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

Phase 1

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

• Registration Number: EUCTR2017-002297-39-BG
• Lead Sponsor: Sanofi-Aventis Recherche & Développement

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-07-31
• Last Update Posted: 10 August 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

-Patients genetically diagnosed with homozygous familial hypercholesterolemia (hoFH).
-Patients treated with optimal dose of statin +/- other lipid modifying therapies (LMTs), or non-statin LMTs if statin-intolerant at stable dose(s) for at least 4 weeks prior to screening lipid sample.
-A signed informed consent indicating parental permission with or without patient assent.
-For patients on apheresis, currently undergoing stable low-density lipoprotein (LDL) apheresis therapy prior to the screening and have initiated apheresis treatment for at least 6 months.
Are the trial subjects under 18? yes
Number of subjects for this age range: 50
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

-Patients with low-density lipoprotein - cholesterol (LDL-C) less than 130 mg/dL (3.37 mmol/L) obtained during the screening period after the patient has been on stable apheresis procedure or lipid modifying therapy (LMT) (i.e., stable optimal dose of statin ± other stable LMTs, or stable non statin LMTs in statin-intolerant patients) treatment for at least 4 weeks.
-Patients with body weight less than 25 kg.
-Patients aged 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 uncontrolled Type 1 or 2 diabetes mellitus.
-Patients with known uncontrolled thyroid disease.
-Patients with uncontrolled hypertension.
-Fasting triglycerides >350 mg/dL.
-Severe renal impairment (i.e., estimated glomerular filtration rate [eGFR] <30 mL/min/1.73m^2) at the screening visit.
-Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 x upper limit of normal (ULN).
-Creatine phosphokinase (CPK) >3 x ULN.

Study & Design

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