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WithdrawnNCT02765841Updated Feb 22, 2018

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

A Phase 3 interventional study of Lomitapide in Homozygous Familial Hypercholesterolemia, sponsored by Aegerion Pharmaceuticals, Inc.. Withdrawn. Open to participants aged 5 Years to 17 Years. Per ClinicalTrials.gov, last updated 2018-02-22.

Sponsored by Aegerion Pharmaceuticals, Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
0
Allocation
Not applicable
Ages
5 Years to 17 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Homozygous Familial Hypercholesterolemia

Keywords

  • HoFH
03

In context

Hyperlipoproteinemia Type II

245 studies on the registry are indexed under Hyperlipoproteinemia Type II; 52 are open to participants now.

Browse Hyperlipoproteinemia Type II studies →

Lead sponsor

Aegerion Pharmaceuticals, Inc. is the lead sponsor of 17 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
5 Years to 17 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
0 participants (actual)

Study arms

  • Experimental
    Lomitapide

    Drug: Lomitapide

Interventions

  • DrugLomitapide

    Also known as: Juxtapid, Lojuxta

06

What researchers measure

Primary outcomes

  1. Percent change in LDL-C

    Time frame: Baseline, Week 24

Secondary outcomes

  1. 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

  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

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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

  10. Changes in lipid-lowering therapy

    Time frame: Week 24 through Week 104

  11. Changes in LDL apheresis

    Time frame: Week 24 through Week 104

  12. 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

  13. 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

  14. Changes in laboratory parameters (including hepatic and renal function)

    Time frame: Baseline through Year 2

  15. Reported Adverse Events

    Time frame: Baseline through Year 2

  16. Electrocardiogram (ECG) changes

    Time frame: Baseline through Year 2

  17. Pulmonary function tests (PFTs)

    Time frame: Baseline through Year 2

  18. Bone health/age (x-ray of the wrist)

    Time frame: Baseline through Year 2

  19. Height Measurement

    Time frame: Baseline through Year 2

  20. Weight Measurement

    Time frame: Baseline through Year 2

  21. Body Mass Measurement

    Time frame: Baseline through Year 2

  22. Tanner Staging

    Time frame: Baseline through Year 2

  23. Percent change in hepatic fat

    Time frame: Baseline through Year 2

  24. Blood Pressure

    Time frame: Baseline through Year 2

  25. Heart Rate

    Time frame: Baseline through Year 2

  26. Temperature

    Time frame: Baseline through Year 2

  27. Respiration (breaths/min)

    Time frame: Baseline through Year 2

07

Study locations

No study locations are listed for this record.

08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 22, 2018, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT02765841
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
May 9, 2016
Start date
May 2016
Primary completion
Mar 2017 (estimated)
Completion
Dec 2019 (estimated)
Last update
Feb 22, 2018

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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This study is withdrawn, as verified in Feb 2018. You cannot join it, but the record below documents what was studied.

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