An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia
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
1 other identifier
interventional
315
5 countries
15
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Feb 2010
Typical duration for phase_3
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2010
CompletedFirst Submitted
Initial submission to the registry
February 25, 2010
CompletedFirst Posted
Study publicly available on registry
March 2, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2013
CompletedStudy Completion
Last participant's last visit for all outcomes
February 1, 2013
CompletedResults Posted
Study results publicly available
February 2, 2015
CompletedApril 7, 2015
March 1, 2015
3 years
February 25, 2010
February 6, 2014
March 19, 2015
Conditions
Keywords
Outcome Measures
Primary Outcomes (8)
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
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
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.
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
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
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
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
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
Secondary Outcomes (5)
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
Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)
At Month 12 and Month 24
Adverse Events
2-year study period
Total Duration of Exposure
2-year study period
Overal Treatment Adherence
2-year study period
Study Arms (1)
1
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- History of muscle or sensitivity reactions to any statin medicines
- Current active liver disease or dysfunction (except a confirmed diagnosis of Gilbert's disease)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AstraZenecalead
Study Sites (15)
Research Site
Cincinnati, Ohio, United States
Research Site
Leuven, Belgium, Belgium
Research Site
Leuven, Belgium
Research Site
Vancouver, British Columbia, Canada
Research Site
Hamilton, Ontario, Canada
Research Site
Toronto, Ontario, Canada
Research Site
Chicoutimi, Quebec, Canada
Research Site
Québec, Quebec, Canada
Research Site
Amsterdam, Netherlands
Research Site
Groningen, Netherlands
Research Site
Hoorn, Netherlands
Research Site
Leiderdorp, Netherlands
Research Site
Rotterdam, Netherlands
Research Site
Waalwijk, Netherlands
Research Site
Oslo, Norway
Related Publications (3)
Braamskamp MJAM, Langslet G, McCrindle BW, Cassiman D, Francis GA, Gagne C, Gaudet D, Morrison KM, Wiegman A, Turner T, Miller E, Kusters DM, Raichlen JS, Martin PD, Stein EA, Kastelein JJP, Hutten BA. Effect of Rosuvastatin on Carotid Intima-Media Thickness in Children With Heterozygous Familial Hypercholesterolemia: The CHARON Study (Hypercholesterolemia in Children and Adolescents Taking Rosuvastatin Open Label). Circulation. 2017 Jul 25;136(4):359-366. doi: 10.1161/CIRCULATIONAHA.116.025158. Epub 2017 Jun 7.
PMID: 28592434DERIVEDKusters DM, Wiegman A, Kastelein JJ, Hutten BA. Carotid intima-media thickness in children with familial hypercholesterolemia. Circ Res. 2014 Jan 17;114(2):307-10. doi: 10.1161/CIRCRESAHA.114.301430. Epub 2013 Nov 5.
PMID: 24192652DERIVEDTolani S, Pagler TA, Murphy AJ, Bochem AE, Abramowicz S, Welch C, Nagareddy PR, Holleran S, Hovingh GK, Kuivenhoven JA, Tall AR. Hypercholesterolemia and reduced HDL-C promote hematopoietic stem cell proliferation and monocytosis: studies in mice and FH children. Atherosclerosis. 2013 Jul;229(1):79-85. doi: 10.1016/j.atherosclerosis.2013.03.031. Epub 2013 Apr 19.
PMID: 23684512DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Niklas Berglind, GPS
- Organization
- AstraZeneca, R&D, B&I
Study Officials
- PRINCIPAL INVESTIGATOR
John J.P. Kastelein, MD, PhD
Chairman, Dept. of Vascular Medicine, Academic Medical Center, Meibergdreef 9
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 25, 2010
First Posted
March 2, 2010
Study Start
February 1, 2010
Primary Completion
February 1, 2013
Study Completion
February 1, 2013
Last Updated
April 7, 2015
Results First Posted
February 2, 2015
Record last verified: 2015-03