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

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ClinicalTrials.gov Identifier: NCT03510715
Recruitment Status : Active, not recruiting
First Posted : April 27, 2018
Last Update Posted : June 6, 2019
Sponsor:
Collaborator:
Regeneron Pharmaceuticals
Information provided by (Responsible Party):
Sanofi

Brief Summary:

Primary Objective:

To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels of treatment in children with homozygous familial hypercholesterolemia (hoFH) 8 to 17 years of age on top of background treatments.

Secondary Objectives:

  • To evaluate the efficacy of alirocumab after treatment on LDL-C levels.
  • To evaluate the effects of alirocumab on other lipid parameters.
  • To evaluate the safety and tolerability of alirocumab.

Condition or disease Intervention/treatment Phase
Hypercholesterolemia Drug: Alirocumab SAR236553 (REGN727) Drug: Rosuvastatin Drug: Ezetimibe Drug: Cholestyramine Drug: Nicotinic acid Drug: Fenofibrate Drug: Omega-3 fatty acids Drug: Atorvastatin Drug: Simvastatin Drug: Fluvastatin Drug: Pravastatin Drug: Lovastatin Phase 3

Detailed Description:
The study duration is up to 62 weeks, which includes (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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Actual Study Start Date : August 31, 2018
Estimated Primary Completion Date : August 2019
Estimated Study Completion Date : February 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Alirocumab

Arm Intervention/treatment
Experimental: Alirocumab
All patients will receive subcutaneously (SC) (one of 2 doses, depending on body weight) alirocumab Q2W at entry on top of background treatments.
Drug: Alirocumab SAR236553 (REGN727)
Pharmaceutical form:solution for injection Route of administration: subcutaneous

Drug: Rosuvastatin
Pharmaceutical form:tablet Route of administration: oral

Drug: Ezetimibe
Pharmaceutical form:tablet Route of administration: oral

Drug: Cholestyramine
Pharmaceutical form:oral suspension Route of administration: oral

Drug: Nicotinic acid
Pharmaceutical form:tablet Route of administration: oral

Drug: Fenofibrate
Pharmaceutical form:tablet Route of administration: oral

Drug: Omega-3 fatty acids
Pharmaceutical form:capsule Route of administration: oral

Drug: Atorvastatin
Pharmaceutical form:tablet Route of administration: oral

Drug: Simvastatin
Pharmaceutical form:tablet Route of administration: oral

Drug: Fluvastatin
Pharmaceutical form:capsule Route of administration: oral

Drug: Pravastatin
Pharmaceutical form:tablet Route of administration: oral

Drug: Lovastatin
Pharmaceutical form:tablet Route of administration: oral




Primary Outcome Measures :
  1. Percent change in LDL-C [ Time Frame: From baseline to Week 12 ]
    Percent change in LDL-C (pre-apheresis, if applicable) from baseline to Week 12, using all LDL-C values (pre-apheresis, if applicable) regardless of adherence to treatment.


Secondary Outcome Measures :
  1. Percent change in LDL-C [ Time Frame: From baseline to Week 12 ]
    Percent change in LDL-C (pre-apheresis, if applicable) from baseline to Week 12, using all LDL-C values during the treatment period.

  2. Percent change in LDL-C [ Time Frame: From baseline to Weeks 24 and 48 ]
    Percent change in LDL-C (pre-apheresis, if applicable) from baseline to Week 24 and to Week 48.

  3. Percent change in Apo B [ Time Frame: From baseline to Week 12, to Week 24, and to Week 48 ]
    Percent change in apolipoprotein B (Apo B) (pre-apheresis, if applicable) from baseline to Week 12, to Week 24, and to Week 48.

  4. Percent change in non-HDL-C [ Time Frame: From baseline to Week 12, to Week 24, and to Week 48 ]
    Percent change in non-high density lipoprotein cholesterol (non-HDL-C) (pre-apheresis, if applicable) from baseline to Week 12, to Week 24, and to Week 48.

  5. Percent change in total-C [ Time Frame: From baseline to Week 12, to Week 24, and to Week 48 ]
    Percent change in total cholesterol (pre-apheresis, if applicable) from baseline to Week 12, to Week 24, and to Week 48.

  6. Percent change in Lp(a) [ Time Frame: From baseline to Week 12, to Week 24, and to Week 48 ]
    Percent change in lipoprotein (a) (pre-apheresis, if applicable) from baseline to Week 12, to Week 24, and to Week 48.

  7. Percent change in HDL-C [ Time Frame: From baseline to Week 12, to Week 24, and to Week 48 ]
    Percent change in high-density lipoprotein cholesterol (HDL-C) (pre-apheresis, if applicable) from baseline to Week 12, to Week 24, and to Week 48.

  8. Percent change in fasting Triglycerides (TG) [ Time Frame: From baseline to Week 12, to Week 24, and to Week 48 ]
    Percent change in fasting triglycerides (TG) (pre-apheresis, if applicable) from baseline to Week 12, to Week 24, and to Week 48.

  9. Percent change in Apo A-1 [ Time Frame: From baseline to Week 12, to Week 24, and to Week 48 ]
    Percent change in apolipoprotein A1 (Apo A-1) (pre-apheresis, if applicable) from baseline to Week 12, to Week 24, and to Week 48.

  10. Proportion of patients with ≥15% reduction in LDL-C [ Time Frame: From baseline to Weeks 12, 24 and 48 ]
    Proportion of patients with ≥15% reduction in LDL-C (pre-apheresis, if applicable) at Weeks 12, 24, and 48.

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

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

  13. Tanner stage [ Time Frame: At Weeks 24, 24, and 48 ]
    The Tanner stage will be measured to assess stages of pubertal development



Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03510715


Locations
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Brazil
Investigational Site Number 0760001
Sao Paulo, Brazil, 05403-000
Canada
Investigational Site Number 1240001
Quebec, Canada, G1V 4W2
Denmark
Investigational Site Number 2080001
Viborg, Denmark, 8800
Mexico
Investigational Site Number 4840006
Oaxaca, Mexico, 68000
Netherlands
Investigational Site Number 5280001
Amsterdam, Netherlands, 1105AZ
Russian Federation
Investigational Site Number 6430002
Kemerovo, Russian Federation, 650002
Slovenia
Investigational Site Number 7050001
Ljubljana, Slovenia, 1000
Spain
Investigational Site Number 7240001
A Coruna, Spain, 15001
Taiwan
Investigational Site Number 1580001
Taipei, Taiwan, 112
Turkey
Investigational Site Number 7920001
Izmir, Turkey, 35040
Sponsors and Collaborators
Sanofi
Regeneron Pharmaceuticals
Investigators
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Study Director: Clinical Sciences & Operations Sanofi

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Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT03510715     History of Changes
Other Study ID Numbers: EFC14660
2017-002297-39 ( EudraCT Number )
U1111-1200-2046 ( Other Identifier: UTN )
First Posted: April 27, 2018    Key Record Dates
Last Update Posted: June 6, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Cholestyramine Resin
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Atorvastatin
Rosuvastatin Calcium
Simvastatin
Ezetimibe
Pravastatin
Lovastatin
Fenofibrate
Niacin
Antibodies, Monoclonal
Nicotinic Acids
Niacinamide
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors
Immunologic Factors