Trial Outcomes & Findings for Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) (NCT NCT04681170)

Last Updated: 2025-08-27

Results Overview

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)

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

46 participants

Primary outcome timeframe

Baseline through Week 24

Results posted on

2025-08-27

Participant Flow

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.

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.

Participant milestones

Participant milestones
Measure	Age 5-10 Years Lomitapide dosing commenced with 2mg at week 1 for 8 Weeks, then increased to 5mg Week 8±3 days, 10 mg 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	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 increased to 10mg Week 4±3 days, 20 mg at Week 8±3 days,40mg 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
Pre-run in STARTED	21	25
Pre-run in COMPLETED	21	25
Pre-run in NOT COMPLETED	0	0
Stratified Enrolment & run-in STARTED	21	25
Stratified Enrolment & run-in COMPLETED	20	23
Stratified Enrolment & run-in NOT COMPLETED	1	2
Open Label Efficacy Phase STARTED	20	23
Open Label Efficacy Phase COMPLETED	20	21
Open Label Efficacy Phase NOT COMPLETED	0	2
Open Label Safety Phase STARTED	20	21
Open Label Safety Phase COMPLETED	20	19
Open Label Safety Phase NOT COMPLETED	0	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Age 5-10 Years Lomitapide dosing commenced with 2mg at week 1 for 8 Weeks, then increased to 5mg Week 8±3 days, 10 mg 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	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 increased to 10mg Week 4±3 days, 20 mg at Week 8±3 days,40mg 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
Stratified Enrolment & run-in Death	1	0
Stratified Enrolment & run-in Withdrawal by Subject	0	1
Stratified Enrolment & run-in Protocol Violation	0	1
Open Label Efficacy Phase Adverse Event	0	2
Open Label Safety Phase Withdrawal by Subject	0	1
Open Label Safety Phase AEs and none compliance	0	1

Baseline Characteristics

Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Age 5-10 Years n=20 Participants 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	Age 11-17 Years n=23 Participants 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	Total n=43 Participants Total of all reporting groups
Age, Categorical <=18 years	20 Participants n=93 Participants	23 Participants n=4 Participants	43 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Continuous	7.0 years STANDARD_DEVIATION 1.54 • n=93 Participants	14.0 years STANDARD_DEVIATION 1.97 • n=4 Participants	10.7 years STANDARD_DEVIATION 3.99 • n=27 Participants
Sex: Female, Male Female	10 Participants n=93 Participants	14 Participants n=4 Participants	24 Participants n=27 Participants
Sex: Female, Male Male	10 Participants n=93 Participants	9 Participants n=4 Participants	19 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	19 Participants n=93 Participants	23 Participants n=4 Participants	42 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) White	19 Participants n=93 Participants	23 Participants n=4 Participants	42 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Region of Enrollment Saudi Arabia	3 participants n=93 Participants	10 participants n=4 Participants	13 participants n=27 Participants
Region of Enrollment Italy	5 participants n=93 Participants	2 participants n=4 Participants	7 participants n=27 Participants
Region of Enrollment Israel	3 participants n=93 Participants	0 participants n=4 Participants	3 participants n=27 Participants
Region of Enrollment Tunisia	5 participants n=93 Participants	1 participants n=4 Participants	6 participants n=27 Participants
Region of Enrollment Germany	1 participants n=93 Participants	6 participants n=4 Participants	7 participants n=27 Participants
Region of Enrollment Spain	3 participants n=93 Participants	4 participants n=4 Participants	7 participants n=27 Participants
Body mass index	15.78 kg/m2 STANDARD_DEVIATION 3.338 • n=93 Participants	21.52 kg/m2 STANDARD_DEVIATION 4.840 • n=4 Participants	18.85 kg/m2 STANDARD_DEVIATION 5.071 • n=27 Participants

PRIMARY outcome

Timeframe: Baseline through Week 24

Population: Patients have been evaluated both, as individual age groups and all together.

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)

Outcome measures

Outcome measures
Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 11-17 years.
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	-56.529 Percent change from Baseline Standard Deviation 26.5401	-51.633 Percent change from Baseline Standard Deviation 25.5658

SECONDARY outcome

Timeframe: Baseline through Week 24

Population: Patients have been evaluated both, as individual age groups and all together.

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)

Outcome measures

Outcome measures
Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 11-17 years.
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	-56.655 Percent change from Baseline Standard Deviation 25.9804	-52.038 Percent change from Baseline Standard Deviation 24.5423
Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters TC	-50.176 Percent change from Baseline Standard Deviation 24.5397	-52.883 Percent change from Baseline Standard Deviation 25.6260	-47.822 Percent change from Baseline Standard Deviation 23.8756
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	-53.030 Percent change from Baseline Standard Deviation 28.0698	-46.763 Percent change from Baseline Standard Deviation 34.7606
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	-56.2 Percent change from Baseline Standard Deviation 24.45	-50.4 Percent change from Baseline Standard Deviation 25.26
Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters TG	-49.28 Percent change from Baseline Standard Deviation 31.7290	-52.292 Percent change from Baseline Standard Deviation 27.8956	-46.674 Percent change from Baseline Standard Deviation 35.1359

SECONDARY outcome

Timeframe: 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.

To evaluate the efficacy of lomitapide, as defined by the percent change in Lp(a) at the maximum tolerated dose (MTD)

Outcome measures

Outcome measures
Measure	All Age Groups n=27 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=14 Participants Patients aged 5-10 years.	Age 11-17 Years n=13 Participants Patients aged 11-17 years.
Efficacy Endpoint: Percent Change From Baseline at Week 24 for Lp(a)	-26.757 Percent change from Baseline Standard Deviation 32.5675	-17.248 Percent change from Baseline Standard Deviation 36.4246	-36.996 Percent change from Baseline Standard Deviation 25.3314

SECONDARY outcome

Timeframe: Baseline through Week 104

Population: Patients have been evaluated both, as individual age groups and all together.

To evaluate the efficacy of lomitapide, as defined by the percent change in LDL-C at the maximum tolerated dose (MTD)

Outcome measures

SECONDARY outcome

Timeframe: Baseline through Week 104

Population: Patients have been evaluated both, as individual age groups and all together.

To evaluate the efficacy of lomitapide, as defined by the percent change in Non-HDL-C at the maximum tolerated dose (MTD)

Outcome measures

SECONDARY outcome

Timeframe: Baseline through Week 104

Population: Patients have been evaluated both, as individual age groups and all together.

To evaluate the efficacy of lomitapide, as defined by the percent change in TC at the maximum tolerated dose (MTD)

Outcome measures

SECONDARY outcome

Timeframe: Baseline through Week 104

Population: Patients have been evaluated both, as individual age groups and all together.

To evaluate the efficacy of lomitapide, as defined by the percent change in VLDL-C at the maximum tolerated dose (MTD)

Outcome measures

SECONDARY outcome

Timeframe: Baseline through Week 104

Population: Patients have been evaluated both, as individual age groups and all together.

To evaluate the efficacy of lomitapide, as defined by the percent change in apo B at the maximum tolerated dose (MTD)

Outcome measures

SECONDARY outcome

Timeframe: Baseline through Week 104

Population: Patients from all age groups have been evaluated together.

To evaluate the efficacy of lomitapide, as defined by the percent change in TG at the maximum tolerated dose (MTD)

Outcome measures

SECONDARY outcome

Timeframe: 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)

Outcome measures

SECONDARY outcome

Timeframe: Baseline through Week 104

Population: Patients have been evaluated both, as individual age groups and all together.

To evaluate the efficacy of lomitapide, as defined by the percent change in TC/HDL-C ratio at the maximum tolerated dose (MTD)

Outcome measures

SECONDARY outcome

Timeframe: Baseline through Week 104 week

Population: Patients have been evaluated both, as individual age groups and all together.

To evaluate the efficacy of lomitapide, as defined by the percent change in HDL-C at the maximum tolerated dose (MTD)

Outcome measures

SECONDARY outcome

Timeframe: 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

To evaluate the efficacy of lomitapide, as defined by the change in background lipid lowering therapy

Outcome measures

Outcome measures
Measure	All Age Groups n=41 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=21 Participants Patients aged 11-17 years.
Change in LLT From Week 28 Through Week 104 Discontinuation due to other reason	2 Participants	1 Participants	1 Participants
Change in LLT From Week 28 Through Week 104 Increase due to high LDL-C	3 Participants	3 Participants	0 Participants
Change in LLT From Week 28 Through Week 104 Reduction due to low LDL-C	2 Participants	1 Participants	1 Participants
Change in LLT From Week 28 Through Week 104 Discontinuation due to lomitapide increase	1 Participants	1 Participants	0 Participants
Change in LLT From Week 28 Through Week 104 Increase due to other reason	1 Participants	1 Participants	0 Participants

SECONDARY outcome

Timeframe: 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

To evaluate the efficacy of lomitapide, as defined by the change in lipoprotein apheresis

Outcome measures

Outcome measures
Measure	All Age Groups n=17 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=6 Participants Patients aged 5-10 years.	Age 11-17 Years n=11 Participants Patients aged 11-17 years.
Change in LA From Week 28 Through Week 104 Reduction due to low LDL-C	3 Participants	1 Participants	2 Participants
Change in LA From Week 28 Through Week 104 Reduction due to Adverse Event (AE)	1 Participants	1 Participants	0 Participants
Change in LA From Week 28 Through Week 104 Reduction due to other reason	4 Participants	3 Participants	1 Participants
Change in LA From Week 28 Through Week 104 Discontinuation due to low LDL-C	1 Participants	1 Participants	0 Participants
Change in LA From Week 28 Through Week 104 Discontinuation due to other reason	1 Participants	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline through Week 24

Population: Patients have been evaluated both, as individual age groups and all together.

To evaluate the efficacy of lomitapide, as defined by the number and percentage of patients achieving EAS recommended target (2013) of LDL-C

Outcome measures

Outcome measures
Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 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	18 Participants	7 Participants	11 Participants
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	5 Participants	11 Participants
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	4 Participants	10 Participants

SECONDARY outcome

Timeframe: Baseline through Week 104

Population: Patients have been evaluated both, as individual age groups and all together.

To evaluate the efficacy of lomitapide, as defined by the number and percentage of patients achieving EAS recommended target (2013) of LDL-C

Outcome measures

Outcome measures
Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 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	23 Participants	10 Participants	13 Participants
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	7 Participants	13 Participants
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	6 Participants	13 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline through Week 104

Population: Patients from all age groups have been evaluated together.

To evaluate the efficacy of lomitapide, as defined by the percent change of BMI

Outcome measures

Outcome measures
Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years Patients aged 5-10 years.	Age 11-17 Years Patients aged 11-17 years.
Percent Change of Body Mass Index (BMI) Week 104	-0.11 Percent change from Baseline Standard Deviation 13.718	—	—
Percent Change of Body Mass Index (BMI) Week 4	-1.06 Percent change from Baseline Standard Deviation 3.466	—	—
Percent Change of Body Mass Index (BMI) Week 8	-1.32 Percent change from Baseline Standard Deviation 3.954	—	—
Percent Change of Body Mass Index (BMI) Week 12	-2.36 Percent change from Baseline Standard Deviation 4.768	—	—
Percent Change of Body Mass Index (BMI) Week 16	-2.96 Percent change from Baseline Standard Deviation 5.288	—	—
Percent Change of Body Mass Index (BMI) Week 20	-4.21 Percent change from Baseline Standard Deviation 5.708	—	—
Percent Change of Body Mass Index (BMI) Week 24	-5.04 Percent change from Baseline Standard Deviation 5.997	—	—
Percent Change of Body Mass Index (BMI) Week 28	-4.13 Percent change from Baseline Standard Deviation 6.427	—	—
Percent Change of Body Mass Index (BMI) Week 32	-3.60 Percent change from Baseline Standard Deviation 7.145	—	—
Percent Change of Body Mass Index (BMI) Week 36	-3.92 Percent change from Baseline Standard Deviation 7.949	—	—
Percent Change of Body Mass Index (BMI) Week 40	-3.78 Percent change from Baseline Standard Deviation 8.293	—	—
Percent Change of Body Mass Index (BMI) Week 44	-3.71 Percent change from Baseline Standard Deviation 10.406	—	—
Percent Change of Body Mass Index (BMI) Week 48	-3.92 Percent change from Baseline Standard Deviation 10.420	—	—
Percent Change of Body Mass Index (BMI) Week 52	-3.21 Percent change from Baseline Standard Deviation 10.738	—	—
Percent Change of Body Mass Index (BMI) Week 56	-2.90 Percent change from Baseline Standard Deviation 11.520	—	—
Percent Change of Body Mass Index (BMI) Week 68	-3.00 Percent change from Baseline Standard Deviation 12.103	—	—
Percent Change of Body Mass Index (BMI) Week 80	-1.90 Percent change from Baseline Standard Deviation 12.665	—	—
Percent Change of Body Mass Index (BMI) Week 92	-1.00 Percent change from Baseline Standard Deviation 14.108	—	—

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

To evaluate the safety of lomitapide, as defined by lipid accumulation in the liver as measured by nuclear magnetic resonance (NMR)

Outcome measures

Outcome measures
Measure	All Age Groups n=4 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=19 Participants Patients aged 5-10 years.	Age 11-17 Years Patients aged 11-17 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	2 Participants	16 Participants	—
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	1 Participants	—
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	0 Participants	—
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	12 Participants	—
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	2 Participants	—
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	1 Participants	—
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	4 Participants	—
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	9 Participants	—
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	4 Participants	—
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	0 Participants	—
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	5 Participants	—
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	11 Participants	—
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	4 Participants	—
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	0 Participants	—

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline through Week 104

To evaluate the safety of lomitapide, as defined by lipid accumulation in the liver as measured by ultrasound

Outcome measures

Outcome measures
Measure	All Age Groups n=17 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=2 Participants Patients aged 5-10 years.	Age 11-17 Years Patients aged 11-17 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	17 Participants	2 Participants	—
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	0 Participants	—
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	0 Participants	—
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	2 Participants	—
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	0 Participants	—
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	0 Participants	—
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	0 Participants	—
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	2 Participants	—
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	0 Participants	—
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	0 Participants	—
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	0 Participants	—
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	0 Participants	—
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	1 Participants	—
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	0 Participants	—

Adverse Events

Age 5-10 Years

Serious events: 4 serious events

Other events: 19 other events

Deaths: 1 deaths

Age 11-17 Years

Serious events: 7 serious events

Other events: 23 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Additional Information

Head of Clinical Operations

Amryt Pharmaceuticals DAC

Phone: +35315180200

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The investigators may use the study data only after prior written consent of Amryt Pharmaceuticals DAC. Amryt will review the proposed publication/disclosure prior to submission for publication, presenting, using for instructional purposes or otherwise disclosing the study results. If Amryt supposes the publication/disclosure to risk its ability to patent any invention related to the study, the publication/disclosure will be modified or delayed to allow Amryt to file a patent application.
Publication restrictions are in place

Restriction type: OTHER

Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 11-17 years.
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 34.9400	-49.410 Percent change from Baseline Standard Deviation 28.8677	-35.227 Percent change from Baseline Standard Deviation 39.6221
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	-5.936 Percent change from Baseline Standard Deviation 10.8438	1.651 Percent change from Baseline Standard Deviation 26.5109
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	-2.037 Percent change from Baseline Standard Deviation 11.8697	-11.058 Percent change from Baseline Standard Deviation 23.3978
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	-11.742 Percent change from Baseline Standard Deviation 16.1686	-21.289 Percent change from Baseline Standard Deviation 27.9618
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	-32.971 Percent change from Baseline Standard Deviation 14.6467	-40.163 Percent change from Baseline Standard Deviation 20.8120
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	-49.906 Percent change from Baseline Standard Deviation 25.1366	-51.476 Percent change from Baseline Standard Deviation 25.1505
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	-55.825 Percent change from Baseline Standard Deviation 27.9112	-53.554 Percent change from Baseline Standard Deviation 25.7903
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	-57.929 Percent change from Baseline Standard Deviation 29.0704	-53.108 Percent change from Baseline Standard Deviation 28.6469
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	-55.561 Percent change from Baseline Standard Deviation 29.7938	-48.086 Percent change from Baseline Standard Deviation 27.8122
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	-45.072 Percent change from Baseline Standard Deviation 32.4809	-34.574 Percent change from Baseline Standard Deviation 32.9076
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 29.5830	-52.511 Percent change from Baseline Standard Deviation 27.4375	-46.637 Percent change from Baseline Standard Deviation 31.8088
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	-53.502 Percent change from Baseline Standard Deviation 25.8673	-35.122 Percent change from Baseline Standard Deviation 32.3611
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	-53.965 Percent change from Baseline Standard Deviation 23.2741	-37.963 Percent change from Baseline Standard Deviation 32.9544
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	-52.956 Percent change from Baseline Standard Deviation 25.8671	-35.718 Percent change from Baseline Standard Deviation 37.6118
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	-57.616 Percent change from Baseline Standard Deviation 28.1958	-33.761 Percent change from Baseline Standard Deviation 36.6832
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	-52.220 Percent change from Baseline Standard Deviation 24.9512	-31.151 Percent change from Baseline Standard Deviation 43.6898
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	-41.059 Percent change from Baseline Standard Deviation 33.6178	-32.910 Percent change from Baseline Standard Deviation 46.0836
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	-43.168 Percent change from Baseline Standard Deviation 25.4634	-37.150 Percent change from Baseline Standard Deviation 35.3743

Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 11-17 years.
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	-50.150 Percent change from Baseline Standard Deviation 24.7267	-51.632 Percent change from Baseline Standard Deviation 24.6817
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	-5.950 Percent change from Baseline Standard Deviation 10.5881	2.039 Percent change from Baseline Standard Deviation 27.2319
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	-1.938 Percent change from Baseline Standard Deviation 12.1118	-11.045 Percent change from Baseline Standard Deviation 22.5075
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 22.9850	-12.039 Percent change from Baseline Standard Deviation 15.5376	-20.733 Percent change from Baseline Standard Deviation 28.0917
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	-33.214 Percent change from Baseline Standard Deviation 14.6890	-40.218 Percent change from Baseline Standard Deviation 19.7812
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	-56.006 Percent change from Baseline Standard Deviation 27.3439	-54.200 Percent change from Baseline Standard Deviation 24.3220
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	-58.201 Percent change from Baseline Standard Deviation 28.4121	-53.768 Percent change from Baseline Standard Deviation 27.2620
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	-55.742 Percent change from Baseline Standard Deviation 29.1688	-49.171 Percent change from Baseline Standard Deviation 26.7611
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	-45.464 Percent change from Baseline Standard Deviation 31.6491	-35.685 Percent change from Baseline Standard Deviation 31.3277
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	-52.837 Percent change from Baseline Standard Deviation 26.6409	-47.458 Percent change from Baseline Standard Deviation 30.4850
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	-53.685 Percent change from Baseline Standard Deviation 25.3674	-35.720 Percent change from Baseline Standard Deviation 31.9421
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	-55.873 Percent change from Baseline Standard Deviation 23.6964	-38.577 Percent change from Baseline Standard Deviation 32.1148
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	-53.190 Percent change from Baseline Standard Deviation 25.4004	-36.785 Percent change from Baseline Standard Deviation 36.5745
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	-57.724 Percent change from Baseline Standard Deviation 27.7033	-34.805 Percent change from Baseline Standard Deviation 35.6510
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	-52.331 Percent change from Baseline Standard Deviation 24.4942	-32.433 Percent change from Baseline Standard Deviation 42.0777
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	-49.605 Percent change from Baseline Standard Deviation 28.2105	-35.171 Percent change from Baseline Standard Deviation 38.9712
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	-41.420 Percent change from Baseline Standard Deviation 32.7680	-33.682 Percent change from Baseline Standard Deviation 44.4970
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	-43.555 Percent change from Baseline Standard Deviation 24.5843	-37.468 Percent change from Baseline Standard Deviation 35.2966

Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 11-17 years.
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	-5.487 Percent change from Baseline Standard Deviation 10.2352	1.062 Percent change from Baseline Standard Deviation 24.8373
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	-3.092 Percent change from Baseline Standard Deviation 13.2354	-10.643 Percent change from Baseline Standard Deviation 20.7648
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	-12.881 Percent change from Baseline Standard Deviation 15.4304	-19.041 Percent change from Baseline Standard Deviation 26.1667
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	-30.986 Percent change from Baseline Standard Deviation 14.0719	-37.154 Percent change from Baseline Standard Deviation 17.1693
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	-48.524 Percent change from Baseline Standard Deviation 23.7013	-47.847 Percent change from Baseline Standard Deviation 23.4897
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	-52.132 Percent change from Baseline Standard Deviation 26.9234	-49.885 Percent change from Baseline Standard Deviation 23.7998
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	-54.806 Percent change from Baseline Standard Deviation 27.4139	-49.190 Percent change from Baseline Standard Deviation 26.9564
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	-52.642 Percent change from Baseline Standard Deviation 27.5342	-44.838 Percent change from Baseline Standard Deviation 24.8402
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	-45.656 Percent change from Baseline Standard Deviation 29.7175	-35.365 Percent change from Baseline Standard Deviation 31.2402
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	-46.997 Percent change from Baseline Standard Deviation 27.2264	-43.882 Percent change from Baseline Standard Deviation 28.4123
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	-50.186 Percent change from Baseline Standard Deviation 24.6881	-32.699 Percent change from Baseline Standard Deviation 29.7008
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	-53.422 Percent change from Baseline Standard Deviation 22.4426	-35.369 Percent change from Baseline Standard Deviation 29.8353
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	-51.169 Percent change from Baseline Standard Deviation 24.8422	-33.371 Percent change from Baseline Standard Deviation 34.4186
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	-54.109 Percent change from Baseline Standard Deviation 26.0580	-31.787 Percent change from Baseline Standard Deviation 33.0105
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	-49.108 Percent change from Baseline Standard Deviation 24.7124	-30.236 Percent change from Baseline Standard Deviation 38.2710
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	-48.031 Percent change from Baseline Standard Deviation 26.5964	-32.143 Percent change from Baseline Standard Deviation 35.5890
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	-38.474 Percent change from Baseline Standard Deviation 31.6123	-31.025 Percent change from Baseline Standard Deviation 40.6102
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	-42.430 Percent change from Baseline Standard Deviation 24.0856	-29.605 Percent change from Baseline Standard Deviation 38.3282

Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 11-17 years.
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	1.911 Percent change from Baseline Standard Deviation 30.6624	16.914 Percent change from Baseline Standard Deviation 50.1439
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	5.190 Percent change from Baseline Standard Deviation 35.0331	0.771 Percent change from Baseline Standard Deviation 35.0086
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	-6.638 Percent change from Baseline Standard Deviation 54.7493	-8.133 Percent change from Baseline Standard Deviation 43.9360
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 31.6590	-36.773 Percent change from Baseline Standard Deviation 23.3455	-32.509 Percent change from Baseline Standard Deviation 39.3305
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	-51.943 Percent change from Baseline Standard Deviation 26.4251	-46.467 Percent change from Baseline Standard Deviation 25.3796
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	-54.447 Percent change from Baseline Standard Deviation 27.8824	-51.853 Percent change from Baseline Standard Deviation 27.2551
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	-58.209 Percent change from Baseline Standard Deviation 24.3762	-52.579 Percent change from Baseline Standard Deviation 26.0764
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	-54.787 Percent change from Baseline Standard Deviation 28.1792	-39.796 Percent change from Baseline Standard Deviation 76.9320
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	-47.963 Percent change from Baseline Standard Deviation 24.7584	-45.230 Percent change from Baseline Standard Deviation 27.8348
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	-55.266 Percent change from Baseline Standard Deviation 21.3853	-47.458 Percent change from Baseline Standard Deviation 30.8760
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	-54.803 Percent change from Baseline Standard Deviation 19.0837	-38.696 Percent change from Baseline Standard Deviation 42.1620
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 50.6100	-50.109 Percent change from Baseline Standard Deviation 35.8714	-27.981 Percent change from Baseline Standard Deviation 59.4366
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	-55.576 Percent change from Baseline Standard Deviation 20.3440	-43.004 Percent change from Baseline Standard Deviation 37.4046
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	-55.292 Percent change from Baseline Standard Deviation 28.1394	-38.375 Percent change from Baseline Standard Deviation 35.6732
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	-50.902 Percent change from Baseline Standard Deviation 21.1040	-45.223 Percent change from Baseline Standard Deviation 37.3270
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	-46.520 Percent change from Baseline Standard Deviation 29.3256	-27.242 Percent change from Baseline Standard Deviation 55.0014
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	-44.652 Percent change from Baseline Standard Deviation 23.2247	-37.718 Percent change from Baseline Standard Deviation 39.5304
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	-44.612 Percent change from Baseline Standard Deviation 26.8209	-33.498 Percent change from Baseline Standard Deviation 50.5480

Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 11-17 years.
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	-0.5 Percent change from Baseline Standard Deviation 16.45	0.9 Percent change from Baseline Standard Deviation 21.41
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	-2.4 Percent change from Baseline Standard Deviation 16.63	-10.3 Percent change from Baseline Standard Deviation 21.15
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	-55.7 Percent change from Baseline Standard Deviation 27.78	-47.8 Percent change from Baseline Standard Deviation 29.80
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	-47.4 Percent change from Baseline Standard Deviation 31.45	-40.3 Percent change from Baseline Standard Deviation 30.74
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	-50.6 Percent change from Baseline Standard Deviation 27.92	-47.3 Percent change from Baseline Standard Deviation 30.81
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	-53.5 Percent change from Baseline Standard Deviation 25.61	-38.7 Percent change from Baseline Standard Deviation 30.42
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	-56.9 Percent change from Baseline Standard Deviation 24.99	-40.7 Percent change from Baseline Standard Deviation 30.97
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	-55.4 Percent change from Baseline Standard Deviation 26.77	-39.1 Percent change from Baseline Standard Deviation 32.14
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	-57.7 Percent change from Baseline Standard Deviation 28.41	-35.9 Percent change from Baseline Standard Deviation 34.63
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	-10.3 Percent change from Baseline Standard Deviation 14.37	-20.8 Percent change from Baseline Standard Deviation 26.84
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 16.70	-32.6 Percent change from Baseline Standard Deviation 14.52	-39.7 Percent change from Baseline Standard Deviation 18.50
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	-52.1 Percent change from Baseline Standard Deviation 24.26	-51.2 Percent change from Baseline Standard Deviation 24.76
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	-55.4 Percent change from Baseline Standard Deviation 25.73	-52.7 Percent change from Baseline Standard Deviation 24.96
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	-59.2 Percent change from Baseline Standard Deviation 25.77	-53.2 Percent change from Baseline Standard Deviation 26.91
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	-53.9 Percent change from Baseline Standard Deviation 24.92	-35.7 Percent change from Baseline Standard Deviation 39.71
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	-53.7 Percent change from Baseline Standard Deviation 25.96	-40.0 Percent change from Baseline Standard Deviation 34.09
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	-45.5 Percent change from Baseline Standard Deviation 28.97	-37.7 Percent change from Baseline Standard Deviation 41.39
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	-51.1 Percent change from Baseline Standard Deviation 22.42	-38.1 Percent change from Baseline Standard Deviation 35.85

Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 11-17 years.
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	-48.286 Percent change from Baseline Standard Deviation 23.9243	-45.361 Percent change from Baseline Standard Deviation 27.5563
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	-55.105 Percent change from Baseline Standard Deviation 20.8662	-48.451 Percent change from Baseline Standard Deviation 29.9921
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	-51.803 Percent change from Baseline Standard Deviation 35.2601	-28.939 Percent change from Baseline Standard Deviation 56.3536
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	-55.142 Percent change from Baseline Standard Deviation 20.0234	-43.352 Percent change from Baseline Standard Deviation 34.8302
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	-45.140 Percent change from Baseline Standard Deviation 23.5731	-37.558 Percent change from Baseline Standard Deviation 36.5005
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	1.699 Percent change from Baseline Standard Deviation 32.1342	16.376 Percent change from Baseline Standard Deviation 48.5494
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	5.577 Percent change from Baseline Standard Deviation 36.3036	0.029 Percent change from Baseline Standard Deviation 35.1807
Percent Change From Baseline at All Other Time Points Through Week 104 for TG Week 12	-7.258 Percent change from Baseline Standard Deviation 49.6990	-6.133 Percent change from Baseline Standard Deviation 56.7548	-8.327 Percent change from Baseline Standard Deviation 43.4273
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	-37.161 Percent change from Baseline Standard Deviation 23.9242	-32.643 Percent change from Baseline Standard Deviation 36.4318
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	-51.346 Percent change from Baseline Standard Deviation 27.1808	-46.856 Percent change from Baseline Standard Deviation 25.1360
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	-53.866 Percent change from Baseline Standard Deviation 27.5741	-52.031 Percent change from Baseline Standard Deviation 26.8624
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	-57.852 Percent change from Baseline Standard Deviation 24.1398	-53.426 Percent change from Baseline Standard Deviation 25.5749
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	-54.381 Percent change from Baseline Standard Deviation 28.4810	-42.380 Percent change from Baseline Standard Deviation 73.2737
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	-54.885 Percent change from Baseline Standard Deviation 18.5911	-39.245 Percent change from Baseline Standard Deviation 39.9507
Percent Change From Baseline at All Other Time Points Through Week 104 for TG Week 56	-47.707 Percent change from Baseline Standard Deviation 32.0150	-55.648 Percent change from Baseline Standard Deviation 26.7620	-39.349 Percent change from Baseline Standard Deviation 35.5528
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	-51.253 Percent change from Baseline Standard Deviation 21.5583	-46.107 Percent change from Baseline Standard Deviation 37.3356
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	-47.765 Percent change from Baseline Standard Deviation 27.6927	-28.860 Percent change from Baseline Standard Deviation 55.1679
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	-45.352 Percent change from Baseline Standard Deviation 25.8574	-34.126 Percent change from Baseline Standard Deviation 50.4118

Measure	All Age Groups n=28 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=14 Participants Patients aged 5-10 years.	Age 11-17 Years n=14 Participants Patients aged 11-17 years.
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	13.509 Percent change from Baseline Standard Deviation 36.2732	-15.847 Percent change from Baseline Standard Deviation 17.2462
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 26.3140	-6.564 Percent change from Baseline Standard Deviation 29.3156	-26.775 Percent change from Baseline Standard Deviation 15.7748
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	-25.915 Percent change from Baseline Standard Deviation 38.3512	-37.940 Percent change from Baseline Standard Deviation 28.7510
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 34.5930	-24.776 Percent change from Baseline Standard Deviation 37.6534	-40.212 Percent change from Baseline Standard Deviation 24.8480
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	24.269 Percent change from Baseline Standard Deviation 84.1451	-6.072 Percent change from Baseline Standard Deviation 23.8773
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	13.870 Percent change from Baseline Standard Deviation 54.0530	-13.327 Percent change from Baseline Standard Deviation 30.7067
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	-3.961 Percent change from Baseline Standard Deviation 58.6273	-31.540 Percent change from Baseline Standard Deviation 26.7998
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	-15.021 Percent change from Baseline Standard Deviation 38.1874	-42.728 Percent change from Baseline Standard Deviation 21.9120
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	-23.314 Percent change from Baseline Standard Deviation 36.5744	-46.081 Percent change from Baseline Standard Deviation 20.1331
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 50.1160	-27.534 Percent change from Baseline Standard Deviation 46.6328	-10.877 Percent change from Baseline Standard Deviation 55.6561
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	-21.547 Percent change from Baseline Standard Deviation 36.2351	-32.741 Percent change from Baseline Standard Deviation 23.6913
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	-20.400 Percent change from Baseline Standard Deviation 39.7347	-40.480 Percent change from Baseline Standard Deviation 21.6021
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	-8.621 Percent change from Baseline Standard Deviation 43.0927	-35.145 Percent change from Baseline Standard Deviation 20.2017
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	-17.270 Percent change from Baseline Standard Deviation 47.3261	-41.170 Percent change from Baseline Standard Deviation 27.8522
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	-14.691 Percent change from Baseline Standard Deviation 33.9040	-36.621 Percent change from Baseline Standard Deviation 33.5132
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	-9.136 Percent change from Baseline Standard Deviation 47.0506	-41.989 Percent change from Baseline Standard Deviation 24.2691
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	-19.135 Percent change from Baseline Standard Deviation 48.3708	-30.295 Percent change from Baseline Standard Deviation 26.4884
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	-16.468 Percent change from Baseline Standard Deviation 44.0841	-37.229 Percent change from Baseline Standard Deviation 31.2661

Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 11-17 years.
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	-10.581 Percent change from Baseline Standard Deviation 14.9489	9.825 Percent change from Baseline Standard Deviation 32.7827
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	-7.941 Percent change from Baseline Standard Deviation 22.3051	-2.921 Percent change from Baseline Standard Deviation 34.6428
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	-38.768 Percent change from Baseline Standard Deviation 21.4831	-27.664 Percent change from Baseline Standard Deviation 29.4691
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	-56.120 Percent change from Baseline Standard Deviation 26.2125	-32.988 Percent change from Baseline Standard Deviation 41.9967
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	-54.594 Percent change from Baseline Standard Deviation 28.2165	-26.197 Percent change from Baseline Standard Deviation 42.1040
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	-54.921 Percent change from Baseline Standard Deviation 27.6549	-29.839 Percent change from Baseline Standard Deviation 39.5405
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	-57.568 Percent change from Baseline Standard Deviation 26.0987	-28.596 Percent change from Baseline Standard Deviation 43.8568
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	-57.116 Percent change from Baseline Standard Deviation 31.2556	-20.317 Percent change from Baseline Standard Deviation 68.9419
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 41.9020	-54.241 Percent change from Baseline Standard Deviation 26.5277	-22.111 Percent change from Baseline Standard Deviation 47.2744
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	-49.165 Percent change from Baseline Standard Deviation 33.2083	-29.213 Percent change from Baseline Standard Deviation 40.5453
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	-47.527 Percent change from Baseline Standard Deviation 33.5384	-25.882 Percent change from Baseline Standard Deviation 46.3515
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	-48.484 Percent change from Baseline Standard Deviation 25.6330	-30.233 Percent change from Baseline Standard Deviation 39.9837
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	-13.695 Percent change from Baseline Standard Deviation 24.7560	-15.266 Percent change from Baseline Standard Deviation 30.9105
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	-51.512 Percent change from Baseline Standard Deviation 24.7014	-40.421 Percent change from Baseline Standard Deviation 28.5476
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	-60.174 Percent change from Baseline Standard Deviation 23.4381	-45.281 Percent change from Baseline Standard Deviation 25.1244
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	-59.358 Percent change from Baseline Standard Deviation 27.4688	-42.614 Percent change from Baseline Standard Deviation 32.8937
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	-55.596 Percent change from Baseline Standard Deviation 32.8921	-40.934 Percent change from Baseline Standard Deviation 37.5141
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	-45.043 Percent change from Baseline Standard Deviation 34.3337	-26.677 Percent change from Baseline Standard Deviation 35.1331

Measure	All Age Groups n=43 Participants Patients from all age groups have been evaluated together.	Age 5-10 Years n=20 Participants Patients aged 5-10 years.	Age 11-17 Years n=23 Participants Patients aged 11-17 years.
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	25.145 Percent change from Baseline Standard Deviation 32.0096	-3.620 Percent change from Baseline Standard Deviation 32.4844
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	22.486 Percent change from Baseline Standard Deviation 34.6933	3.044 Percent change from Baseline Standard Deviation 29.3561
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	29.691 Percent change from Baseline Standard Deviation 32.6733	3.574 Percent change from Baseline Standard Deviation 28.2358
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 19.6280	7.659 Percent change from Baseline Standard Deviation 16.3760	-4.236 Percent change from Baseline Standard Deviation 20.9511
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	12.855 Percent change from Baseline Standard Deviation 29.0476	-3.010 Percent change from Baseline Standard Deviation 21.1047
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	8.692 Percent change from Baseline Standard Deviation 27.6073	1.322 Percent change from Baseline Standard Deviation 30.7607
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	19.392 Percent change from Baseline Standard Deviation 24.6238	-3.984 Percent change from Baseline Standard Deviation 34.1243
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	17.873 Percent change from Baseline Standard Deviation 30.1872	-4.387 Percent change from Baseline Standard Deviation 33.2219
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	24.967 Percent change from Baseline Standard Deviation 28.3089	-0.208 Percent change from Baseline Standard Deviation 32.8835
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	19.883 Percent change from Baseline Standard Deviation 27.9809	2.425 Percent change from Baseline Standard Deviation 42.0330
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 29.1750	21.636 Percent change from Baseline Standard Deviation 31.0975	0.887 Percent change from Baseline Standard Deviation 23.3045
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	21.183 Percent change from Baseline Standard Deviation 29.0036	-0.326 Percent change from Baseline Standard Deviation 29.1404
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	17.203 Percent change from Baseline Standard Deviation 31.1788	6.010 Percent change from Baseline Standard Deviation 30.2457
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	26.224 Percent change from Baseline Standard Deviation 38.9780	5.994 Percent change from Baseline Standard Deviation 33.6550
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	19.607 Percent change from Baseline Standard Deviation 31.2092	10.171 Percent change from Baseline Standard Deviation 36.9795
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	14.424 Percent change from Baseline Standard Deviation 29.7110	2.651 Percent change from Baseline Standard Deviation 30.4028
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	25.097 Percent change from Baseline Standard Deviation 42.3692	3.256 Percent change from Baseline Standard Deviation 30.0858
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	31.674 Percent change from Baseline Standard Deviation 35.7852	3.587 Percent change from Baseline Standard Deviation 28.7665

Measure	Age 5-10 Years n=20 participants at risk Patients aged 5-10 years.	Age 11-17 Years n=23 participants at risk Patients aged 11-17 years.
Investigations Transaminases increased	5.0% 1/20 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	0.00% 0/23 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Injury, poisoning and procedural complications Arteriovenous fistula thrombosis	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	4.3% 1/23 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Injury, poisoning and procedural complications Shunt occlusion	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	4.3% 1/23 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Injury, poisoning and procedural complications Subdural haematoma	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	4.3% 1/23 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Vascular disorders Aortic arteriosclerosis	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	4.3% 1/23 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Cardiac disorders Aortic valve disease mixed	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	4.3% 1/23 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Cardiac disorders Chest pain	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	4.3% 1/23 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
General disorders Vascular device occlusion	10.0% 2/20 • Number of events 2 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	0.00% 0/23 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Eye disorders Cataract	5.0% 1/20 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	0.00% 0/23 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	4.3% 1/23 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Renal and urinary disorders Nephrolithiasis	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	4.3% 1/23 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Musculoskeletal and connective tissue disorders Tendon disorder	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	4.3% 1/23 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Infections and infestations Vascular device infection	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	8.7% 2/23 • Number of events 2 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Infections and infestations Appendicitis	5.0% 1/20 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	0.00% 0/23 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.
Infections and infestations Septic shock	0.00% 0/20 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.	4.3% 1/23 • Number of events 1 • AEs were monitored throughout the study from the time of informed consent through Week 108. Safety variables included evaluations of AEs, laboratory test results (including assessment of bone health), vital signs, ECGs, pulmonary function tests, and imaging of the liver. In addition, available standard of care echocardiography results were reviewed.