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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Recruitment Status : Withdrawn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.

Tracking Information
First Submitted Date  ICMJE April 27, 2016
First Posted Date  ICMJE May 9, 2016
Last Update Posted Date February 22, 2018
Study Start Date  ICMJE May 2016
Estimated Primary Completion Date March 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 6, 2016)
Percent change in LDL-C [ Time Frame: Baseline, Week 24 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 6, 2016)
  • 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 ]
  • 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 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 ]
  • 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 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 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 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 ]
  • 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 ]
  • Changes in lipid-lowering therapy [ Time Frame: Week 24 through Week 104 ]
  • Changes in LDL apheresis [ Time Frame: Week 24 through Week 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 ]
  • 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 ]
  • Changes in laboratory parameters (including hepatic and renal function) [ Time Frame: Baseline through Year 2 ]
  • Reported Adverse Events [ Time Frame: Baseline through Year 2 ]
  • Electrocardiogram (ECG) changes [ Time Frame: Baseline through Year 2 ]
  • Pulmonary function tests (PFTs) [ Time Frame: Baseline through Year 2 ]
  • Bone health/age (x-ray of the wrist) [ Time Frame: Baseline through Year 2 ]
  • Height Measurement [ Time Frame: Baseline through Year 2 ]
  • Weight Measurement [ Time Frame: Baseline through Year 2 ]
  • Body Mass Measurement [ Time Frame: Baseline through Year 2 ]
  • Tanner Staging [ Time Frame: Baseline through Year 2 ]
  • Percent change in hepatic fat [ Time Frame: Baseline through Year 2 ]
  • Blood Pressure [ Time Frame: Baseline through Year 2 ]
  • Heart Rate [ Time Frame: Baseline through Year 2 ]
  • Temperature [ Time Frame: Baseline through Year 2 ]
  • Respiration (breaths/min) [ Time Frame: Baseline through Year 2 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Homozygous Familial Hypercholesterolemia
Intervention  ICMJE Drug: Lomitapide
Other Name: Juxtapid, Lojuxta
Study Arms  ICMJE Experimental: Lomitapide
Intervention: Drug: Lomitapide
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: September 1, 2016)
0
Original Estimated Enrollment  ICMJE
 (submitted: May 6, 2016)
45
Estimated Study Completion Date  ICMJE December 2019
Estimated Primary Completion Date March 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02765841
Other Study ID Numbers  ICMJE AEGR-733-020
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Aegerion Pharmaceuticals, Inc.
Study Sponsor  ICMJE Aegerion Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Aegerion Pharmaceuticals, Inc.
Verification Date February 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP