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

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ClinicalTrials.gov Identifier: NCT02765841
Recruitment Status : Withdrawn
First Posted : May 9, 2016
Last Update Posted : February 22, 2018
Sponsor:
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.

Brief 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.

Condition or disease Intervention/treatment Phase
Homozygous Familial Hypercholesterolemia Drug: Lomitapide Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : May 2016
Estimated Primary Completion Date : March 2017
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Lomitapide

Arm Intervention/treatment
Experimental: Lomitapide Drug: Lomitapide
Other Name: Juxtapid, Lojuxta




Primary Outcome Measures :
  1. Percent change in LDL-C [ Time Frame: Baseline, Week 24 ]

Secondary Outcome Measures :
  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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

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Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02765841    
Other Study ID Numbers: AEGR-733-020
First Posted: May 9, 2016    Key Record Dates
Last Update Posted: February 22, 2018
Last Verified: February 2018
Keywords provided by Aegerion Pharmaceuticals, Inc.:
HoFH
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias