---
title: An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
nct_id: NCT03510715
overall_status: COMPLETED
phase: PHASE3
sponsor: Sanofi
study_type: INTERVENTIONAL
primary_condition: Hypercholesterolemia
countries: Brazil, Canada, Denmark, Mexico, Netherlands, Russia, Slovenia, Spain, Taiwan, Turkey (Türkiye)
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT03510715.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT03510715"
ct_last_update_post_date: 2020-12-29
last_seen_at: "2026-05-12T06:47:09.785Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia

**Official Title:** An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia

**NCT ID:** [NCT03510715](https://clinicaltrials.gov/study/NCT03510715)

## Key Facts

- **Status:** COMPLETED
- **Phase:** PHASE3
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 18
- **Lead Sponsor:** Sanofi
- **Collaborators:** Regeneron Pharmaceuticals
- **Conditions:** Hypercholesterolemia
- **Start Date:** 2018-08-31
- **Completion Date:** 2020-02-17
- **CT.gov Last Update:** 2020-12-29

## Brief Summary

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

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.

## Eligibility

- **Minimum age:** 8 Years
- **Maximum age:** 17 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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.
```

## Arms

- **Alirocumab** (EXPERIMENTAL) — 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.

## Interventions

- **Alirocumab SAR236553 (REGN727)** (DRUG) — Pharmaceutical form: solution for injection in pre-filled syringe,

Route of administration: subcutaneous (SC)
- **Atorvastatin** (DRUG) — Pharmaceutical form: tablet,

Route of administration: oral
- **Simvastatin** (DRUG) — Pharmaceutical form: tablet,

Route of administration: oral
- **Fluvastatin** (DRUG) — Pharmaceutical form: capsule,

Route of administration: oral
- **Pravastatin** (DRUG) — Pharmaceutical form: tablet,

Route of administration: oral
- **Lovastatin** (DRUG) — Pharmaceutical form: tablet,

Route of administration: oral
- **Rosuvastatin** (DRUG) — Pharmaceutical form: tablet,

Route of administration: oral
- **Ezetimibe** (DRUG) — Pharmaceutical form: tablet,

Route of administration: oral
- **Cholestyramine** (DRUG) — Pharmaceutical form: oral suspension,

Route of administration: oral
- **Nicotinic acid** (DRUG) — Pharmaceutical form: tablet,

Route of administration: oral
- **Fenofibrate** (DRUG) — Pharmaceutical form: tablet,

Route of administration: oral
- **Omega-3 fatty acids** (DRUG) — Pharmaceutical form: capsule,

Route of administration: oral

## Primary Outcomes

- **Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis** _(time frame: Baseline to Week 12)_ — Adjusted least square (LS) means and standard errors were obtained from the mixed model analysis with repeated measures (MMRM) to account for missing data using all available post-baseline data from Week 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis).

## Secondary Outcomes

- **Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Week 12: On-treatment Analysis** _(time frame: Baseline to Week 12)_
- **Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis** _(time frame: Baseline to Weeks 24 and 48)_
- **Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis** _(time frame: Baseline to Weeks 12, 24 and 48)_
- **Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis** _(time frame: Baseline to Weeks 12, 24 and 48)_
- **Percent Change From Baseline in Total Cholesterol (Total-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis** _(time frame: Baseline to Weeks 12, 24 and 48)_
- **Percent Change From Baseline in Lipoprotein a (Lp) (a) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis** _(time frame: Baseline to Weeks 12, 24 and 48)_
- **Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis** _(time frame: Baseline to Weeks 12, 24 and 48)_
- **Percent Change From Baseline in Fasting Triglycerides (TG) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis** _(time frame: Baseline to Weeks 12, 24 and 48)_
- **Percent Change From Baseline in Apolipoprotein A1 (Apo A1) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis** _(time frame: Baseline to Weeks 12, 24 and 48)_
- **Percentage of Participants Reporting >=15 Percent (%) Reduction in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis** _(time frame: Baseline to Weeks 12, 24 and 48)_
- **Absolute Change From Baseline in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis** _(time frame: Baseline to Weeks 12, 24 and 48)_
- **Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48** _(time frame: Baseline, Weeks 12, 24 and 48)_

## Locations (10)

- Investigational Site Number 0760001, São Paulo, Brazil
- Investigational Site Number 1240001, Québec, Canada
- Investigational Site Number 2080001, Viborg, Denmark
- Investigational Site Number 4840006, Oaxaca City, Mexico
- Investigational Site Number 5280001, Amsterdam, Netherlands
- Investigational Site Number 6430002, Kemerovo, Russia
- Investigational Site Number 7050001, Ljubljana, Slovenia
- Investigational Site Number 7240001, A Coruña, Spain
- Investigational Site Number 1580001, Taipei, Taiwan
- Investigational Site Number 7920001, Izmir, Turkey (Türkiye)

## Recent Field Changes (last 30 days)

- `sponsor.collaborators` — added _(2026-05-12)_
- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.investigational site number 0760001|são paulo||brazil` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `locations.investigational site number 1240001|québec||canada` — added _(2026-05-12)_
- `locations.investigational site number 2080001|viborg||denmark` — added _(2026-05-12)_
- `locations.investigational site number 4840006|oaxaca city||mexico` — added _(2026-05-12)_
- `locations.investigational site number 5280001|amsterdam||netherlands` — added _(2026-05-12)_
- `locations.investigational site number 6430002|kemerovo||russia` — added _(2026-05-12)_
- `locations.investigational site number 7050001|ljubljana||slovenia` — added _(2026-05-12)_
- `locations.investigational site number 7240001|a coruña||spain` — added _(2026-05-12)_
- `locations.investigational site number 1580001|taipei||taiwan` — added _(2026-05-12)_
- `locations.investigational site number 7920001|izmir||turkey (türkiye)` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT03510715.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT03510715*  
*This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*