Atorvastatin Three Year Pediatric Study

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00827606
First received: January 21, 2009
Last updated: June 1, 2015
Last verified: June 2015
  Purpose

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.


Condition Intervention Phase
Familial Hypercholesterolemia
Drug: atorvastatin
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • 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]) ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

  • Percent Change From Baseline in LDL-C [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or ET) ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast).

  • 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 ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

  • Percent Change From Baseline in HDL-C [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast).

  • Total Cholesterol (mMol/L) During the Study [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

  • Percent Change From Baseline in Total Cholesterol [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast).

  • Trigylcerides (mMol/L) During the Study [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

  • Percent Change From Baseline in Trigylcerides [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast).

  • 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 ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

  • Percent Change From Baseline in VLDL [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast).

  • 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 ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

  • Percent Change From Baseline in Apo A-1 [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast).

  • Apoliprotein B (Apo B; g/L) During the Study [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.

  • Percent Change From Baseline in Apo B [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    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_Stage [ Time Frame: Baseline, Months 6, 12, 18, 24, 30, and 36/ET ] [ Designated as safety issue: No ]
    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.

  • Height (Centimeters [cm]) During the Study: Males [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of height changes during the study. Change from baseline was also determined.

  • Percent Change From Baseline in Height: Males [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of height changes during the study.

  • Height (cm) During the Study: Females [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of height changes during the study. Change from baseline was also determined.

  • Percent Change From Baseline in Height: Females [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of height changes during the study.

  • Weight (Kilograms [kg]) During the Study: Males [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of weight changes during the study. Change from baseline was also determined.

  • Percent Change From Baseline in Weight: Males [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of weight changes during the study.

  • Weight (kg) During the Study: Females [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of weight changes during the study. Change from baseline was also determined.

  • Percent Change From Baseline in Weight: Females [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of weight changes during the study.

  • 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 ] [ Designated as safety issue: No ]
    Investigator assessment of BMI changes during the study. Change from baseline was also determined.

  • Percent Change From Baseline in BMI: Males [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of BMI changes during the study.

  • BMI (kg/m^2) During the Study: Females [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of BMI changes during the study. Change from baseline was also determined.

  • Percent Change From Baseline in BMI: Females [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of BMI changes during the study.

  • Age (Years) During the Study: Males [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of age during the study. Change from baseline was also determined.

  • Percent Change From Baseline in Age: Males [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of age during the study.

  • Age (Years) During the Study: Females [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of age during the study. Change from baseline was also determined.

  • Percent Change From Baseline in Age: Females [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Investigator assessment of age during the study.

  • Flow-Mediated Dilatation (FMD) During the Study [ Time Frame: Baseline, Months 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Percent (%) FMD was calculated as (hyperemic diameter minus resting diameter) divided by the resting diameter multiplied by 100. Change from baseline was also determined.

  • Percent Change From Baseline in FMD [ Time Frame: Months 6, 12, 18, 24, 30 and 36/ET ] [ Designated as safety issue: No ]
    Percent (%) FMD was calculated as (hyperemic diameter minus resting diameter) divided by the resting diameter multiplied by 100.


Secondary Outcome Measures:
  • 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) ] [ Designated as safety issue: No ]
  • Percentage of Participants by Study Drug Compliance Category [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30, and 36 (or early termination) ] [ Designated as safety issue: No ]
    Compliance to study drug was categorized as <80%, 80% - 120%, and greater than (>) 120%.


Enrollment: 272
Study Start Date: March 2009
Study Completion Date: October 2013
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Atorvastatin
All subjects will be treated with atorvastatin
Drug: atorvastatin
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)

  Eligibility

Ages Eligible for Study:   6 Years to 15 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00827606

  Show 41 Study Locations
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00827606     History of Changes
Other Study ID Numbers: A2581173, 2008-006130-95
Study First Received: January 21, 2009
Results First Received: September 11, 2014
Last Updated: June 1, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
pediatric heterozygous familial hypercholesterolemia

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Dyslipidemias
Genetic Diseases, Inborn
Hyperlipidemias
Hyperlipoproteinemias
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Metabolic Diseases
Metabolism, Inborn Errors
Atorvastatin
Anticholesteremic Agents
Antimetabolites
Enzyme Inhibitors
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Hypolipidemic Agents
Lipid Regulating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on July 05, 2015