An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia
| Status: | Recruiting |
|---|---|
| Conditions: | High Cholesterol |
| Therapuetic Areas: | Cardiology / Vascular Diseases |
| Healthy: | No |
| Age Range: | 8 - 17 |
| Updated: | 3/27/2019 |
| Start Date: | May 31, 2018 |
| End Date: | March 2022 |
| Contact: | Trial Transparency email recommended (Toll free number for US & Canada) |
| Email: | Contact-Us@sanofi.com |
| Phone: | 800-633-1610 |
A Randomized, Double-Blind, Placebo-Controlled Study Followed by an Open Label Treatment Period to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia
Primary Objective:
To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W) and every 4 weeks
(Q4W) versus placebo after 24 weeks of double-blind (DB) treatment on low-density lipoprotein
cholesterol (LDL-C) levels in patients with heterozygous familial hypercholesterolemia (heFH)
8 to 17 years of age 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.
Secondary Objective:
- To evaluate the efficacy of alirocumab versus placebo on low-density lipoprotein
cholesterol (LDL-C) levels.
- To evaluate the effects of alirocumab versus placebo on other lipid parameters.
- To evaluate the safety and tolerability of alirocumab in comparison with placebo.
- To evaluate the efficacy, safety, and tolerability of alirocumab after open label
treatment.
- To evaluate the development of anti-alirocumab antibodies.
To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W) and every 4 weeks
(Q4W) versus placebo after 24 weeks of double-blind (DB) treatment on low-density lipoprotein
cholesterol (LDL-C) levels in patients with heterozygous familial hypercholesterolemia (heFH)
8 to 17 years of age 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.
Secondary Objective:
- To evaluate the efficacy of alirocumab versus placebo on low-density lipoprotein
cholesterol (LDL-C) levels.
- To evaluate the effects of alirocumab versus placebo on other lipid parameters.
- To evaluate the safety and tolerability of alirocumab in comparison with placebo.
- To evaluate the efficacy, safety, and tolerability of alirocumab after open label
treatment.
- To evaluate the development of anti-alirocumab antibodies.
The study duration is approximately up to 110 weeks (run-in period [if needed]: up to 4 weeks
[+2 days], screening period: up to 2 weeks [+5 days], double-blind treatment period: 24
weeks, open label treatment period: 80 weeks).
[+2 days], screening period: up to 2 weeks [+5 days], double-blind treatment period: 24
weeks, open label treatment period: 80 weeks).
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.
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.
The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial.
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