Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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18 March 2020 |
Main ID: |
EUCTR2017-000351-95-FR |
Date of registration:
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23/06/2017 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol level
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Scientific title:
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A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH |
Date of first enrolment:
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18/07/2017 |
Target sample size:
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54 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-000351-95 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: yes
Randomised: yes
Open: no
Single blind: no
Double blind: yes
Parallel group: yes
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: yes
Other: no
Number of treatment arms in the trial: 2
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): yes
Therapeutic use (Phase IV): no
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Countries of recruitment
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Austria
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Belgium
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Bulgaria
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Canada
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Croatia
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Czech Republic
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Denmark
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France
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Germany
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Greece
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Israel
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Italy
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Netherlands
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Poland
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Portugal
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Serbia
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Slovenia
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South Africa
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Spain
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Sweden
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Tunisia
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Turkey
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Ukraine
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United Kingdom
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United States
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Contacts
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Name:
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Clinical Trial Information
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Address:
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777 Old Saw Mill River Road
10591
Tarrytown
United States |
Telephone:
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Email:
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clinicaltrials@regeneron.com |
Affiliation:
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Regeneron Pharmaceuticals, Inc. |
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Name:
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Clinical Trial Information
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Address:
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777 Old Saw Mill River Road
10591
Tarrytown
United States |
Telephone:
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Email:
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clinicaltrials@regeneron.com |
Affiliation:
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Regeneron Pharmaceuticals, Inc. |
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Key inclusion & exclusion criteria
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Inclusion criteria: 1. Males and females =12 years of age at the time of the screening visit.
2. Adolescents =12 years of age and <18 years of age should be >50 kg at the time of the screening visit.
3. Diagnosis of HoFH homozygous familial hypercholesterolemia (HoFH)
4. Receiving a stable dose of a statin at the screening visit Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 44 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
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 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.
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
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Health Condition(s) or Problem(s) studied
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HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA MedDRA version: 20.0
Level: LLT
Classification code 10057080
Term: Homozygous familial hypercholesterolemia
System Organ Class: 100000004850
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Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
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Intervention(s)
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Trade Name: Praluent Pharmaceutical Form: Solution for injection in pre-filled pen INN or Proposed INN: ALIROCUMAB CAS Number: 1245916-14-6 Other descriptive name: ALIROCUMAB Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 150- Pharmaceutical form of the placebo: Solution for injection in pre-filled pen Route of administration of the placebo: Subcutaneous use
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Primary Outcome(s)
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Main Objective: The primary objective of the study is to demonstrate the reduction of LDL-C with alirocumab 150 mg subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment.
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Secondary Objective: The secondary objectives of the study are: • To evaluate the effect of alirocumab 150 mg Q2W on other lipid parameters (i.e., apolipoprotein [Apo] A-1 and B, non-high-density lipoprotein cholesterol [non-HDL-C], total cholesterol [TC], proportion of patients with 15%, 30%, and 50% LDL-C reductions, Lp(a), HDL-C, triglycerides [TG]) in patients with HoFH • To evaluate the safety and tolerability of alirocumab 150 mg SC Q2W in patients with HoFH • To assess the pharmacokinetics of alirocumab 150 mg SC Q2W in patients with HoFH • To assess the potential development of anti-drug (alirocumab) antibodies
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Timepoint(s) of evaluation of this end point: Screening, Baseline, Week 12
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Primary end point(s): The primary efficacy endpoint is the percent change in LDL-C from baseline to week 12 in the ITT population for alirocumab 150 mg Q2W as compared with placebo in patients with HoFH. The percent change in LDL-C from baseline to week 12 is defined as: 100 x (LDL-C value at week 12 -LDL-C value at baseline) LDL-C value at baseline
For LDL-C analysis, both calculated and measured LDL-C values will be taken into account. In case both calculated and measured LDL-C values are available for the same sampling time point, the measured LDL-C will be considered. The baseline LDL-C value will be the last LDL-C value obtained before the first dose of double-blind-study drug. For randomized but not-treated patients, baseline will be defined as the last value before randomization. The LDL-C at week 12 will be the LDL-C value obtained within the week 12 analysis window, regardless of adherence to treatment (ITT estimand). All calculated and measured LDL-C values (scheduled or unscheduled, fasting or not fasting) may be used for the primary efficacy endpoint, if appropriate, according to the above definition. The analysis window used to allocate a time point to a measurement will be defined in the statistical analysis plan (SAP).
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Secondary Outcome(s)
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Secondary end point(s): 4.2.2.1. Key Secondary Efficacy Endpoints
• The percent change in Apo B from baseline to week 12 (ITT estimand).
• The percent change in non-HDL-C from baseline to week 12 (ITT estimand).
• The percent change in total cholesterol from baseline to week 12 (ITT estimand).
• Proportion of patients with =15% reduction in LDL-C at week 12 (ITT estimand).
• Proportion of patients with =30% reduction in LDL-C at week 12 (ITT estimand).
• The percent change in Lp(a) from baseline to week 12 (ITT estimand).
• Proportion of patients with =50% reduction in LDL-C at week 12 (ITT estimand).
• The percent change in HDL-C from baseline to week 12 (ITT estimand).
• The percent change in fasting TG from baseline to week 12 (ITT estimand).
• The percent change in Apo A-1 from baseline to week 12 (ITT estimand).
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Timepoint(s) of evaluation of this end point: Screening, Baseline, Week 12
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Secondary ID(s)
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R727-CL-1628
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Source(s) of Monetary Support
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Regeneron Pharmaceuticals, Inc.
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Ethics review
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Status: Approved
Approval date: 06/07/2017
Contact:
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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