -38.148 Percent change from Baseline | Standard Deviation 50.61 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 52 | -49.469 Percent change from Baseline | Standard Deviation 30.0996 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 56 | -47.051 Percent change from Baseline | Standard Deviation 32.7429 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 68 | -47.832 Percent change from Baseline | Standard Deviation 30.6844 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 80 | -36.881 Percent change from Baseline | Standard Deviation 44.5589 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 92 | -41.368 Percent change from Baseline | Standard Deviation 31.7375 |
| All Age Groups | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 104 | -39.055 Percent change from Baseline | Standard Deviation 40.2422 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 24 | -54.447 Percent change from Baseline | Standard Deviation 27.8824 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 68 | -50.902 Percent change from Baseline | Standard Deviation 21.104 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 28 | -58.209 Percent change from Baseline | Standard Deviation 24.3762 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 32 | -54.787 Percent change from Baseline | Standard Deviation 28.1792 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 36 | -47.963 Percent change from Baseline | Standard Deviation 24.7584 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 40 | -55.266 Percent change from Baseline | Standard Deviation 21.3853 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 80 | -46.520 Percent change from Baseline | Standard Deviation 29.3256 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 44 | -54.803 Percent change from Baseline | Standard Deviation 19.0837 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 48 | -50.109 Percent change from Baseline | Standard Deviation 35.8714 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 104 | -44.612 Percent change from Baseline | Standard Deviation 26.8209 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 4 | 1.911 Percent change from Baseline | Standard Deviation 30.6624 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 52 | -55.576 Percent change from Baseline | Standard Deviation 20.344 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 8 | 5.190 Percent change from Baseline | Standard Deviation 35.0331 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 12 | -6.638 Percent change from Baseline | Standard Deviation 54.7493 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 92 | -44.652 Percent change from Baseline | Standard Deviation 23.2247 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 56 | -55.292 Percent change from Baseline | Standard Deviation 28.1394 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 16 | -36.773 Percent change from Baseline | Standard Deviation 23.3455 |
| Age 5-10 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 20 | -51.943 Percent change from Baseline | Standard Deviation 26.4251 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 12 | -8.133 Percent change from Baseline | Standard Deviation 43.936 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 20 | -46.467 Percent change from Baseline | Standard Deviation 25.3796 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 4 | 16.914 Percent change from Baseline | Standard Deviation 50.1439 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 28 | -52.579 Percent change from Baseline | Standard Deviation 26.0764 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 68 | -45.223 Percent change from Baseline | Standard Deviation 37.327 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 92 | -37.718 Percent change from Baseline | Standard Deviation 39.5304 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 32 | -39.796 Percent change from Baseline | Standard Deviation 76.932 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 16 | -32.509 Percent change from Baseline | Standard Deviation 39.3305 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 36 | -45.230 Percent change from Baseline | Standard Deviation 27.8348 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 104 | -33.498 Percent change from Baseline | Standard Deviation 50.548 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 8 | 0.771 Percent change from Baseline | Standard Deviation 35.0086 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 40 | -47.458 Percent change from Baseline | Standard Deviation 30.876 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 52 | -43.004 Percent change from Baseline | Standard Deviation 37.4046 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 56 | -38.375 Percent change from Baseline | Standard Deviation 35.6732 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 44 | -38.696 Percent change from Baseline | Standard Deviation 42.162 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 80 | -27.242 Percent change from Baseline | Standard Deviation 55.0014 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 24 | -51.853 Percent change from Baseline | Standard Deviation 27.2551 |
| Age 11-17 Years | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Week 48 | -27.981 Percent change from Baseline | Standard Deviation 59.4366 |
Secondary
Percent Change in HDL-C From Baseline at All Other Time Points to Week 104
To evaluate the efficacy of lomitapide, as defined by the percent change in HDL-C at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 104 week
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 20 | 6.186 Percent change from Baseline | Standard Deviation 33.3664 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 8 | 4.482 Percent change from Baseline | Standard Deviation 26.0635 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 12 | 4.912 Percent change from Baseline | Standard Deviation 29.1215 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 16 | 8.059 Percent change from Baseline | Standard Deviation 31.4604 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 4 | 1.428 Percent change from Baseline | Standard Deviation 19.628 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 24 | 11.412 Percent change from Baseline | Standard Deviation 33.0044 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 28 | 11.154 Percent change from Baseline | Standard Deviation 36.3133 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 32 | 15.137 Percent change from Baseline | Standard Deviation 33.9304 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 36 | 8.537 Percent change from Baseline | Standard Deviation 30.2218 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 40 | 10.043 Percent change from Baseline | Standard Deviation 35.0097 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 44 | 12.765 Percent change from Baseline | Standard Deviation 33.2136 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 48 | 13.602 Percent change from Baseline | Standard Deviation 37.5732 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 52 | 18.032 Percent change from Baseline | Standard Deviation 35.1079 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 56 | 11.528 Percent change from Baseline | Standard Deviation 29.175 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 68 | 9.557 Percent change from Baseline | Standard Deviation 30.6616 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 80 | 11.606 Percent change from Baseline | Standard Deviation 30.8242 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 92 | 17.320 Percent change from Baseline | Standard Deviation 33.0024 |
| All Age Groups | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 104 | 16.109 Percent change from Baseline | Standard Deviation 37.2987 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 104 | 26.224 Percent change from Baseline | Standard Deviation 38.978 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 4 | 7.659 Percent change from Baseline | Standard Deviation 16.376 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 40 | 25.145 Percent change from Baseline | Standard Deviation 32.0096 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 52 | 31.674 Percent change from Baseline | Standard Deviation 35.7852 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 8 | 12.855 Percent change from Baseline | Standard Deviation 29.0476 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 68 | 21.183 Percent change from Baseline | Standard Deviation 29.0036 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 80 | 17.203 Percent change from Baseline | Standard Deviation 31.1788 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 12 | 8.692 Percent change from Baseline | Standard Deviation 27.6073 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 44 | 22.486 Percent change from Baseline | Standard Deviation 34.6933 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 92 | 29.691 Percent change from Baseline | Standard Deviation 32.6733 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 16 | 19.392 Percent change from Baseline | Standard Deviation 24.6238 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 36 | 14.424 Percent change from Baseline | Standard Deviation 29.711 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 56 | 21.636 Percent change from Baseline | Standard Deviation 31.0975 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 20 | 17.873 Percent change from Baseline | Standard Deviation 30.1872 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 32 | 19.607 Percent change from Baseline | Standard Deviation 31.2092 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 48 | 25.097 Percent change from Baseline | Standard Deviation 42.3692 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 24 | 24.967 Percent change from Baseline | Standard Deviation 28.3089 |
| Age 5-10 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 28 | 19.883 Percent change from Baseline | Standard Deviation 27.9809 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 24 | -0.208 Percent change from Baseline | Standard Deviation 32.8835 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 28 | 2.425 Percent change from Baseline | Standard Deviation 42.033 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 32 | 10.171 Percent change from Baseline | Standard Deviation 36.9795 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 68 | -0.326 Percent change from Baseline | Standard Deviation 29.1404 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 36 | 2.651 Percent change from Baseline | Standard Deviation 30.4028 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 104 | 5.994 Percent change from Baseline | Standard Deviation 33.655 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 40 | -3.620 Percent change from Baseline | Standard Deviation 32.4844 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 44 | 3.044 Percent change from Baseline | Standard Deviation 29.3561 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 80 | 6.010 Percent change from Baseline | Standard Deviation 30.2457 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 48 | 3.256 Percent change from Baseline | Standard Deviation 30.0858 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 4 | -4.236 Percent change from Baseline | Standard Deviation 20.9511 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 8 | -3.010 Percent change from Baseline | Standard Deviation 21.1047 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 52 | 3.587 Percent change from Baseline | Standard Deviation 28.7665 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 12 | 1.322 Percent change from Baseline | Standard Deviation 30.7607 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 16 | -3.984 Percent change from Baseline | Standard Deviation 34.1243 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 20 | -4.387 Percent change from Baseline | Standard Deviation 33.2219 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 56 | 0.887 Percent change from Baseline | Standard Deviation 23.3045 |
| Age 11-17 Years | Percent Change in HDL-C From Baseline at All Other Time Points to Week 104 | Week 92 | 3.574 Percent change from Baseline | Standard Deviation 28.2358 |
Secondary
Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104
To evaluate the efficacy of lomitapide, as defined by the percent change in TC/HDL-C ratio at the maximum tolerated dose (MTD)
Time frame: Baseline through Week 104
Population: Patients have been evaluated both, as individual age groups and all together.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 20 | -45.689 Percent change from Baseline | Standard Deviation 27.0372 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 8 | -5.292 Percent change from Baseline | Standard Deviation 29.1737 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 12 | -14.501 Percent change from Baseline | Standard Deviation 27.7248 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 16 | -33.384 Percent change from Baseline | Standard Deviation 25.8765 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 4 | 0.108 Percent change from Baseline | Standard Deviation 27.5756 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 24 | -52.155 Percent change from Baseline | Standard Deviation 25.1906 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 28 | -50.986 Percent change from Baseline | Standard Deviation 31.0715 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 32 | -48.651 Percent change from Baseline | Standard Deviation 35.4572 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 36 | -35.860 Percent change from Baseline | Standard Deviation 35.4801 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 40 | -43.976 Percent change from Baseline | Standard Deviation 36.8573 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 44 | -40.395 Percent change from Baseline | Standard Deviation 38.1876 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 48 | -41.720 Percent change from Baseline | Standard Deviation 36.2674 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 52 | -43.496 Percent change from Baseline | Standard Deviation 38.2302 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 56 | -39.188 Percent change from Baseline | Standard Deviation 55.5616 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 68 | -36.874 Percent change from Baseline | Standard Deviation 41.902 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 80 | -39.189 Percent change from Baseline | Standard Deviation 37.9268 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 92 | -37.274 Percent change from Baseline | Standard Deviation 41.0451 |
| All Age Groups | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 104 | -39.358 Percent change from Baseline | Standard Deviation 34.3438 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 104 | -48.484 Percent change from Baseline | Standard Deviation 25.633 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 4 | -10.581 Percent change from Baseline | Standard Deviation 14.9489 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 40 | -56.120 Percent change from Baseline | Standard Deviation 26.2125 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 52 | -57.568 Percent change from Baseline | Standard Deviation 26.0987 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 8 | -7.941 Percent change from Baseline | Standard Deviation 22.3051 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 68 | -54.241 Percent change from Baseline | Standard Deviation 26.5277 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 80 | -49.165 Percent change from Baseline | Standard Deviation 33.2083 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 12 | -13.695 Percent change from Baseline | Standard Deviation 24.756 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 44 | -54.594 Percent change from Baseline | Standard Deviation 28.2165 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 92 | -47.527 Percent change from Baseline | Standard Deviation 33.5384 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 16 | -38.768 Percent change from Baseline | Standard Deviation 21.4831 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 36 | -45.043 Percent change from Baseline | Standard Deviation 34.3337 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 56 | -57.116 Percent change from Baseline | Standard Deviation 31.2556 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 20 | -51.512 Percent change from Baseline | Standard Deviation 24.7014 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 32 | -55.596 Percent change from Baseline | Standard Deviation 32.8921 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 48 | -54.921 Percent change from Baseline | Standard Deviation 27.6549 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 24 | -60.174 Percent change from Baseline | Standard Deviation 23.4381 |
| Age 5-10 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 28 | -59.358 Percent change from Baseline | Standard Deviation 27.4688 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 24 | -45.281 Percent change from Baseline | Standard Deviation 25.1244 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 28 | -42.614 Percent change from Baseline | Standard Deviation 32.8937 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 32 | -40.934 Percent change from Baseline | Standard Deviation 37.5141 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 68 | -22.111 Percent change from Baseline | Standard Deviation 47.2744 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 36 | -26.677 Percent change from Baseline | Standard Deviation 35.1331 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 104 | -30.233 Percent change from Baseline | Standard Deviation 39.9837 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 40 | -32.988 Percent change from Baseline | Standard Deviation 41.9967 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 44 | -26.197 Percent change from Baseline | Standard Deviation 42.104 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 80 | -29.213 Percent change from Baseline | Standard Deviation 40.5453 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 48 | -29.839 Percent change from Baseline | Standard Deviation 39.5405 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 4 | 9.825 Percent change from Baseline | Standard Deviation 32.7827 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 8 | -2.921 Percent change from Baseline | Standard Deviation 34.6428 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 52 | -28.596 Percent change from Baseline | Standard Deviation 43.8568 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 12 | -15.266 Percent change from Baseline | Standard Deviation 30.9105 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 16 | -27.664 Percent change from Baseline | Standard Deviation 29.4691 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 20 | -40.421 Percent change from Baseline | Standard Deviation 28.5476 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 56 | -20.317 Percent change from Baseline | Standard Deviation 68.9419 |
| Age 11-17 Years | Percent Change in TC/HDL-C Ratio From Baseline at All Other Time Points to Week 104 | Week 92 | -25.882 Percent change from Baseline | Standard Deviation 46.3515 |
Other Pre-specified
Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104
To evaluate the safety of lomitapide, as defined by lipid accumulation in the liver as measured by nuclear magnetic resonance (NMR)
Time frame: Baseline through Week 104
Population: All subjects were to undergo NMR imaging unless it was contraindicated or not feasible (e.g., due to the need for sedation or general anaesthesia in very young or anxious subjects). In this case, ultrasound scans were to be used at the discretion of the Investigator.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - ≤10% liver fat | 2 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - >10% and ≤20% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - >20% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - ≤10% liver fat | 1 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - >10% and ≤20% liver fat | 1 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - >20% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - No result | 2 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - ≤10% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - >10% and ≤20% liver fat | 1 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - >20% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - No result | 2 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - ≤10% liver fat | 3 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - >10% and ≤20% liver fat | 1 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - >20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - No result | 5 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - ≤10% liver fat | 16 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - ≤10% liver fat | 9 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - >10% and ≤20% liver fat | 1 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - >10% and ≤20% liver fat | 4 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - >20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - >10% and ≤20% liver fat | 4 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - ≤10% liver fat | 12 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - ≤10% liver fat | 11 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - >10% and ≤20% liver fat | 2 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - >20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - >20% liver fat | 1 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - >20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Nuclear Magnetic Resonance (NMR) at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - No result | 4 Participants |
Other Pre-specified
Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104
To evaluate the safety of lomitapide, as defined by lipid accumulation in the liver as measured by ultrasound
Time frame: Baseline through Week 104
Population: All subjects were to undergo NMR imaging unless it was contraindicated or not feasible (e.g., due to the need for sedation or general anaesthesia in very young or anxious subjects). In this case, ultrasound scans were to be used at the discretion of the Investigator.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - ≤10% liver fat | 17 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - >10% and ≤20% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - >20% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - ≤10% liver fat | 15 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - >10% and ≤20% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - >20% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - No result | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - ≤10% liver fat | 15 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - >10% and ≤20% liver fat | 1 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - >20% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - No result | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - ≤10% liver fat | 15 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - >10% and ≤20% liver fat | 0 Participants |
| All Age Groups | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - >20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - No result | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - ≤10% liver fat | 2 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - ≤10% liver fat | 2 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - >10% and ≤20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - >10% and ≤20% liver fat | 1 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Baseline - >20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - >10% and ≤20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - ≤10% liver fat | 2 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - ≤10% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - >10% and ≤20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 56 - >20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - >20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 104/EoT - >20% liver fat | 0 Participants |
| Age 5-10 Years | Lipid Accumulation in the Liver Over Time Measured by Ultrasound at Baseline and at Week 24, Week 56 and at Week 104 | Week 24 - No result | 0 Participants |
Other Pre-specified
Percent Change of Body Mass Index (BMI)
To evaluate the efficacy of lomitapide, as defined by the percent change of BMI
Time frame: Baseline through Week 104
Population: Patients from all age groups have been evaluated together.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 4 | -1.06 Percent change from Baseline | Standard Deviation 3.466 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 8 | -1.32 Percent change from Baseline | Standard Deviation 3.954 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 12 | -2.36 Percent change from Baseline | Standard Deviation 4.768 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 16 | -2.96 Percent change from Baseline | Standard Deviation 5.288 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 20 | -4.21 Percent change from Baseline | Standard Deviation 5.708 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 24 | -5.04 Percent change from Baseline | Standard Deviation 5.997 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 28 | -4.13 Percent change from Baseline | Standard Deviation 6.427 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 32 | -3.60 Percent change from Baseline | Standard Deviation 7.145 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 36 | -3.92 Percent change from Baseline | Standard Deviation 7.949 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 40 | -3.78 Percent change from Baseline | Standard Deviation 8.293 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 44 | -3.71 Percent change from Baseline | Standard Deviation 10.406 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 48 | -3.92 Percent change from Baseline | Standard Deviation 10.42 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 52 | -3.21 Percent change from Baseline | Standard Deviation 10.738 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 56 | -2.90 Percent change from Baseline | Standard Deviation 11.52 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 68 | -3.00 Percent change from Baseline | Standard Deviation 12.103 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 80 | -1.90 Percent change from Baseline | Standard Deviation 12.665 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 92 | -1.00 Percent change from Baseline | Standard Deviation 14.108 |
| All Age Groups | Percent Change of Body Mass Index (BMI) | Week 104 | -0.11 Percent change from Baseline | Standard Deviation 13.718 |