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NCT Number: NCT01078675

An Study to Evaluate Rosuvastatin in Children and Adolescents 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.

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Key information

Conditions

Familial Hypercholesterolaemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

6 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Research Site, Leuven, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

rosuvastatin calcium

Drug

5 mg, oral, once daily, 24 months

Other names: Crestor

Primary outcomes

  1. 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.

  2. 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.

  3. 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

  4. 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.

  5. 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.

  6. 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.

  7. 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

  8. 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

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

Sponsors and collaborators

Lead sponsor

AstraZeneca

Industry

Registry information

Official study 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

Important dates

Study start
2010
Primary completion
2013
Study completion
2013
First posted
Mar 2, 2010
Registry last updated
Apr 7, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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