NCT02890992

An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

An 8-Week Open-Label, Sequential, Repeated Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia Followed by an Extension Phase

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 42 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 8 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed 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.

Conditions

• Hypercholesterolaemia

Interventions

Type	Name	Description
DRUG	alirocumab SAR236553 (REGN727)	Pharmaceutical form: solution Route of administration: subcutaneous injection
DRUG	statins	Pharmaceutical form: tablet Route of administration: oral
DRUG	ezetimibe	Pharmaceutical form:tablet Route of administration: oral
DRUG	cholestyramine	Pharmaceutical form:tablet Route of administration: oral
DRUG	fenofibrate	Pharmaceutical form: tablet Route of administration: oral
DRUG	omega-3 fatty acids	Pharmaceutical form: tablet Route of administration: oral
DRUG	nicotinic acid	Pharmaceutical form: tablet Route of administration: oral

Timeline

• Start date: 2016-09-15
• Primary completion: 2018-09-13
• Completion: 2019-02-22
• First posted: 2016-09-07
• Last updated: 2019-09-06
• Results posted: 2019-09-06

Locations

16 sites across 10 countries: United States, Canada, Czechia, France, Netherlands, Norway, Russia, South Africa, Spain, Sweden