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

Phase 1

• Conditions: Hypercholesterolaemia; MedDRA version: 19.1 Level: PT Classification code 10020603 Term: Hypercholesterolaemia System Organ Class: 10027433 - Metabolism and nutrition disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2015-003766-85-SE
• Lead Sponsor: sanofi-aventis recherche&developpement

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-06-01
• Study Completion: 2019-02-22
• Last Update Posted: 30 April 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 42

Inclusion Criteria

-Children and adolescent male and female patients age of 8 to 17 years at the time of signed informed consent. For Russia only: Male and female patients aged =12 and =17 years at the time of signed informed consent.
-Patients with diagnosis of heterozygous familial hypercholesterolemia (heFH) through genotyping or clinical criteria.
-Patients 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
-Patients with calculated LDL-C greater than or equal to 130 mg/dL (=3.37 mmol/L) at the screening visit
-Patients with body weight greater than or equal to 25kg.
-Patients age of 8 to 9 years to be at Tanner stage 1 and patients age 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 patient assent.

Are the trial subjects under 18? yes
Number of subjects for this age range: 80
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

-Patient with secondary hyperlipidemia.
-Diagnosis of homozygous familial hypercholesterolemia.
-Patient who has 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 (ie, 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<br> Secondary Objective: -To evaluate the safety and tolerability of alirocumab.<br> -To evaluate the pharmacokinetics profile of alirocumab.<br> -To evaluate the effects of alirocumab on other lipid parameters. <br> ;Main 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) patients age of 8 to 17 years, with LDL-C =130 mg/dL (3.37 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. ;Primary end point(s): Percent change in calculated LDL-C;Timepoint(s) of evaluation of this end point: From baseline to Week 8