Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02765841
• Date of registration: 27/04/2016
• Prospective Registration: Yes
• Primary sponsor: Aegerion Pharmaceuticals, Inc.
• Public title: Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy
• Scientific title: A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy
• Date of first enrolment: May 2016
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT02765841
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female aged =5 and <18 years with diagnosed functional HoFH

2. Patient must weigh at least 15 kg and be at or above the 10th percentile in BMI and at
least 10th percentile in height for age and gender based on CDC growth charts

3. Negative pregnancy test at Screening and during the study for females of child bearing
age

4. Potentially sexually active female patients who are of child-bearing age must either
be sexually abstinent or follow two acceptable methods of contraception

Exclusion Criteria:

1. Other forms of primary hyperlipoproteinemia and secondary causes of
hypercholesterolemia (e.g., nephrotic syndrome, hypothyroidism).

2. Abnormal liver function test at Screening

3. Moderate or severe hepatic impairment or active liver disease

4. Serum creatine phosphokinase (CPK) level >2 × ULN.

5. Chronic renal insufficiency

6. History of drug abuse within the last 3 years or habitual alcohol consumption

7. New York Heart Association (NYHA) Class III or IV congestive heart failure.

8. Uncontrolled hypertension

9. In the judgment of the PI, precocious or delayed puberty or endocrine disorder that
would affect growth

10. History of non-skin malignancy or other cancers occurring within the past 3 years

11. History of inflammatory bowel disease or other malabsorption syndrome or a history of
bowel resection, gastric bypass, or other weight loss surgical procedure.

12. Use of mipomersen within 6 months of Screening.

13. Any medical condition for which the life expectancy is predicted to be less than 5
years.

14. Any patient who is unable to avoid treatment with strong or moderate cytochrome P450
3A4 (CYP3A4) inhibitors, or other drugs contraindicated for use with lomitapide during
the study.

15. Participation in an interventional clinical study within 6 weeks for a statin therapy
or within 6 months for any other unapproved therapy.



Age minimum: 5 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Homozygous Familial Hypercholesterolemia

Intervention(s)

Drug: Lomitapide

Primary Outcome(s)

Percent change in LDL-C [Time Frame: Baseline, Week 24]

Secondary Outcome(s)

Changes in laboratory parameters (including hepatic and renal function) [Time Frame: Baseline through Year 2]

Heart Rate [Time Frame: Baseline through Year 2]

Percent change in Lp(a) [Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104]

Percent of patients achieving goal (LDL-C of <100 mg/dL [2.6 mmol/L] for patients without documented cardiovascular disease [CVD] at Baseline [Time Frame: Week 24 and through Week 108]

Body Mass Measurement [Time Frame: Baseline through Year 2]

Changes in LDL apheresis [Time Frame: Week 24 through Week 104]

Blood Pressure [Time Frame: Baseline through Year 2]

Height Measurement [Time Frame: Baseline through Year 2]

Percent change in HDL-C [Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104]

Percent Change in TC [Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104]

Changes in lipid-lowering therapy [Time Frame: Week 24 through Week 104]

Reported Adverse Events [Time Frame: Baseline through Year 2]

Pulmonary function tests (PFTs) [Time Frame: Baseline through Year 2]

Percent change in LDL-C [Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104]

Percent change in hepatic fat [Time Frame: Baseline through Year 2]

Temperature [Time Frame: Baseline through Year 2]

Weight Measurement [Time Frame: Baseline through Year 2]

Percent change in apo B [Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104]

Percent change in VLDL-C [Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104]

Percent of patients achieving goal LDL-C of <70 mg/dL [1.8 mmol/L]) for patients with documented CVD at Baseline. [Time Frame: Week 24 and through Week 108]

Electrocardiogram (ECG) changes [Time Frame: Baseline through Year 2]

Percent change in apo A-1 [Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104]

Bone health/age (x-ray of the wrist) [Time Frame: Baseline through Year 2]

Percent change in non-HDL-C [Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104]

Percent change in TG [Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104]

Respiration (breaths/min) [Time Frame: Baseline through Year 2]

Tanner Staging [Time Frame: Baseline through Year 2]

Secondary ID(s)

AEGR-733-020

Source(s) of Monetary Support

Please refer to primary and secondary sponsors