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

NCT ID: NCT03510715

Last Updated: 2020-12-29

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-08-31

Study Completion Date

2020-02-17

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Hypercholesterolemia

Keywords

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Pediatrics, Homozygous Familial Hypercholesterolemia, Alirocumab, PCSK9 inhibitor, Low-density Lipoprotein Cholesterol (LDL-C)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Alirocumab

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.

Group Type EXPERIMENTAL

Alirocumab SAR236553 (REGN727)

Intervention Type DRUG

Pharmaceutical form: solution for injection in pre-filled syringe,

Route of administration: subcutaneous (SC)

Atorvastatin

Intervention Type DRUG

Pharmaceutical form: tablet,

Route of administration: oral

Simvastatin

Intervention Type DRUG

Pharmaceutical form: tablet,

Route of administration: oral

Fluvastatin

Intervention Type DRUG

Pharmaceutical form: capsule,

Route of administration: oral

Pravastatin

Intervention Type DRUG

Pharmaceutical form: tablet,

Route of administration: oral

Lovastatin

Intervention Type DRUG

Pharmaceutical form: tablet,

Route of administration: oral

Rosuvastatin

Intervention Type DRUG

Pharmaceutical form: tablet,

Route of administration: oral

Ezetimibe

Intervention Type DRUG

Pharmaceutical form: tablet,

Route of administration: oral

Cholestyramine

Intervention Type DRUG

Pharmaceutical form: oral suspension,

Route of administration: oral

Nicotinic acid

Intervention Type DRUG

Pharmaceutical form: tablet,

Route of administration: oral

Fenofibrate

Intervention Type DRUG

Pharmaceutical form: tablet,

Route of administration: oral

Omega-3 fatty acids

Intervention Type DRUG

Pharmaceutical form: capsule,

Route of administration: oral

Interventions

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Alirocumab SAR236553 (REGN727)

Pharmaceutical form: solution for injection in pre-filled syringe,

Route of administration: subcutaneous (SC)

Intervention Type DRUG

Atorvastatin

Pharmaceutical form: tablet,

Route of administration: oral

Intervention Type DRUG

Simvastatin

Pharmaceutical form: tablet,

Route of administration: oral

Intervention Type DRUG

Fluvastatin

Pharmaceutical form: capsule,

Route of administration: oral

Intervention Type DRUG

Pravastatin

Pharmaceutical form: tablet,

Route of administration: oral

Intervention Type DRUG

Lovastatin

Pharmaceutical form: tablet,

Route of administration: oral

Intervention Type DRUG

Rosuvastatin

Pharmaceutical form: tablet,

Route of administration: oral

Intervention Type DRUG

Ezetimibe

Pharmaceutical form: tablet,

Route of administration: oral

Intervention Type DRUG

Cholestyramine

Pharmaceutical form: oral suspension,

Route of administration: oral

Intervention Type DRUG

Nicotinic acid

Pharmaceutical form: tablet,

Route of administration: oral

Intervention Type DRUG

Fenofibrate

Pharmaceutical form: tablet,

Route of administration: oral

Intervention Type DRUG

Omega-3 fatty acids

Pharmaceutical form: capsule,

Route of administration: oral

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

8 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role collaborator

Sanofi

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

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Investigational Site Number 0760001

São Paulo, , Brazil

Site Status

Investigational Site Number 1240001

Québec, , Canada

Site Status

Investigational Site Number 2080001

Viborg, , Denmark

Site Status

Investigational Site Number 4840006

Oaxaca City, , Mexico

Site Status

Investigational Site Number 5280001

Amsterdam, , Netherlands

Site Status

Investigational Site Number 6430002

Kemerovo, , Russia

Site Status

Investigational Site Number 7050001

Ljubljana, , Slovenia

Site Status

Investigational Site Number 7240001

A Coruña, , Spain

Site Status

Investigational Site Number 1580001

Taipei, , Taiwan

Site Status

Investigational Site Number 7920001

Izmir, , Turkey (Türkiye)

Site Status

Countries

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Austria France Italy Norway United States Brazil Canada Denmark Mexico Netherlands Russia Slovenia Spain Taiwan Turkey (Türkiye)

References

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Bruckert E, Caprio S, Wiegman A, Charng MJ, Zarate-Morales CA, Baccara-Dinet MT, Manvelian G, Ourliac A, Scemama M, Daniels SR. Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia: Phase 3, Multinational Open-Label Study. Arterioscler Thromb Vasc Biol. 2022 Dec;42(12):1447-1457. doi: 10.1161/ATVBAHA.122.317793. Epub 2022 Nov 3.

Reference Type DERIVED
PMID: 36325897 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2017-002297-39

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1200-2046

Identifier Type: OTHER

Identifier Source: secondary_id

EFC14660

Identifier Type: -

Identifier Source: org_study_id