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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: EUCTR
Last refreshed on: 18 March 2020
Main ID:  EUCTR2017-000351-95-FR
Date of registration: 23/06/2017
Prospective Registration: Yes
Primary sponsor: Regeneron Pharmaceuticals, Inc.
Public title: A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol level
Scientific title: 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: 18/07/2017
Target sample size: 54
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-000351-95
Study type:  Interventional clinical trial of medicinal product
Study design:  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  
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Austria Belgium Bulgaria Canada Croatia Czech Republic Denmark France
Germany Greece Israel Italy Netherlands Poland Portugal Serbia
Slovenia South Africa Spain Sweden Tunisia Turkey Ukraine United Kingdom
United States
Contacts
Name: Clinical Trial Information   
Address:  777 Old Saw Mill River Road 10591 Tarrytown United States
Telephone:
Email: clinicaltrials@regeneron.com
Affiliation:  Regeneron Pharmaceuticals, Inc.
Name: Clinical Trial Information   
Address:  777 Old Saw Mill River Road 10591 Tarrytown United States
Telephone:
Email: clinicaltrials@regeneron.com
Affiliation:  Regeneron Pharmaceuticals, Inc.
Key inclusion & exclusion criteria
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
Health Condition(s) or Problem(s) studied
HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
MedDRA version: 20.0 Level: LLT Classification code 10057080 Term: Homozygous familial hypercholesterolemia System Organ Class: 100000004850
Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Intervention(s)

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

Primary Outcome(s)
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.
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
Timepoint(s) of evaluation of this end point: Screening, Baseline, Week 12
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).
Secondary Outcome(s)
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).
Timepoint(s) of evaluation of this end point: Screening, Baseline, Week 12
Secondary ID(s)
R727-CL-1628
Source(s) of Monetary Support
Regeneron Pharmaceuticals, Inc.
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 06/07/2017
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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