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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03510715
• Date of registration: 18/04/2018
• Prospective Registration: Yes
• Primary sponsor: Sanofi
• Public title: An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
• Scientific title: An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
• Date of first enrolment: August 31, 2018
• Target sample size: 18
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03510715
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Austria, Brazil, Canada, Denmark, France, Italy, Mexico, Netherlands, Norway, Russian Federation, Slovenia, Spain, Taiwan, Turkey, United States

Contacts

• Name: Clinical Sciences & Operations
• Affiliation: Sanofi

Key inclusion & exclusion criteria

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.



Age minimum: 8 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Hypercholesterolemia

Intervention(s)

Drug: Cholestyramine

Drug: Rosuvastatin

Drug: Alirocumab SAR236553 (REGN727)

Drug: Fluvastatin

Drug: Atorvastatin

Drug: Lovastatin

Drug: Ezetimibe

Drug: Fenofibrate

Drug: Pravastatin

Drug: Nicotinic acid

Drug: Simvastatin

Drug: Omega-3 fatty acids

Primary Outcome(s)

Percent change in LDL-C [Time Frame: From baseline to Week 12]

Secondary Outcome(s)

Percent change in Lp(a) [Time Frame: From baseline to Week 12, to Week 24, and to Week 48]

Absolute change in LDL-C [Time Frame: From baseline to Weeks 12, 24 and 48]

Percent change in LDL-C [Time Frame: From baseline to Weeks 24 and 48]

Proportion of patients with =15% reduction in LDL-C [Time Frame: From baseline to Weeks 12, 24 and 48]

Number of patients with adverse events [Time Frame: Up to Week 62]

Percent change in Apo B [Time Frame: From baseline to Week 12, to Week 24, and to Week 48]

Percent change in non-HDL-C [Time Frame: From baseline to Week 12, to Week 24, and to Week 48]

Percent change in total-C [Time Frame: From baseline to Week 12, to Week 24, and to Week 48]

Percent change in fasting Triglycerides (TG) [Time Frame: From baseline to Week 12, to Week 24, and to Week 48]

Percent change in HDL-C [Time Frame: From baseline to Week 12, to Week 24, and to Week 48]

Percent change in LDL-C [Time Frame: From baseline to Week 12]

Percent change in Apo A-1 [Time Frame: From baseline to Week 12, to Week 24, and to Week 48]

Tanner stage [Time Frame: At Weeks 24, 24, and 48]

Secondary ID(s)

EFC14660

2017-002297-39

U1111-1200-2046

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Regeneron Pharmaceuticals