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Homozygous Familial HypercholesterolemiaPhase 3NCT02765841

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

A Phase 3 study testing Lomitapide in people with Homozygous Familial Hypercholesterolemia, run by Aegerion Pharmaceuticals, Inc..

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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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. Read more about this study

Who is it for?
Ages 5 to 17 years
Conditions the study is for
Homozygous Familial Hypercholesterolemia
What is being tested?
Lomitapide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
May 2016to Dec 2019
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Male or female aged ≥5 and <18 years with diagnosed functional HoFH
  • 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
  • Negative pregnancy test at Screening and during the study for females of child bearing age
  • Potentially sexually active female patients who are of child-bearing age must either be sexually abstinent or follow two acceptable methods of contraception

Who cannot join

  • Other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolemia (e.g., nephrotic syndrome, hypothyroidism).
  • Abnormal liver function test at Screening
  • Moderate or severe hepatic impairment or active liver disease
  • Serum creatine phosphokinase (CPK) level >2 × ULN.
  • Chronic renal insufficiency
  • History of drug abuse within the last 3 years or habitual alcohol consumption
Show all 15Show fewer
  • New York Heart Association (NYHA) Class III or IV congestive heart failure.
  • Uncontrolled hypertension
  • In the judgment of the PI, precocious or delayed puberty or endocrine disorder that would affect growth
  • History of non-skin malignancy or other cancers occurring within the past 3 years
  • History of inflammatory bowel disease or other malabsorption syndrome or a history of bowel resection, gastric bypass, or other weight loss surgical procedure.
  • Use of mipomersen within 6 months of Screening.
  • Any medical condition for which the life expectancy is predicted to be less than 5 years.
  • 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.
  • Participation in an interventional clinical study within 6 weeks for a statin therapy or within 6 months for any other unapproved therapy.

About this study

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

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.

Being tested
Lomitapide
Keywords
HoFH
Medical subject headings
Homozygous Familial Hypercholesterolemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LomitapideDrug

    Also called Juxtapid, Lojuxta

Study groups in NCT02765841
Study groupRoleWhat they receive
LomitapideExperimentalNot described
Receives: Drug: Lomitapide

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percent change in LDL-C

    Measured: Baseline, Week 24

Secondary outcomes (27)

  1. Percent Change in TC

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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)

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: Week 24 through Week 104

  11. Changes in LDL apheresis

    Measured: 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

    Measured: 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.

    Measured: Week 24 and through Week 108

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

    Measured: Baseline through Year 2

  15. Reported Adverse Events

    Measured: Baseline through Year 2

  16. Electrocardiogram (ECG) changes

    Measured: Baseline through Year 2

  17. Pulmonary function tests (PFTs)

    Measured: Baseline through Year 2

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

    Measured: Baseline through Year 2

  19. Height Measurement

    Measured: Baseline through Year 2

  20. Weight Measurement

    Measured: Baseline through Year 2

  21. Body Mass Measurement

    Measured: Baseline through Year 2

  22. Tanner Staging

    Measured: Baseline through Year 2

  23. Percent change in hepatic fat

    Measured: Baseline through Year 2

  24. Blood Pressure

    Measured: Baseline through Year 2

  25. Heart Rate

    Measured: Baseline through Year 2

  26. Temperature

    Measured: Baseline through Year 2

  27. Respiration (breaths/min)

    Measured: Baseline through Year 2

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 May 2016 (estimated)
  2. Study startMay 2016
  3. Primary completionMar 2017 (estimated)
  4. Study completionDec 2019 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02765841
Sponsor's protocol ID
AEGR-733-020
Status last verified
Feb 2018
First submitted
27 Apr 2016
Last updated
22 Feb 2018
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Lomitapide in people with Homozygous Familial Hypercholesterolemia, run by Aegerion Pharmaceuticals, Inc..

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 22 Feb 2018. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent change in LDL-C (time frame: Baseline, Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02765841.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Feb 2018.

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