An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
AstraZeneca
ClinicalTrials.gov Identifier:
NCT01078675
First received: February 25, 2010
Last updated: June 25, 2013
Last verified: June 2013
  Purpose

This study is being carried out to see if the study medication, rosuvastatin, is effective in treating familial hypercholesterolaemia in children and adolescents, and to determine the long term (over 2 years) safety, tolerability and efficacy of the study medication in these patients.

This study will also measure levels of drug in the blood and see how well it is tolerated. This is known as pharmacokinetic (PK) analysis.

At baseline only a small number of patients will participate in a single dose PK phase over 24 hours.

In order to see if this medication works, a control group of healthy siblings will help the researchers to compare certain results.


Condition Intervention Phase
Familial Hypercholesterolaemia
Drug: rosuvastatin calcium
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Efficacy and 2-Year Safety Study of Open-label Rosuvastatin in Children and Adolescents (Aged From 6 to Less Than 18 Years) With Familial Hypercholesterolaemia

Resource links provided by NLM:


Further study details as provided by AstraZeneca:

Primary Outcome Measures:
  • Percent change from baseline in low-density lipoprotein cholesterol [ Time Frame: following 3 months, 12 months and 24 months of treatment ] [ Designated as safety issue: No ]
  • Assessments of growth by assessment of height (including linear growth [cm and standard deviation score]) and secondary characteristics of sexual maturation by Tanner staging [ Time Frame: at baseline, 12 months and 24 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Percent change from baseline (Visit 3, Week 0) in HDL-C, total cholesterol, triglycerides, non-HDL-C, LDL-C/HDL-C, total cholesterol/HDL-C, non-HDL-C/HDL-C, ApoB, ApoB/ApoA-1 and ApoA-1 [ Time Frame: at 3 months, 12 months and 24 months ] [ Designated as safety issue: No ]
  • Assessments of intima and media wall thickness of the carotid arteries by sonography in at least 180 enrolled patients in comparison to at least 60 enrolled healthy siblings [ Time Frame: at baseline, 12 months and 24 months ] [ Designated as safety issue: No ]
  • To assess adverse events [ Time Frame: Weeks -4, -1, 0, and Months 1, 3, 6, 9, 12, 15, 18, 21, and 24 ] [ Designated as safety issue: Yes ]

Enrollment: 198
Study Start Date: February 2010
Study Completion Date: February 2013
Primary Completion Date: February 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Drug: rosuvastatin calcium
5 mg, oral, once daily, 24 months
Other Name: Crestor
Drug: rosuvastatin calcium
10 mg, oral, once daily, 24 months
Other Name: Crestor
Drug: rosuvastatin calcium
20 mg, oral, once daily, 24 months
Other Name: Crestor

  Eligibility

Ages Eligible for Study:   6 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • children and adolescents (aged 6 to less than 18 years) with Familial Hypercholesterolaemia
  • Patients aged between 6 and less than 10 years of age must not be taking a statin medicine

Exclusion Criteria:

  • History of muscle or sensitivity reactions to any statin medicines
  • Current active liver disease or dysfunction (except a confirmed diagnosis of Gilbert's disease)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01078675

Locations
United States, Ohio
Research Site
Cincinnati, Ohio, United States
Belgium
Research Site
Leuven, Belgium
Canada, British Columbia
Research Site
Vancouver, British Columbia, Canada
Canada, Ontario
Research Site
Hamilton, Ontario, Canada
Research Site
Toronto, Ontario, Canada
Canada, Quebec
Research Site
Chicoutimi, Quebec, Canada
Canada
Research Site
Quebec, Canada
Netherlands
Research Site
Amsterdam, Netherlands
Research Site
Groningen, Netherlands
Research Site
Hoorn, Netherlands
Research Site
Leiderdorp, Netherlands
Research Site
Rotterdam, Netherlands
Research Site
Waalwijk, Netherlands
Norway
Research Site
Oslo, Norway
Sponsors and Collaborators
AstraZeneca
Investigators
Principal Investigator: John J.P. Kastelein, MD, PhD Chairman, Dept. of Vascular Medicine, Academic Medical Center, Meibergdreef 9
  More Information

No publications provided by AstraZeneca

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: AstraZeneca
ClinicalTrials.gov Identifier: NCT01078675     History of Changes
Other Study ID Numbers: D3561C00002
Study First Received: February 25, 2010
Last Updated: June 25, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by AstraZeneca:
Familial Hypercholesterolaemia
pediatric

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Calcium, Dietary
Rosuvastatin
Bone Density Conservation Agents
Physiological Effects of Drugs
Pharmacologic Actions
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Enzyme Inhibitors
Lipid Regulating Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on August 18, 2014