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Atorvastatin Three Year Pediatric Study

This study has been completed.
Information provided by (Responsible Party):
Pfizer Identifier:
First received: January 21, 2009
Last updated: November 4, 2013
Last verified: November 2013

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 2
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:
  • To characterize long-term, three year descriptive growth and development (ie, height, weight, body mass index, Tanner Stage) in pediatric subjects with HeFH receiving atorvastatin treatment [ Time Frame: 3 monthly visits post treatment initiation, next visit 3 months later, then every 6 months for the r ] [ Designated as safety issue: No ]
  • To characterize long-term, three year descriptive efficacy (LDL-C, TC, TG, HDL, VLDL, Apo A-1, Apo B), tolerability and safety in pediatric subjects with HeFH receiving atorvastatin treatment. [ Time Frame: 3 monthly visits post treatment initiation, next visit 3 months later, then every 6 months for the r ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • To estimate long-term, three year efficacy via the descriptive exploratory biomarker of Flow Mediated Dilation (FMD) in a subset of the HeFH pediatric subjects treated with atorvastatin in this study [ Time Frame: 3 monthly visits post treatment initiation, next visit 3 months later, then every 6 months for the r ] [ Designated as safety issue: No ]

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)


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

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 identifier: NCT00827606

  Show 42 Study Locations
Sponsors and Collaborators
Study Director: Pfizer Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer Identifier: NCT00827606     History of Changes
Other Study ID Numbers: A2581173
Study First Received: January 21, 2009
Last Updated: November 4, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
pediatric heterozygous familial hypercholesterolemia

Additional relevant MeSH terms:
Hyperlipoproteinemia Type II
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Metabolic Diseases
Metabolism, Inborn Errors
Anticholesteremic Agents
Enzyme Inhibitors
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Hypolipidemic Agents
Lipid Regulating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Therapeutic Uses processed this record on February 27, 2015