An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
| Status: | Recruiting |
|---|---|
| Conditions: | High Cholesterol |
| Therapuetic Areas: | Cardiology / Vascular Diseases |
| Healthy: | No |
| Age Range: | 8 - 17 |
| Updated: | 2/20/2019 |
| Start Date: | August 31, 2018 |
| End Date: | February 2020 |
| Contact: | Trial Transparency email recommended (Toll free number for US & Canada) |
| Email: | Contact-US@sanofi.com |
| Phone: | 800-633-1610 |
An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Primary Objective:
To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W), on low-density
lipoprotein cholesterol (LDL-C) levels of treatment in children with homozygous familial
hypercholesterolemia (hoFH) 8 to 17 years of age on top of background treatments.
Secondary Objectives:
- To evaluate the efficacy of alirocumab after treatment on LDL-C levels.
- To evaluate the effects of alirocumab on other lipid parameters.
- To evaluate the safety and tolerability of alirocumab.
To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W), on low-density
lipoprotein cholesterol (LDL-C) levels of treatment in children with homozygous familial
hypercholesterolemia (hoFH) 8 to 17 years of age on top of background treatments.
Secondary Objectives:
- To evaluate the efficacy of alirocumab after treatment on LDL-C levels.
- To evaluate the effects of alirocumab on other lipid parameters.
- To evaluate the safety and tolerability of alirocumab.
The study duration is up to 62 weeks, which includes (if needed) a run-in period of up to 4
weeks, a screening period of up to 2 weeks, a treatment period of up to 48 weeks, and a
follow-up of 8 weeks.
weeks, a screening period of up to 2 weeks, a treatment period of up to 48 weeks, and a
follow-up of 8 weeks.
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.
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 Stage1 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.
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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