ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 25 of 153 for:    "familial hypercholesterolemia"

Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH) (ODYSSEY HoFH)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03156621
Recruitment Status : Recruiting
First Posted : May 17, 2017
Last Update Posted : September 7, 2018
Sponsor:
Collaborator:
Sanofi
Information provided by (Responsible Party):
Regeneron Pharmaceuticals

Brief Summary:
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) with alirocumab subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment.

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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia
Actual Study Start Date : October 10, 2017
Estimated Primary Completion Date : January 10, 2019
Estimated Study Completion Date : June 17, 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Alirocumab

Arm Intervention/treatment
Experimental: Alirocumab SC Q2W

Alirocumab SC every 2 weeks (Q2W) from baseline (day 1) through week 10 during the double-blind treatment period

Starting at week 12, and continuing through week 22, participants will receive open-label alirocumab SC Q2W

Drug: Alirocumab
Alirocumab SC Q2W
Other Names:
  • PRALUENT®
  • REGN727
  • SAR236553

Experimental: Placebo SC Q2W

Matching placebo SC Q2W from baseline through week 10 during the double-blind treatment period

Starting at week 12, and continuing through week 22, participants will receive open-label alirocumab SC Q2W

Drug: Placebo
Matching placebo SC Q2W




Primary Outcome Measures :
  1. Percent change in LDL-C from baseline to Week 12 (Intent-to-Treat (ITT) population) [ Time Frame: Baseline to Week 12 ]
    ITT population: All randomized participants who had an evaluable primary endpoint


Secondary Outcome Measures :
  1. Percent change in apolipoprotein (Apo) B from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population

  2. Percent change in non-high-density lipoprotein cholesterol (non-HDL-C) from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population

  3. Percent change in total cholesterol (TC) from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population

  4. Proportion of participants with ≥15% reduction in LDL-C at week 12 [ Time Frame: At Week 12 ]
    ITT population

  5. Proportion of participants with ≥30% reduction in LDL-C at week 12 [ Time Frame: At Week 12 ]
    ITT population

  6. Percent change in lipoprotein(a) [Lp(a)] from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population

  7. Proportion of participants with ≥50% reduction in LDL-C at week 12 [ Time Frame: At Week 12 ]
    ITT population

  8. Percent change in HDL-C from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population

  9. Percent change in fasting triglycerides (TG) from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population

  10. Percent change in Apo A-1 from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population

  11. Percent change in LDL-C from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population

  12. Percent change in LDL-C from baseline to week 12 (Modified Intent-to-Treat (m)ITT population) [ Time Frame: Baseline to Week 12 ]
    Modified Intent-to-Treat (m)ITT population: All randomized participants who took at least 1 dose or part of a dose of double-blind investigational study drug and has an evaluable primary endpoint

  13. Percent change in Apo B from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT) population

  14. Percent change in non-HDL-C from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT) population

  15. Percent change in TC from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT population)

  16. Percent change in Lp(a) from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT population)

  17. Percent change in HDL-C from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT) population

  18. Percent change in fasting TG from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT) population

  19. Percent change in Apo A-1 from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT) population

  20. Proportion of patients with ≥15% reduction, ≥30% reduction, and ≥50% reduction in LDL-C at week 12 [ Time Frame: At Week 12 ]
    m(ITT) population

  21. Absolute change in the ratio of Apo B/Apo A-1 from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population

  22. Incidence of Adverse Events (AEs) [ Time Frame: Baseline to week 32 (End of Study) ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Note: The information listed below is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial, therefore not all inclusion/exclusion criteria are listed.

Key Inclusion Criteria

  1. Diagnosis of HoFH by at least 1 of the following genotype or clinical criteria (all patients on LDL apheresis must be diagnosed based on genotype):

    1. Documented homozygous or compound heterozygous mutations in both low-density lipoprotein receptor (LDLR) alleles
    2. Presence of homozygous or compound heterozygous mutations in Apo B or PCSK9
    3. Presence of double heterozygous mutations, i.e, mutations on different genes in the LDLR, Apo B or PCSK9 alleles
    4. Untreated TC >500 mg/dL (12.93 mmol/L) and TG <300 mg/dL (3.39 mmol/L) AND Both parents with history of TC >250 mg/dL (6.46 mmol/L) OR cutaneous or tendinous xanthoma before age 10
  2. Receiving a stable dose of a statin at the screening visit (documentation if statin ineffective or patient unable to tolerate statin)

Key Exclusion Criteria:

  1. Documented evidence of a null mutation in both LDLR alleles
  2. Use of a PCSK9 inhibitor within 10 weeks from screening visit
  3. Background medical lipid modifying therapy (LMT) that has not been stable for at least 4 weeks (6 weeks for fibrates, 24 weeks for mipomersen, 12 weeks for maximum tolerated dose of lomitapide) before the screening visit.
  4. LDL apheresis schedule/apheresis settings that have not been stable for at least 8 weeks before the screening visit or an apheresis schedule/settings that is not anticipated to be stable over the next 24 weeks.
  5. Use of nutraceuticals or over-the-counter (OTC) therapies known to affect lipids, at a dose/amount that has not been stable for at least 4 weeks prior to the screening visit or between the screening and randomization visits.
  6. Chronic use of systemic corticosteroids, unless on a stable regimen of 10 mg daily prednisone equivalent or less for at least 6 weeks prior to randomization. Note: topical, intra-articular, nasal, inhaled and ophthalmic steroid therapies are not considered as 'systemic' and are allowed
  7. Systolic blood pressure >160 mmHg or diastolic blood pressure >100 mmHg at the screening visit (1 repeat measurement is allowed).
  8. History of a myocardial infarction (MI), unstable angina leading to hospitalization, coronary artery bypass graft surgery, percutaneous coronary intervention , uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, valve replacement surgery, carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease within 3 months prior to the screening visit.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03156621


Contacts
Contact: Clinical Trials Administrator 844-734-6643 clinicaltrials@regeneron.com

  Show 36 Study Locations
Sponsors and Collaborators
Regeneron Pharmaceuticals
Sanofi
Investigators
Study Director: Clinical Trial Management Regeneron Pharmaceuticals

Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03156621     History of Changes
Other Study ID Numbers: R727-CL-1628
2017-000351-95 ( EudraCT Number )
First Posted: May 17, 2017    Key Record Dates
Last Update Posted: September 7, 2018
Last Verified: September 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs