8-Week PK/PD Atorvastatin Study In Children And Adolescents With Heterozygous Familial Hypercholesterolemia - Full Text View

Purpose

To evaluate pharmacokinetics, pharmacodynamics, safety and tolerability of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia

Condition	Intervention	Phase
Hypercholesterolemia, Familial	Drug: Atorvastatin	Phase I

Genetics Home Reference related topics:

cholesteryl ester storage disease Farber lipogranulomatosis hypercholesterolemia long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency mitochondrial trifunctional protein deficiency primary carnitine deficiency

MedlinePlus related topics:

Cholesterol

ChemIDplus related topics:

Atorvastatin Atorvastatin calcium

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Other, Open Label, Single Group Assignment, Pharmacokinetics/Dynamics Study

Official Title:	A 8-Week, Open-Label, Phase 1 Study To Evaluate Pharmacokinetics, Pharmacodynamics, Safety And Tolerability Of Atorvastatin In Children And Adolescents With Heterozygous Familial Hypercholesterolemia

Further study details as provided by Pfizer:

Primary Outcome Measures:

Population PK models for atorvastatin and its metabolites will be developed. Key parameters may include apparent clearance CL/F and/or apparent volume of distribution V/F of the plasma/central compartment [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Change and % change from baseline in pharmacodynamic responses of LDL, TC, TG, HDL, VLDL, and Apo A-1, and Apo B [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
Safety laboratory tests, vital signs, and adverse events [ Time Frame: 8 weeks ] [ Designated as safety issue: Yes ]
Descriptive parameter: FMD assessments [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment:	36
Study Start Date:	October 2008
Estimated Study Completion Date:	June 2009
Estimated Primary Completion Date:	June 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1 6-17 years will be administered with atorvastatin with initial doses based on age cohort	Drug: Atorvastatin 10-17 years will be a 10-mg daily dose of atorvastatin tablet formulation. 6-10 years will be a 5-mg daily dose of an atorvastatin pediatric formulation. dose may be doubled if subjects have not attained target LDL (<3.35 mmol/L) after 4-week treatment

Eligibility

Ages Eligible for Study:	6 Years to 17 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Genetically confirmed HeFH with LDL greater or equal 4 mmol/L at baseline

Exclusion Criteria:

Evidence or history of clinically significant diseases, homozygous FH

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00739999

Contacts


Contact: Pfizer CT.gov Call Center	1-800-718-1021

Sponsors and Collaborators

Pfizer

Investigators


Study Director:	Pfizer CT.gov Call Center	Pfizer

More Information

To obtain contact information for a study center near you, click here. This link exits the ClinicalTrials.gov site

Responsible Party:	Pfizer, Inc. ( Director, Clinical Trial Disclosure Group )
Study ID Numbers:	A2581172
First Received:	August 21, 2008
Last Updated:	October 7, 2008
ClinicalTrials.gov Identifier:	NCT00739999
Health Authority:	Netherlands: Independent Ethics Committee

Keywords provided by Pfizer:

heterozygous familial hypercholesterolemia (HeFH); atorvastatin; pediatric

Study placed in the following topic categories:

Lipid Metabolism, Inborn Errors

Hypercholesterolemia, autosomal dominant

Hyperlipidemias

Metabolic Diseases

Hyperlipoproteinemia Type II

Metabolism, Inborn Errors

Genetic Diseases, Inborn

Metabolic disorder

Hypercholesterolemia

Hyperlipoproteinemias

Dyslipidemias

Atorvastatin

Lipid Metabolism Disorders

Additional relevant MeSH terms:

Antimetabolites

Molecular Mechanisms of Pharmacological Action

Therapeutic Uses

Antilipemic Agents

Enzyme Inhibitors

Anticholesteremic Agents

Hydroxymethylglutaryl-CoA Reductase Inhibitors

Pharmacologic Actions

ClinicalTrials.gov processed this record on October 15, 2008

Sponsored by:	Pfizer
Information provided by:	Pfizer
ClinicalTrials.gov Identifier:	NCT00739999