Atorvastatin Three Year Pediatric Study

NCT00827606 | Completed Phase 3 Results DMC

• 2 other identifiers: A25811732008-006130-95
• Study Type: interventional
• Target: 272
• Locations: 15 countries
• Sites: 40

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.

Conditions

Familial Hypercholesterolemia

Keywords

pediatric heterozygous familial hypercholesterolemia

Outcome Measures

Primary Outcomes (33)

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

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination [ET])

• Percent Change From Baseline in LDL-C

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

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

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

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in HDL-C

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

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

• Total Cholesterol (mMol/L) During the Study

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in Total Cholesterol

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

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

• Trigylcerides (mMol/L) During the Study

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in Trigylcerides

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

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

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

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in VLDL

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

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

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

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in Apo A-1

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

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

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

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in Apo B

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

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

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

  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.

  Baseline, Months 6, 12, 18, 24, 30, and 36/ET

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

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in Height: Males

  Investigator assessment of height changes during the study.

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

• Height (cm) During the Study: Females

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in Height: Females

  Investigator assessment of height changes during the study.

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

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

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in Weight: Males

  Investigator assessment of weight changes during the study.

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

• Weight (kg) During the Study: Females

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in Weight: Females

  Investigator assessment of weight changes during the study.

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

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

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in BMI: Males

  Investigator assessment of BMI changes during the study.

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

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

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in BMI: Females

  Investigator assessment of BMI changes during the study.

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

• Age (Years) During the Study: Males

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in Age: Males

  Investigator assessment of age during the study.

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

• Age (Years) During the Study: Females

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

  Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in Age: Females

  Investigator assessment of age during the study.

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

• Flow-Mediated Dilatation (FMD) During the Study

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

  Baseline, Months 6, 12, 18, 24, 30 and 36/ET

• Percent Change From Baseline in FMD

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

  Months 6, 12, 18, 24, 30 and 36/ET

Secondary Outcomes (2)

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

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

• Percentage of Participants by Study Drug Compliance Category

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

Study Arms (1)

Atorvastatin

EXPERIMENTAL

All subjects will be treated with atorvastatin

Drug: atorvastatin

Interventions

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)

Atorvastatin

Eligibility Criteria

• Age: 6 Years - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Heterozygous familial hypercholesterolemia, ages 6-15, LDL greater than 4 mmol/l

You may not qualify if:

• 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

Sponsors & Collaborators

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

Study Sites (40)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

University of Florida

Gainesville, Florida, 32608, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

Johns Hopkins

Baltimore, Maryland, 21287, United States

Location

MEDPACE Clinical Pharmacology Unit

Cincinnati, Ohio, 45212, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Cliniques Universitaires Saint-Luc / Pediatrie generale

Brussels, 1200, Belgium

Location

Universitaire Ziekenhuizen Leuven / Center for Metabolic Diseases

Leuven, 3000, Belgium

Location

Clinique des Maladies Lipidiques de Quebec

Québec, Quebec, G1V 4M6, Canada

Location

Clinique des maladies lipidiques de Quebec Inc

Sainte-Foy, Quebec, G1V 4M6, Canada

Location

Charite Campus Virchow-Klinikum

Berlin, 13353, Germany

Location

Augenaerztliche Gemeinschaftspraxis

Freiburg im Breisgau, 79098, Germany

Location

1st Pediatrics Clinic , University of Athens, Agia Sofia Hospital

Athens, 115 27, Greece

Location

Semmelweis Medical University

Budapest, H-1094, Hungary

Location

Fejer Megyei Szent Gyorgy Korhaz

Székesfehérvár, 8003, Hungary

Location

Dipartimento di Medicina Clinica e delle Patologie Emergenti-University Hospital of Palermo

Palermo, 90127, Italy

Location

Dipartimento di Clinica e Terapia Medica, Università degli Studi di Roma La Sapienza

Roma, 00161, Italy

Location

Rikshospitalet Lipidklinikken

Oslo, 0027, Norway

Location

Poradnia Chorob Metabolicznych

Bydgoszcz, 85-667, Poland

Location

Klinika Chorob Metabolicznych

Warsaw, 04-730, Poland

Location

Hospital de la Concepcion

San Germán, 683, Puerto Rico

Location

Moscow State Healthcare Institution 'Morozovskaya Children's City Clinical Hospital'

Moscow, 119049, Russia

Location

Autonomous Non-Profit Organization Medical Center 21 Century

Saint Petersburg, 194044, Russia

Location

Saint-Petersburg State Pediatric Medical Academy

Saint Petersburg, 194100, Russia

Location

Saint-Petersburg State Healthcare Institution "Children's Polyclinic #35"

Saint Petersburg, 196084, Russia

Location

Metabolicka ambulancia, 2. Detská klinika Lekárskej fakulty Univerzity Komenského

Bratislava, 833 40, Slovakia

Location

Detska fakultna nemocnica Kosice-old

Košice, 040 01, Slovakia

Location

Detska fakultna nemocnica Kosice

Košice, 040 11, Slovakia

Location

Lipmet, s.r.o.

Poprad, 05801, Slovakia

Location

Fakultna nemocnica Trencin

Trenčín, 911 71, Slovakia

Location

Hospital General Universitario de Elche

Elche, Alicante, 03202, Spain

Location

Hospital de Merida

Mérida, Badajoz, 6800, Spain

Location

Hospital Universitario Marques de Valdecilla

Santander, Cantabria, 39008, Spain

Location

Hospital Santa Creu I Sant Pau

Barcelona, 08041, Spain

Location

Hospital Sant Joan de Deu

Esplugues de Llobregat, 08950, Spain

Location

Dr. med. Jean-Marc Nuoffer

Ch-3010 Bern, Switzerland

Location

Hacettepe University Medical Faculty. Department of Pediatrics. Nutrition and Metabolism Unit

Ankara, 06100, Turkey (Türkiye)

Location

Gazi University, Medical Faculty

Ankara, 6500, Turkey (Türkiye)

Location

Ege University Medical Faculty

Izmir, 35100, Turkey (Türkiye)

Location

Related Publications (1)

• Langslet G, Breazna A, Drogari E. A 3-year study of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia. J Clin Lipidol. 2016 Sep-Oct;10(5):1153-1162.e3. doi: 10.1016/j.jacl.2016.05.010. Epub 2016 Jun 7.

  PMID: 27678432 DERIVED

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hyperlipoproteinemia Type II

Interventions

Atorvastatin

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pyrroles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heptanoic Acids; Fatty Acids; Lipids

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 21, 2009
• First Posted: January 23, 2009
• Study Start: March 1, 2009
• Primary Completion: October 1, 2013
• Study Completion: October 1, 2013
• Last Updated: February 21, 2021
• Results First Posted: June 2, 2015

Record last verified: 2021-02

Locations

SL (5); US (7); TU (3); CA (2); GR (1); NO (1); IT (2); HU (2); PL (2); ES (5); DE (2); PR (1); CH (1); BE (2); RU (4)