Hypercholesterolaemia MedDRA version: 19.1 Level: PT Classification code 10020603 Term: Hypercholesterolaemia System Organ Class: 10027433 - Metabolism and nutrition disorders
Conditions
Interventions
Product Name: alirocumab
Product Code: SAR236553
Pharmaceutical Form: Solution for injection
INN or Proposed INN: alirocumab
CAS Number:
Sponsors
sanofi-aventis recherche&developpement
Eligibility
Sex/Gender
All
Inclusion criteria
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
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] 2 x upper limit of normal (ULN). -Creatinine phosphokinase (CPK) >3 x ULN.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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. ;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 | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: a) From baseline to Week 8 b), c) : At week 8 d), e), f), g) h), I), l), m), n), o), p), q), r), s), t) : From baseline to Week 8 ; Secondary end point(s): a) Absolute change in calculated LDL-C b) Percentage of participants achieving a calculated LDL-C level lower than 130 mg/dL (3.37 mmol/L) c) Percentage of participants achieving a calculated LDL-C level lower than 110 mg/dL (2.84 mmol/L) d) Percent change in Apolipoprotein B (Apo B) e) Percent change in non-high density lipoprotein cholesterol (non HDL-C) f) Percent change in Total-C g) Percent change in Lipoprotein (a) (Lp[a]) h) Percent change in triglycerides (TG) i) Percent change in HDL-C l) Percent change in Apo A-1 m) Absolute change in Apo B n) Absolute change in non-HDL-C o) Absolute change in Total-C p) Absolute change in Lp(a) q) Absolute change in TG r) Absolute change in HDL-C s) Absolute change in Apo A-1 t) Absolute change in ratio Apo B/Apo A-1 | — |
Countries
Australia, Canada, Czech Republic, France, Israel, Netherlands, Norway, Russian Federation, South Africa, Spain, Sweden
Contacts
Public ContactCountry Team Manager Sweden
Sanofi AB
Outcome results
None listed