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

March 19, 2015 updated by: AstraZeneca

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

315

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Leuven, Belgium
        • Research Site
  • Canada
      • Quebec, Canada
        • Research Site
    • British Columbia
      • Vancouver, British Columbia, Canada
        • Research Site
    • Ontario
      • Hamilton, Ontario, Canada
        • Research Site
      • Toronto, Ontario, Canada
        • Research Site
    • Quebec
      • Chicoutimi, Quebec, Canada
        • Research Site
  • Netherlands
      • Amsterdam, Netherlands
        • Research Site
      • Groningen, Netherlands
        • Research Site
      • Hoorn, Netherlands
        • Research Site
      • Leiderdorp, Netherlands
        • Research Site
      • Rotterdam, Netherlands
        • Research Site
      • Waalwijk, Netherlands
        • Research Site
  • Norway
      • Oslo, Norway
        • Research Site
  • United States
    • Ohio
      • Cincinnati, Ohio, United States
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 17 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 1
Drug: rosuvastatin calcium
5 mg, oral, once daily, 24 months
Other Names:
  • Crestor
Drug: rosuvastatin calcium
10 mg, oral, once daily, 24 months
Other Names:
  • Crestor
Drug: rosuvastatin calcium
20 mg, oral, once daily, 24 months
Other Names:
  • Crestor

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percent Change From Baseline in LDL-C
Time Frame: At Month 3, Month 12 and Month 24
Negative values represent a decrease and positive values represent an increase. In total, 198 patients were treated. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
At Month 3, Month 12 and Month 24
Sexual Maturation by Tanner Staging at Baseline
Time Frame: At Baseline
Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.
At Baseline
Single Dose PK - Cmax
Time Frame: Serial blood samples over 24 hours.
Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing
Serial blood samples over 24 hours.
Percent Change From Baseline in Height
Time Frame: At Month 12 and Month 24
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
At Month 12 and Month 24
Sexual Maturation by Tanner Staging at Month 12
Time Frame: At Baseline
Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.
At Baseline
Sexual Maturation by Tanner Staging at Month 24
Time Frame: At Baseline
Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.
At Baseline
Single Dose PK - Tmax
Time Frame: Serial blood samples over 24 hours
Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing
Serial blood samples over 24 hours
Single Dose PK - AUC(0-24)
Time Frame: Serial blood samples over 24 hours
Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing
Serial blood samples over 24 hours

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
Time Frame: At Month 3, Month 12 and Month 24
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
At Month 3, Month 12 and Month 24
Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)
Time Frame: At Month 12 and Month 24
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
At Month 12 and Month 24
Adverse Events
Time Frame: 2-year study period
Number of participants with Various Categories of AE's. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
2-year study period
Total Duration of Exposure
Time Frame: 2-year study period
Total duration of exposure was calculated as [last dose date of rosuva - first dose date of rosuva + 1 day]. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
2-year study period
Overal Treatment Adherence
Time Frame: 2-year study period
Overall adherence rate was calculated as the weighted mean of adherence rates of all consecutive visits after baseline, in which the adherence rate between 2 consecutive visits was a percentage of the number of rosuvastatin taken divided by duration of exposure. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
2-year study period

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

AstraZeneca

Investigators

  • Principal Investigator: John J.P. Kastelein, MD, PhD, Chairman, Dept. of Vascular Medicine, Academic Medical Center, Meibergdreef 9

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2010

Primary Completion (Actual)

February 1, 2013

Study Completion (Actual)

February 1, 2013

Study Registration Dates

First Submitted

February 25, 2010

First Submitted That Met QC Criteria

March 1, 2010

First Posted (Estimate)

March 2, 2010

Study Record Updates

Last Update Posted (Estimate)

April 7, 2015

Last Update Submitted That Met QC Criteria

March 19, 2015

Last Verified

March 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • D3561C00002

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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