An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Heterozygous Familial Hypercholesterolemia

Phase 1

• Conditions: Hypercholesterolaemia; MedDRA version: 20.0Level: PTClassification code 10020603Term: HypercholesterolaemiaSystem Organ Class: 10027433 - Metabolism and nutrition disorders; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

• Registration Number: EUCTR2017-001903-60-NO
• Lead Sponsor: Sanofi-aventis recherche & développement

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-01-08
• Last Update Posted: 5 April 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 500

Inclusion Criteria

-Children and adolescent male and female patients aged 8 to 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 except for patients who have previously participated in the DFI14223 study.
-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: 500
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 body weight less than 25 kg.
-Patients aged of 8 to 9 years not at Tanner stage 1 and patients aged of 10 to 17 years not at least at Tanner stage 2 in their development.
-Patients 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.
-Patients with uncontrolled type 1 or type 2 diabetes mellitus.
-Patients with known uncontrolled thyroid disease.
-Patients with uncontrolled 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