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Atorvastatin Three Year Pediatric Study

Phase 3
Completed
Conditions
Familial Hypercholesterolemia
Interventions
Registration Number
NCT00827606
Lead Sponsor
Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Brief Summary

The purpose of this study is to characterize three year descriptive growth and development (ie, height, weight, body mass index, Tanner Stage) and efficacy of cholesterol reduction in pediatric subjects with Heterozygous Familial Hypercholesterolemia receiving atorvastatin treatment.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
272
Inclusion Criteria
  • Heterozygous familial hypercholesterolemia, ages 6-15, LDL greater than 4 mmol/l
Exclusion Criteria
  • Active liver disease or hepatic dysfunction, or persistent elevations of serum transaminases exceeding three times the upper limit of normal (ULN).

Female of childbearing potential who is not using adequate contraceptive measures or any female who is pregnant or breastfeeding. Any female who becomes pregnant during study participation will be immediately discontinued from treatment and counseled appropriately about the in utero exposure.

Known hypersensitivities to HMG-CoA reductase inhibitors

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
AtorvastatinatorvastatinAll subjects will be treated with atorvastatin
Primary Outcome Measures
NameTimeMethod
Apoliprotein B (Apo B; g/L) During the StudyBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

Age (Years) During the Study: FemalesBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of age during the study. Change from baseline was also determined.

High-Density Lipoprotein Cholesterol (HDL-C; mMol/L) During the StudyBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

Total Cholesterol (mMol/L) During the StudyBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

Percent Change From Baseline in TrigylceridesMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast).

Percent Change From Baseline in VLDLMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast).

Percent Change From Baseline in Height: MalesMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of height changes during the study.

Height (cm) During the Study: FemalesBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of height changes during the study. Change from baseline was also determined.

Body Mass Index (BMI in kg Per Square Meter [kg/m^2]) During the Study: MalesBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of BMI changes during the study. Change from baseline was also determined.

Percent Change From Baseline in BMI: MalesMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of BMI changes during the study.

Percent Change From Baseline in BMI: FemalesMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of BMI changes during the study.

Percent Change From Baseline in Age: FemalesMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of age during the study.

Flow-Mediated Dilatation (FMD) During the StudyBaseline, Months 6, 12, 18, 24, 30 and 36/ET

Percent (%) FMD was calculated as (hyperemic diameter minus resting diameter) divided by the resting diameter multiplied by 100. Change from baseline was also determined.

Trigylcerides (mMol/L) During the StudyBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

Percent Change From Baseline in Apo BMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast).

Number of Participants With Shift From Baseline in Tanner_Stage by Timepoint and Baseline Tanner_StageBaseline, Months 6, 12, 18, 24, 30, and 36/ET

Tanner_Stage was assessed based on 2 components by gender, pubic hair and breasts for females and pubic hair and genitalia for males. If these values of components were not same, then the Tanner_Stage had the higher value of 2 components for each gender by visit.

Weight (kg) During the Study: FemalesBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of weight changes during the study. Change from baseline was also determined.

Percent Change From Baseline in Weight: FemalesMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of weight changes during the study.

BMI (kg/m^2) During the Study: FemalesBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of BMI changes during the study. Change from baseline was also determined.

Age (Years) During the Study: MalesBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of age during the study. Change from baseline was also determined.

Percent Change From Baseline in HDL-CMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast).

Very Low-Density Lipoprotein (VLDL; mMol/L) During the StudyBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

Apoliprotein A-1 (Apo A-1; Grams Per Liter [g/L]) During the StudyBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

Percent Change From Baseline in Apo A-1Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast).

Height (Centimeters [cm]) During the Study: MalesBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of height changes during the study. Change from baseline was also determined.

Percent Change From Baseline in Height: FemalesMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of height changes during the study.

Weight (Kilograms [kg]) During the Study: MalesBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of weight changes during the study. Change from baseline was also determined.

Percent Change From Baseline in Weight: MalesMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of weight changes during the study.

Percent Change From Baseline in Age: MalesMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Investigator assessment of age during the study.

Percent Change From Baseline in FMDMonths 6, 12, 18, 24, 30 and 36/ET

Percent (%) FMD was calculated as (hyperemic diameter minus resting diameter) divided by the resting diameter multiplied by 100.

Low Density Lipoprotein Cholesterol (LDL-C; Millimoles Per Liter [mMol/L]) During the StudyBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination [ET])

Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

Percent Change From Baseline in LDL-CMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or ET)

Assessments were performed in the fasting state (minimum 10-hour fast).

Percent Change From Baseline in Total CholesterolMonths 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

Assessments were performed in the fasting state (minimum 10-hour fast).

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants With Overall Expected Maturation and Development Consistent With Expectations as Assessed by the InvestigatorBaseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination)
Percentage of Participants by Study Drug Compliance CategoryMonths 1, 2, 3, 6, 12, 18, 24, 30, and 36 (or early termination)

Compliance to study drug was categorized as \<80%, 80% - 120%, and greater than (\>) 120%.

Trial Locations

Locations (40)

Phoenix Children's Hospital

🇺🇸

Phoenix, Arizona, United States

Children's National Medical Center

🇺🇸

Washington, District of Columbia, United States

University of Florida

🇺🇸

Gainesville, Florida, United States

Johns Hopkins University

🇺🇸

Baltimore, Maryland, United States

Johns Hopkins

🇺🇸

Baltimore, Maryland, United States

MEDPACE Clinical Pharmacology Unit

🇺🇸

Cincinnati, Ohio, United States

The Children's Hospital of Philadelphia

🇺🇸

Philadelphia, Pennsylvania, United States

Cliniques Universitaires Saint-Luc / Pediatrie generale

🇧🇪

Bruxelles, Belgium

Universitaire Ziekenhuizen Leuven / Center for Metabolic Diseases

🇧🇪

Leuven, Belgium

Clinique des maladies lipidiques de Quebec Inc

🇨🇦

Sainte-Foy,, Quebec, Canada

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Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
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