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

Homozygous Familial Hypercholesterolemia Clinical Trial

Official 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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02765841
• Other study ID #: AEGR-733-020
• Status: Withdrawn
• Phase: Phase 3
• First received: April 27, 2016
• Last updated: February 21, 2018
• Start date: May 2016
• Est. completion date: December 2019

Study information

• Verified date: February 2018
• Source: Aegerion Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a Phase 3 single-arm, open-label, international, multi-center clinical trial to evaluate the efficacy and safety of lomitapide in pediatric patients with HoFH who are receiving stable lipid-lowering therapy, including LDL apheresis. The study is comprised of a 12-week Run-in Period, a primary 24-week Efficacy Phase, followed by an 80-week Safety Phase.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: December 2019
• Est. primary completion date: March 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Homozygous Familial Hypercholesterolemia
• Hypercholesterolemia
• Hyperlipoproteinemia Type II

Intervention

Drug:
• Lomitapide

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Aegerion Pharmaceuticals, Inc.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent change in LDL-C		Baseline, Week 24
Secondary	Percent Change in TC		Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Secondary	Percent change in non-HDL-C		Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Secondary	Percent change in HDL-C		Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Secondary	Percent change in TG		Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Secondary	Percent change in VLDL-C		Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Secondary	Percent change in Lp(a)		Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Secondary	Percent change in apo B		Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Secondary	Percent change in apo A-1		Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Secondary	Percent change in LDL-C		Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Secondary	Changes in lipid-lowering therapy		Week 24 through Week 104
Secondary	Changes in LDL apheresis		Week 24 through Week 104
Secondary	Percent of patients achieving goal (LDL-C of <100 mg/dL [2.6 mmol/L] for patients without documented cardiovascular disease [CVD] at Baseline		Week 24 and through Week 108
Secondary	Percent of patients achieving goal LDL-C of <70 mg/dL [1.8 mmol/L]) for patients with documented CVD at Baseline.		Week 24 and through Week 108
Secondary	Changes in laboratory parameters (including hepatic and renal function)		Baseline through Year 2
Secondary	Reported Adverse Events		Baseline through Year 2
Secondary	Electrocardiogram (ECG) changes		Baseline through Year 2
Secondary	Pulmonary function tests (PFTs)		Baseline through Year 2
Secondary	Bone health/age (x-ray of the wrist)		Baseline through Year 2
Secondary	Height Measurement		Baseline through Year 2
Secondary	Weight Measurement		Baseline through Year 2
Secondary	Body Mass Measurement		Baseline through Year 2
Secondary	Tanner Staging		Baseline through Year 2
Secondary	Percent change in hepatic fat		Baseline through Year 2
Secondary	Blood Pressure		Baseline through Year 2
Secondary	Heart Rate		Baseline through Year 2
Secondary	Temperature		Baseline through Year 2
Secondary	Respiration (breaths/min)		Baseline through Year 2