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

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Sanofi

Status and phase

Completed
Phase 3

Conditions

Hypercholesterolemia

Treatments

Drug: Atorvastatin
Drug: Ezetimibe
Drug: Simvastatin
Drug: Alirocumab SAR236553 (REGN727)
Drug: Omega-3 fatty acids
Drug: Lovastatin
Drug: Pravastatin
Drug: Rosuvastatin
Drug: Nicotinic acid
Drug: Fenofibrate
Drug: Cholestyramine
Drug: Fluvastatin

Study type

Interventional

Funder types

Industry

Identifiers

NCT03510715
2017-002297-39 (EudraCT Number)
U1111-1200-2046 (Other Identifier)
EFC14660

Details and patient eligibility

About

Primary Objective:

To evaluate the efficacy of alirocumab (75 or 150 milligrams [mg] depending on body weight [BW]), administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels at Week 12 of treatment in children and adolescents with homozygous familial hypercholesterolemia (hoFH) of 8 to 17 years of age on top of background treatments.

Secondary Objectives:

  • To evaluate the efficacy of alirocumab after 24 and 48 weeks of treatment on LDL-C levels.
  • To evaluate the effects of alirocumab on other lipid parameters (eg, apolipoprotein B [Apo B], non-high density lipoprotein cholesterol [non-HDL-C], total cholesterol [Total-C], high density lipoprotein cholesterol [HDL-C], lipoprotein a [Lp (a)], triglycerides [TG], apolipoprotein A-1 [Apo A-1] levels) after 12, 24, and 48 weeks of treatment.
  • To evaluate the safety and tolerability of alirocumab up to 48 weeks of treatment.

Full description

The study duration was up to 62 weeks, which included (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.

Enrollment

18 patients

Sex

All

Ages

8 to 17 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion criteria :

  • Participants genetically diagnosed with hoFH.
  • Participants 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.
  • A signed informed consent indicating parental permission with or without participants assent.
  • For participants on apheresis, currently undergoing stable LDL apheresis therapy prior to the screening visit (Week -2) and had initiated apheresis treatment for at least 6 months.

Exclusion criteria:

  • Participants with LDL-C <130 milligram per deciliter [mg/dL] (3.37 millimoles per liter [mmol/L]) obtained during the screening period after the participant had been on stable apheresis procedure or LMT (i.e., stable optimal dose of statin ± other stable LMTs, or stable non statin LMTs in statin-intolerant participants) treatment for at least 4 weeks.
  • Participants with BW <25 kg.
  • Participants aged 8 to 9 years not at Tanner Stage 1 and participants aged of 10 to 17 years not at least at Tanner Stage 2 in their development.
  • Participants with uncontrolled Type 1 or 2 diabetes mellitus.
  • Participants with known uncontrolled thyroid disease.
  • Participants with uncontrolled hypertension.
  • Participants who will receive statin de novo during the run-in period.
  • Fasting triglycerides greater than (>) 350 mg/dL (3.95 mmol/L) at the screening visit.
  • Severe renal impairment (i.e., estimated glomerular filtration rate <30 milliliter per minute/1.73 meter square) at the screening visit.
  • Alanine aminotransferase or aspartate aminotransferase >2 * upper limit of normal (ULN) at the screening visit.
  • Creatine phosphokinase >3 * ULN at the screening visit.

The above information was not intended to contain all considerations relevant to a participants potential participation in a clinical trial.

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

18 participants in 1 patient group

Alirocumab
Experimental group
Description:
Participants with BW less than (\<) 50 kilograms (kg) received subcutaneous (SC) injection of alirocumab 75 mg Q2W for 48 weeks. Alirocumab dose was up-titrated to 150 mg Q2W from Week 12 in case of increase in BW with BW greater than or equal to \[\>=\] 50 kg. Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
Treatment:
Drug: Lovastatin
Drug: Nicotinic acid
Drug: Omega-3 fatty acids
Drug: Fenofibrate
Drug: Ezetimibe
Drug: Fluvastatin
Drug: Simvastatin
Drug: Pravastatin
Drug: Rosuvastatin
Drug: Atorvastatin
Drug: Cholestyramine
Drug: Alirocumab SAR236553 (REGN727)

Trial documents
2

Trial contacts and locations

10

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Data sourced from clinicaltrials.gov

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