An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

Conditions

• Hypercholesterolaemia

Eligibility Criteria

Sex

ALL

Age

8 Years - 17 Years

Healthy Volunteers

Not accepted

Interventions

• alirocumab SAR236553 (REGN727) — DRUG
  Pharmaceutical form: solution Route of administration: subcutaneous injection
• statins — DRUG
  Pharmaceutical form: tablet Route of administration: oral
• ezetimibe — DRUG
  Pharmaceutical form:tablet Route of administration: oral
• cholestyramine — DRUG
  Pharmaceutical form:tablet Route of administration: oral
• fenofibrate — DRUG
  Pharmaceutical form: tablet Route of administration: oral
• omega-3 fatty acids — DRUG
  Pharmaceutical form: tablet Route of administration: oral
• nicotinic acid — DRUG
  Pharmaceutical form: tablet Route of administration: oral

Study Details

Primary Objective: To evaluate the effect of alirocumab on low-density lipoprotein cholesterol (LDL-C) levels after 8 weeks of treatment in heterozygous familial hypercholesterolemia (heFH) participants aged of 8 to 17 years, with LDL-C \>=130 milligrams per deciliter (mg/dL) (3.37 millimoles per litre \[mmol/L\]) on optimal stable daily dose of statin therapy +/- other lipid modifying therapies (LMTs) or a stable dose of non-statin LMTs in case of intolerance to statins for at least 4 weeks prior to the screening period. Secondary Objective: * To evaluate the safety and tolerability of alirocumab. * To evaluate the pharmacokinetics profile of alirocumab. * To evaluate the effects of alirocumab on other lipid parameters.

Key Dates

Start date

Sep 15, 2016

Status verified

Aug 2019

Primary completion

Sep 13, 2018

Completion

Feb 22, 2019

Study Design

Enrollment

42 participants (actual)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1 - Alirocumab 30 mg Q2W: <50 kg
  Period 1: Participants with body weight less than (\<) 50 kilograms (kg) received subcutaneous (SC) injection of alirocumab 30 milligram(mg) administered every 2 weeks (Q2W) up to 8 weeks added to lipid modifying therapy (LMT). Period 2: Participants with body weight \< 50 kg received SC injection of alirocumab 30 mg administered Q2W from Week 16 until they started receiving dose matching to Cohort 2 dosage including dose adjustment to body weight as required. Cohort 2 dosage was: if body weight was still \< 50 kg, participants received SC injection of alirocumab 40 mg administered Q2W until Week 130; if body weight was \> = 50 kg participants received SC injection of alirocumab 75 mg administered Q2W until Week 130.
• Experimental: Cohort 1 - Alirocumab 50 mg Q2W: >=50 kg
  Period 1: Participants with body weight greater than or equal to (\>=) 50 kg received SC injection of alirocumab 50 mg administered Q2W up to 8 weeks added to LMT. Period 2: Participants with body weight \>= 50 kg received SC injection of alirocumab 50 mg administered Q2W from Week 16 until switch to Cohort 2 dosage then SC injection of alirocumab 75 mg administered Q2W until Week 130.
• Experimental: Cohort 2 - Alirocumab 40 mg Q2W: <50 kg
  Period 1: Participants with body weight \< 50 kg received SC injection of alirocumab 40 mg administered Q2W up to 8 weeks added to LMT. Period 2: Participants with body weight \< 50 kg received SC injection of alirocumab 40 mg administered Q2W from Week 16 until switch of dosage in Cohorts 1 and 3. If body weight was still \< 50 kg, participants continued to receive SC injection of alirocumab 40 mg administered Q2W until Week 130; if body weight was \> = 50 kg participants received SC injection of alirocumab 75 mg administered Q2W until Week 130.
• Experimental: Cohort 2 - Alirocumab 75 mg Q2W: >=50 kg
  Period 1: Participants with body weight \>= 50 kg received SC injection of alirocumab 75 mg administered Q2W up to 8 weeks added to LMT. Period 2: Participants with body weight \>= 50 kg received SC injection of alirocumab 75 mg administered Q2W from Week 16 until Week 130.
• Experimental: Cohort 3 - Alirocumab 75 mg Q4W: <50 kg
  Period 1: Participants with body weight \< 50 kg received SC injection of alirocumab 75 mg administered every 4 weeks (Q4W) up to 8 weeks added to LMT. Period 2: Participants with body weight \< 50 kg received SC injection of alirocumab 75 mg administered Q4W from Week 14 until switch to Cohort 2 dosage including dose adjustment to body weight as required, then Cohort 2 dosage: if body weight was still \< 50 kg, participants received SC injection of alirocumab 40 mg administered Q2W until Week 130; if body weight was \> = 50 kg participants received SC injection of alirocumab 75 mg administered Q2W until Week 130.
• Experimental: Cohort 3 - Alirocumab 150 mg Q4W: >=50 kg
  Period 1: Participants with body weight \>= 50 kg received SC injection of alirocumab 150 mg administered Q4W up to Week 8 added to LMT. Period 2: Participants with body weight \>= 50 kg received SC injection of alirocumab 150 mg administered Q4W from Week 14 until switch to Cohort 2 dosage then SC injection of alirocumab 75 mg administered Q2W until Week 130.
• Experimental: Cohort 4 - Alirocumab 150 mg Q4W: <50 kg
  Period 1: Participants with body weight \< 50 kg received SC injection of alirocumab 150 mg administered Q4W up to 12 weeks added to LMT. Period 2: Participants with body weight \< 50 kg received SC injection of Alirocumab 150 mg administered Q4W from Week 12 until Week 48.
• Experimental: Cohort 4 - Alirocumab 300 mg Q4W: >=50 kg
  Period 1: Participants with body weight \>= 50 kg received SC injection of alirocumab 300 mg administered Q4W up to 12 weeks added to LMT. Period 2: Participants with body weight \>= 50 kg received SC injection of alirocumab 300 mg administered Q4W from Week 12 until Week 48.

Primary Outcome Measure

Percent Change From Baseline in Calculated Low Density Lipoprotein Cholesterol (LDL-C) at Week 8 [ Time Frame: Baseline, Week 8 ]

Locations (3)

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