PLUTO: Pediatric Lipid-redUction Trial of rOsuvastatin

Study Overview
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Brief Summary
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The primary objective of this study is to determine the efficacy of once-daily rosuvastatin in reducing LDL-C in children and adolescents aged 10-17 years with HeFH from baseline (Day 0) to the end of the 12-week double-blind treatment period.

Official Title
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A Phase IIIb, Efficacy, and Safety Study of Rosuvastatin in Children 10-17 Years of Age With Heterozygous Familial Hypercholesterolemia: a 12-week, Double-blind, Randomized, Multicenter, Placebo-controlled Study With a 40-week, Open-label, Follow-up

Conditions
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Familial Hypercholesterolemia

Intervention / Treatment
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* Drug: Rosuvastatin
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female (at least 1 year post-menarche) children and adolescents (aged 10 -17 years) with heterozygous familial hypercholesterolemia (HeFH) Exclusion Criteria: Certain medical conditions and lab test results History of a reaction to rosuvastatin or other statin drugs Use of specified disallowed medications

Ages Eligible for Study
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Minimum Age: 10 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: rosuva 5<br>rosuvastatin 5 mg | Drug: Rosuvastatin<br>* oral<br>* Other names: Nexium;|
| Active Comparator: rosuva 10<br>rosuvastatin 10 mg | Drug: Rosuvastatin<br>* oral<br>* Other names: Nexium;|
| Active Comparator: rosuva 20<br>rosuvastatin 20 mg | Drug: Rosuvastatin<br>* oral<br>* Other names: Nexium;|
| Placebo Comparator: Placebo<br>Placebo | Drug: Placebo<br>* oral<br>|
| Other: rosuva ol<br>rosuvastatin open label | Drug: Rosuvastatin<br>* oral<br>* Other names: Nexium;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline (Day 0) to the End of the 12-week Double-blind Treatment Phase | Percent change in low-density lipoprotein cholesterol (LDL-C) = (final value - Baseline value)/Baseline value * 100 | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent Change in LDL-C and Other Lipid Parameters From Baseline to Week 6, and at End of Double-blind Dose Treatment Phase (Week 12) | Percent change from baseline in LDL-C after six week of treatment | 6 weeks | 
| Percent Control Rate Based on Achievement of LDL-C Target of <110 mg/dL During Double-blind Dose Treatment | Percent of patients achieving LDL-C < 110 mg/dL out of the total patients in each treatment group | 12 weeks | 
| Percent Change in HDL-C | Percent change in high-density lipoprotein cholesterol (HDL-C) after 12 weeks of treatment | After 12 weeks of treatment | 
| Percent Change in Non-HDL-C at 12 Weeks | Percent change in non-HDL-C at 12 weeks | After 12 weeks of treatment | 
| Percent Change in Triglycerides (TG) | Percent change in tryglycerides (TG) after 12 weeks of treatment | After 12 weeks of treatment | 
| Percent Change in Total Cholesterol (TC) | Percent change from baseline in total cholesteral after 12 weeks of treatment | After 12 weeks of treatment | 
| Percent Change in Apolipoprotein A-1 (ApoA-1) | Percent change in ApoA-1 after 12 weeks of treatment | After 12 weeks of treatment | 
| Percent Change in Apolipoprotein B (ApoB) | Percent change in ApoB after 12 weeks of treatment | After 12 weeks of treatment | 
| Percent Change in ApoB/ApoA-1 | Percent change in the ratio of ApoB/ApoA-1 after 12 weeks of treatment | After 12 weeks of treatment | 
| Percent Change in LDL-C/HDL-C | Percent change in the ratio of LDL-C/HDL-C after 12 weeks of treatment | After 12 week of treatment | 
| Percent Change in TC/HDL-C | Percent change in the ratio of TC/HDL-C after 12 weeks of treatment | After 12 weeks of treatment | 
| Percent Change in Non-HDL-C/HDL-C | Percent change in the ratio of non-HDL-C/HDL-C after 12 weeks of treatment | After 12 weeks of treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Heterozygous Familial Hypercholesterolemia