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

Familial Hypercholesterolaemia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01078675
• Other study ID #: D3561C00002
• Status: Completed
• Phase: Phase 3
• First received: February 25, 2010
• Last updated: March 19, 2015
• Start date: February 2010
• Est. completion date: February 2013

Study information

• Verified date: March 2015
• Source: AstraZeneca
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 315
• Est. completion date: February 2013
• Est. primary completion date: February 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 17 Years

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Familial Hypercholesterolaemia
• Hypercholesterolemia
• Hyperlipoproteinemia Type II

Intervention

Drug:
• rosuvastatin calcium
5 mg, oral, once daily, 24 months
• rosuvastatin calcium
10 mg, oral, once daily, 24 months
• rosuvastatin calcium
20 mg, oral, once daily, 24 months

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
AstraZeneca

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  Netherlands,  Norway, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent Change From Baseline in LDL-C	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	No
Primary	Sexual Maturation by Tanner Staging 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	No
Primary	Single Dose PK - Cmax	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.	No
Primary	Percent Change From Baseline in Height	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	No
Primary	Sexual Maturation by Tanner Staging at Month 12	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	No
Primary	Sexual Maturation by Tanner Staging at Month 24	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	No
Primary	Single Dose PK - Tmax	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	No
Primary	Single Dose PK - AUC(0-24)	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	No
Secondary	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	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	No
Secondary	Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)	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	No
Secondary	Adverse Events	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	No
Secondary	Total Duration of Exposure	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	No
Secondary	Overal Treatment Adherence	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	No

External Links

•   D3561C00002 Study Report Synopsis