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

Phase 3

Completed

Conditions: Familial Hypercholesterolaemia

Interventions: Drug: rosuvastatin calcium

Registration Number: NCT01078675

Lead Sponsor: AstraZeneca

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 Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 315

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 & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	rosuvastatin calcium	-

Primary Outcome Measures

Name	Time	Method
Single Dose PK - Cmax	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	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.
Percent Change From Baseline in LDL-C	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	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.
Sexual Maturation by Tanner Staging at Month 12	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 - Tmax	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
Single Dose PK - AUC(0-24)	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
Sexual Maturation by Tanner Staging at Month 24	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.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)	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	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.
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	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.
Overal Treatment Adherence	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.
Total Duration of Exposure	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.