An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (ODYSSEY KIDS)
Status and phase
Completed
Phase 2
Conditions
Hypercholesterolaemia
Treatments
Drug: alirocumab SAR236553 (REGN727)
Drug: fenofibrate
Drug: statins
Drug: ezetimibe
Drug: nicotinic acid
Drug: cholestyramine
Drug: omega-3 fatty acids
Study type
Interventional
Funder types
Industry
Identifiers
Details and patient eligibility
About
Primary Objective:
To evaluate the effect of alirocumab on low-density lipoprotein cholesterol (LDL-C) levels after 8 weeks of treatment in heterozygous familial hypercholesterolemia (heFH) participants aged of 8 to 17 years, with LDL-C >=130 milligrams per deciliter (mg/dL) (3.37 millimoles per litre [mmol/L]) on optimal stable daily dose of statin therapy +/- other lipid modifying therapies (LMTs) or a stable dose of non-statin LMTs in case of intolerance to statins for at least 4 weeks prior to the screening period.
Secondary Objective:
- To evaluate the safety and tolerability of alirocumab.
- To evaluate the pharmacokinetics profile of alirocumab.
- To evaluate the effects of alirocumab on other lipid parameters.
Full description
For Cohorts 1 to 3, a study duration of approximately 16-23 weeks (screening period: up to 6 (+1) weeks, open-label dose finding treatment period: 8 weeks, follow up period: 6-8 weeks).
For Cohort 4, a study duration of approximately 14-19 weeks (screening period up to 6 [+1] weeks, open-label dose finding treatment period: 12 weeks).
Optional extension period: up to a maximum of 2 years for the first participants enrolled in Cohorts 1 to 3, but a maximum of approximately 5 months for the first participants enrolled in Cohort 4.
For all participants who declined participation in the phase 3 study, their last alirocumab injection was on December 2018.
Enrollment
42 patients
Sex
All
Ages
8 to 17 years old
Volunteers
No Healthy Volunteers
Inclusion and exclusion criteria
Inclusion criteria :
- Children and adolescent male and female participants aged of 8 to 17 years at the time of signed informed consent. For Russia only: Male and female participants aged >=12 and <=17 years at the time of signed informed consent.
- Participants with diagnosis of heterozygous familial hypercholesterolemia (heFH) through genotyping or clinical criteria.
- Participants treated with optimal dose of statin +/- other LMT(s) or non-statin LMT(s) if statin intolerant at stable dose for at least 4 weeks prior to screening lipid sampling.
- Participants with calculated LDL-C greater than or equal to 130 mg/dL (>=3.37 mmol/L) at the screening visit.
- Participants with body weight greater than or equal to 25 kg.
- Participants aged of 8 to 9 years to be at Tanner stage 1 and participants aged of 10 to 17 years to be at least at Tanner stage 2 in their development.
- A signed informed consent indicating parental permission with or without participant assent.
Exclusion criteria:
- Participant with secondary hyperlipidemia.
- Diagnosis of homozygous familial hypercholesterolemia.
- Participant who had received lipid apheresis treatment within 2 months prior to the screening period, or has plans to receive it during the study.
- Known history of type 1 or type 2 diabetes mellitus.
- Known history of thyroid disease.
- Known history of hypertension.
- Fasting triglycerides >350 mg/dL (3.95 mmol/L).
- Severe renal impairment (i.e., estimated glomerular filtration rate [eGFR] <30 mL/min/1.73 m^2).
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 x upper limit of normal (ULN).
- Creatinine phosphokinase (CPK) >3 x ULN.
The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Trial design
Primary purpose
Treatment
Allocation
Non-Randomized
Interventional model
Sequential Assignment
Masking
None (Open label)
42 participants in 8 patient groups
Cohort 1 - Alirocumab 30 mg Q2W: <50 kg
Experimental group
Description:
Period 1: Participants with body weight less than (\<) 50 kilograms (kg) received subcutaneous (SC) injection of alirocumab 30 milligram(mg) administered every 2 weeks (Q2W) up to 8 weeks added to lipid modifying therapy (LMT).
Period 2: Participants with body weight \< 50 kg received SC injection of alirocumab 30 mg administered Q2W from Week 16 until they started receiving dose matching to Cohort 2 dosage including dose adjustment to body weight as required. Cohort 2 dosage was: if body weight was still \< 50 kg, participants received SC injection of alirocumab 40 mg administered Q2W until Week 130; if body weight was \> = 50 kg participants received SC injection of alirocumab 75 mg administered Q2W until Week 130.
Treatment:
Drug: omega-3 fatty acids
Drug: nicotinic acid
Drug: cholestyramine
Drug: fenofibrate
Drug: statins
Drug: ezetimibe
Drug: alirocumab SAR236553 (REGN727)
Cohort 1 - Alirocumab 50 mg Q2W: >=50 kg
Experimental group
Description:
Period 1: Participants with body weight greater than or equal to (\>=) 50 kg received SC injection of alirocumab 50 mg administered Q2W up to 8 weeks added to LMT.
Period 2: Participants with body weight \>= 50 kg received SC injection of alirocumab 50 mg administered Q2W from Week 16 until switch to Cohort 2 dosage then SC injection of alirocumab 75 mg administered Q2W until Week 130.
Treatment:
Drug: omega-3 fatty acids
Drug: nicotinic acid
Drug: cholestyramine
Drug: fenofibrate
Drug: statins
Drug: ezetimibe
Drug: alirocumab SAR236553 (REGN727)
Cohort 2 - Alirocumab 40 mg Q2W: <50 kg
Experimental group
Description:
Period 1: Participants with body weight \< 50 kg received SC injection of alirocumab 40 mg administered Q2W up to 8 weeks added to LMT.
Period 2: Participants with body weight \< 50 kg received SC injection of alirocumab 40 mg administered Q2W from Week 16 until switch of dosage in Cohorts 1 and 3. If body weight was still \< 50 kg, participants continued to receive SC injection of alirocumab 40 mg administered Q2W until Week 130; if body weight was \> = 50 kg participants received SC injection of alirocumab 75 mg administered Q2W until Week 130.
Treatment:
Drug: omega-3 fatty acids
Drug: nicotinic acid
Drug: cholestyramine
Drug: fenofibrate
Drug: statins
Drug: ezetimibe
Drug: alirocumab SAR236553 (REGN727)
Cohort 2 - Alirocumab 75 mg Q2W: >=50 kg
Experimental group
Description:
Period 1: Participants with body weight \>= 50 kg received SC injection of alirocumab 75 mg administered Q2W up to 8 weeks added to LMT.
Period 2: Participants with body weight \>= 50 kg received SC injection of alirocumab 75 mg administered Q2W from Week 16 until Week 130.
Treatment:
Drug: omega-3 fatty acids
Drug: nicotinic acid
Drug: cholestyramine
Drug: fenofibrate
Drug: statins
Drug: ezetimibe
Drug: alirocumab SAR236553 (REGN727)
Cohort 3 - Alirocumab 75 mg Q4W: <50 kg
Experimental group
Description:
Period 1: Participants with body weight \< 50 kg received SC injection of alirocumab 75 mg administered every 4 weeks (Q4W) up to 8 weeks added to LMT.
Period 2: Participants with body weight \< 50 kg received SC injection of alirocumab 75 mg administered Q4W from Week 14 until switch to Cohort 2 dosage including dose adjustment to body weight as required, then Cohort 2 dosage: if body weight was still \< 50 kg, participants received SC injection of alirocumab 40 mg administered Q2W until Week 130; if body weight was \> = 50 kg participants received SC injection of alirocumab 75 mg administered Q2W until Week 130.
Treatment:
Drug: omega-3 fatty acids
Drug: nicotinic acid
Drug: cholestyramine
Drug: fenofibrate
Drug: statins
Drug: ezetimibe
Drug: alirocumab SAR236553 (REGN727)
Cohort 3 - Alirocumab 150 mg Q4W: >=50 kg
Experimental group
Description:
Period 1: Participants with body weight \>= 50 kg received SC injection of alirocumab 150 mg administered Q4W up to Week 8 added to LMT.
Period 2: Participants with body weight \>= 50 kg received SC injection of alirocumab 150 mg administered Q4W from Week 14 until switch to Cohort 2 dosage then SC injection of alirocumab 75 mg administered Q2W until Week 130.
Treatment:
Drug: omega-3 fatty acids
Drug: nicotinic acid
Drug: cholestyramine
Drug: fenofibrate
Drug: statins
Drug: ezetimibe
Drug: alirocumab SAR236553 (REGN727)
Cohort 4 - Alirocumab 150 mg Q4W: <50 kg
Experimental group
Description:
Period 1: Participants with body weight \< 50 kg received SC injection of alirocumab 150 mg administered Q4W up to 12 weeks added to LMT.
Period 2: Participants with body weight \< 50 kg received SC injection of Alirocumab 150 mg administered Q4W from Week 12 until Week 48.
Treatment:
Drug: omega-3 fatty acids
Drug: nicotinic acid
Drug: cholestyramine
Drug: fenofibrate
Drug: statins
Drug: ezetimibe
Drug: alirocumab SAR236553 (REGN727)
Cohort 4 - Alirocumab 300 mg Q4W: >=50 kg
Experimental group
Description:
Period 1: Participants with body weight \>= 50 kg received SC injection of alirocumab 300 mg administered Q4W up to 12 weeks added to LMT.
Period 2: Participants with body weight \>= 50 kg received SC injection of alirocumab 300 mg administered Q4W from Week 12 until Week 48.
Treatment:
Drug: omega-3 fatty acids
Drug: nicotinic acid
Drug: cholestyramine
Drug: fenofibrate
Drug: statins
Drug: ezetimibe
Drug: alirocumab SAR236553 (REGN727)
Trial contacts and locations
16
Loading...
Data sourced from clinicaltrials.gov
Clinical trials
Research sites
Resources
Legal
© Copyright 2026 Veeva Systems