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Completed

NCT Number: NCT00827606

Atorvastatin Three Year Pediatric Study

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.

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Key information

Conditions

Familial Hypercholesterolemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

6 year–15 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Cliniques Universitaires Saint-Luc / Pediatrie generale, Brussels, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

atorvastatin

Drug

Atorvastatin tablets or chewable tablets, 5, 10, 20, 40 mg strengths, once daily, for three years (an 80 mg maximum daily dose is delivered by taking two 40 mg strengths, once daily)

Primary outcomes

  1. Low Density Lipoprotein Cholesterol (LDL-C; Millimoles Per Liter [mMol/L]) During the Study

    Time frame: Baseline, 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.

  2. Percent Change From Baseline in LDL-C

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or ET)

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

  3. High-Density Lipoprotein Cholesterol (HDL-C; mMol/L) During the Study

    Time frame: Baseline, 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.

  4. Percent Change From Baseline in HDL-C

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

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

  5. Total Cholesterol (mMol/L) During the Study

    Time frame: Baseline, 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.

  6. Percent Change From Baseline in Total Cholesterol

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

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

  7. Trigylcerides (mMol/L) During the Study

    Time frame: Baseline, 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.

  8. Percent Change From Baseline in Trigylcerides

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

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

  9. Very Low-Density Lipoprotein (VLDL; mMol/L) During the Study

    Time frame: Baseline, 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.

  10. Percent Change From Baseline in VLDL

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

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

  11. Apoliprotein A-1 (Apo A-1; Grams Per Liter [g/L]) During the Study

    Time frame: Baseline, 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.

  12. Percent Change From Baseline in Apo A-1

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

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

  13. Apoliprotein B (Apo B; g/L) During the Study

    Time frame: Baseline, 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.

  14. Percent Change From Baseline in Apo B

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

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

  15. Number of Participants With Shift From Baseline in Tanner_Stage by Timepoint and Baseline Tanner_Stage

    Time frame: Baseline, 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.

  16. Height (Centimeters [cm]) During the Study: Males

    Time frame: Baseline, 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.

  17. Percent Change From Baseline in Height: Males

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

    Investigator assessment of height changes during the study.

  18. Height (cm) During the Study: Females

    Time frame: Baseline, 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.

  19. Percent Change From Baseline in Height: Females

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

    Investigator assessment of height changes during the study.

  20. Weight (Kilograms [kg]) During the Study: Males

    Time frame: Baseline, 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.

  21. Percent Change From Baseline in Weight: Males

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

    Investigator assessment of weight changes during the study.

  22. Weight (kg) During the Study: Females

    Time frame: Baseline, 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.

  23. Percent Change From Baseline in Weight: Females

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

    Investigator assessment of weight changes during the study.

  24. Body Mass Index (BMI in kg Per Square Meter [kg/m^2]) During the Study: Males

    Time frame: Baseline, 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.

  25. Percent Change From Baseline in BMI: Males

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

    Investigator assessment of BMI changes during the study.

  26. BMI (kg/m^2) During the Study: Females

    Time frame: Baseline, 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.

  27. Percent Change From Baseline in BMI: Females

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

    Investigator assessment of BMI changes during the study.

  28. Age (Years) During the Study: Males

    Time frame: Baseline, 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.

  29. Percent Change From Baseline in Age: Males

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

    Investigator assessment of age during the study.

  30. Age (Years) During the Study: Females

    Time frame: Baseline, 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.

  31. Percent Change From Baseline in Age: Females

    Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

    Investigator assessment of age during the study.

  32. Flow-Mediated Dilatation (FMD) During the Study

    Time frame: Baseline, 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.

  33. Percent Change From Baseline in FMD

    Time frame: 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.

Secondary outcomes

  1. Percentage of Participants With Overall Expected Maturation and Development Consistent With Expectations as Assessed by the Investigator

    Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination)

  2. Percentage of Participants by Study Drug Compliance Category

    Time frame: Months 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%.

Sponsors and collaborators

Lead sponsor

Pfizer's Upjohn has merged with Mylan to form Viatris Inc.

Industry

Registry information

Official study title

A Three Year, Prospective, Open-label, Study To Evaluate Clinical Efficacy, Safety And Tolerability Of Atorvastatin In Children And Adolescents With Heterozygous Familial Hypercholesterolemia

Important dates

Study start
2009
Primary completion
2013
Study completion
2013
First posted
Jan 23, 2009
Registry last updated
Feb 21, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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