Atorvastatin Three Year Pediatric Study

Familial Hypercholesterolemia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00827606
• Other study ID #: A2581173
• Secondary ID: 2008-006130-95
• Status: Completed
• Phase: Phase 3
• Start date: March 2009
• Est. completion date: October 2013

Study information

• Verified date: February 2021
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 272
• Est. completion date: October 2013
• Est. primary completion date: October 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 15 Years

Eligibility

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

Related Conditions & MeSH terms

• Familial Hypercholesterolemia
• Hypercholesterolemia
• Hyperlipoproteinemia Type II

Intervention

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)

Locations

Country	Name	City	State
Belgium	Cliniques Universitaires Saint-Luc / Pediatrie generale	Bruxelles
Belgium	Universitaire Ziekenhuizen Leuven / Center for Metabolic Diseases	Leuven
Canada	Clinique des Maladies Lipidiques de Quebec	Quebec
Canada	Clinique des maladies lipidiques de Quebec Inc	Sainte-Foy,	Quebec
Germany	Charite Campus Virchow-Klinikum	Berlin
Germany	Augenaerztliche Gemeinschaftspraxis	Freiburg
Greece	1st Pediatrics Clinic , University of Athens, Agia Sofia Hospital	Athens
Hungary	Semmelweis Medical University	Budapest
Hungary	Fejer Megyei Szent Gyorgy Korhaz	Szekesfehervar
Italy	Dipartimento di Medicina Clinica e delle Patologie Emergenti-University Hospital of Palermo	Palermo
Italy	Dipartimento di Clinica e Terapia Medica, Università degli Studi di Roma La Sapienza	Roma
Norway	Rikshospitalet Lipidklinikken	Oslo
Poland	Poradnia Chorob Metabolicznych	Bydgoszcz
Poland	Klinika Chorob Metabolicznych	Warszawa
Puerto Rico	Hospital de la Concepcion	San German
Russian Federation	Moscow State Healthcare Institution 'Morozovskaya Children's City Clinical Hospital'	Moscow
Russian Federation	Saint-Petersburg State Healthcare Institution "Children's Polyclinic #35"	Saint-Petersburg
Russian Federation	Saint-Petersburg State Pediatric Medical Academy	Saint-Petersburg
Russian Federation	Autonomous Non-Profit Organization Medical Center 21 Century	St Petersburg
Slovakia	Metabolicka ambulancia, 2. Detská klinika Lekárskej fakulty Univerzity Komenského	Bratislava
Slovakia	Detska fakultna nemocnica Kosice	Kosice
Slovakia	Detska fakultna nemocnica Kosice-old	Kosice
Slovakia	Lipmet, s.r.o.	Poprad
Slovakia	Fakultna nemocnica Trencin	Trencin
Spain	Hospital Santa Creu I Sant Pau	Barcelona
Spain	Hospital General Universitario de Elche	Elche	Alicante
Spain	Hospital Sant Joan de Deu	Esplugues de Llobregat
Spain	Hospital de Merida	Merida	Badajoz
Spain	Hospital Universitario Marques de Valdecilla	Santander	Cantabria
Switzerland	Dr. med. Jean-Marc Nuoffer	Ch-3010 Bern
Turkey	Gazi University, Medical Faculty	Ankara
Turkey	Hacettepe University Medical Faculty. Department of Pediatrics. Nutrition and Metabolism Unit	Ankara
Turkey	Ege University Medical Faculty	Izmir
United States	Johns Hopkins	Baltimore	Maryland
United States	Johns Hopkins University	Baltimore	Maryland
United States	MEDPACE Clinical Pharmacology Unit	Cincinnati	Ohio
United States	University of Florida	Gainesville	Florida
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (1)

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

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  Germany,  Greece,  Hungary,  Italy,  Norway,  Poland,  Puerto Rico,  Russian Federation,  Slovakia,  Spain,  Switzerland,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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])
Primary	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)
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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	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)
Secondary	Percentage of Participants by Study Drug Compliance Category	Compliance to study drug was categorized as <80%, 80% - 120%, and greater than (>) 120%.	Months 1, 2, 3, 6, 12, 18, 24, 30, and 36 (or early termination)

External Links

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