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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief Summary:

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.


Detailed Summary:
Sponsor: AstraZeneca

Current Primary Outcome:

  • 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.
  • 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.
  • 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
  • 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.
  • 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

    Original Primary Outcome:

    • Percent change from baseline in low-density lipoprotein cholesterol [ Time Frame: following 3 months, 12 months and 24 months of treatment ]
    • 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 ]


    Current Secondary Outcome:

    • 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.
    • 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.
    • 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.
    • 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.
    • 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.


    Original Secondary Outcome:

    • 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 ]
    • 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 ]
    • To assess adverse events [ Time Frame: Weeks -4, -1, 0, and Months 1, 3, 6, 9, 12, 15, 18, 21, and 24 ]


    Information By: AstraZeneca

    Dates:
    Date Received: February 25, 2010
    Date Started: February 2010
    Date Completion:
    Last Updated: March 19, 2015
    Last Verified: March 2015