An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia
| Status: | Completed |
|---|---|
| Conditions: | High Cholesterol |
| Therapuetic Areas: | Cardiology / Vascular Diseases |
| Healthy: | No |
| Age Range: | 8 - 17 |
| Updated: | 3/16/2019 |
| Start Date: | September 15, 2016 |
| End Date: | February 22, 2019 |
An 8-Week Open-Label, Sequential, Repeated Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia Followed by an Extension Phase
Primary 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.
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.
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.
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.
For Cohorts 1 to 3, a study duration of approximately 16-23 weeks (screening period: up to 6
(+1) weeks, open-label dose finding treatment period: 8 weeks, follow up period: 6-8 weeks).
For Cohort 4, a study duration of approximately 14-19 weeks (screening period up to 6 [+1]
weeks, open-label dose finding treatment period: 12 weeks).
Optional extension period: up to a maximum of 2 years for the first patients enrolled in
Cohorts 1 to 3, but a maximum of approximately 5 months for the first patients enrolled in
Cohort 4.
For all patients who decline participation in the phase 3 study, their last alirocumab
injection will be on December 2018.
(+1) weeks, open-label dose finding treatment period: 8 weeks, follow up period: 6-8 weeks).
For Cohort 4, a study duration of approximately 14-19 weeks (screening period up to 6 [+1]
weeks, open-label dose finding treatment period: 12 weeks).
Optional extension period: up to a maximum of 2 years for the first patients enrolled in
Cohorts 1 to 3, but a maximum of approximately 5 months for the first patients enrolled in
Cohort 4.
For all patients who decline participation in the phase 3 study, their last alirocumab
injection will be on December 2018.
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.
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 m².
- 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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