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

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ClinicalTrials.gov Identifier: NCT01078675
Recruitment Status : Completed
First Posted : March 2, 2010
Results First Posted : February 2, 2015
Last Update Posted : April 7, 2015
Sponsor:
Information provided by (Responsible Party):
AstraZeneca

Study Description
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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.


Condition or disease Intervention/treatment Phase
Familial Hypercholesterolaemia Drug: rosuvastatin calcium Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 315 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : February 2010
Actual Primary Completion Date : February 2013
Actual Study Completion Date : February 2013

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 1 Drug: rosuvastatin calcium
5 mg, oral, once daily, 24 months
Other Name: Crestor

Drug: rosuvastatin calcium
10 mg, oral, once daily, 24 months
Other Name: Crestor

Drug: rosuvastatin calcium
20 mg, oral, once daily, 24 months
Other Name: Crestor


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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.


Eligibility Criteria
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Ages Eligible for Study:   6 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01078675


Locations
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United States, Ohio
Research Site
Cincinnati, Ohio, United States
Belgium
Research Site
Leuven, Belgium
Canada, British Columbia
Research Site
Vancouver, British Columbia, Canada
Canada, Ontario
Research Site
Hamilton, Ontario, Canada
Research Site
Toronto, Ontario, Canada
Canada, Quebec
Research Site
Chicoutimi, Quebec, Canada
Canada
Research Site
Quebec, Canada
Netherlands
Research Site
Amsterdam, Netherlands
Research Site
Groningen, Netherlands
Research Site
Hoorn, Netherlands
Research Site
Leiderdorp, Netherlands
Research Site
Rotterdam, Netherlands
Research Site
Waalwijk, Netherlands
Norway
Research Site
Oslo, Norway
Sponsors and Collaborators
AstraZeneca
Investigators
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Principal Investigator: John J.P. Kastelein, MD, PhD Chairman, Dept. of Vascular Medicine, Academic Medical Center, Meibergdreef 9
More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: AstraZeneca
ClinicalTrials.gov Identifier: NCT01078675    
Other Study ID Numbers: D3561C00002
First Posted: March 2, 2010    Key Record Dates
Results First Posted: February 2, 2015
Last Update Posted: April 7, 2015
Last Verified: March 2015
Keywords provided by AstraZeneca:
Familial Hypercholesterolaemia
pediatric
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Calcium, Dietary
Rosuvastatin Calcium
 Calcium
Calcium-Regulating Hormones and Agents
Physiological Effects of Drugs
Bone Density Conservation Agents
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors