Clinical trial NCT00827606: Atorvastatin Three Year Pediatric Study
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Familial Hypercholesterolemia
Keywords: pediatric heterozygous familial hypercholesterolemia
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2009-01-23
Enrollment: 272
Sex: ALL
Minimum age (years): 6
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Low Density Lipoprotein Cholesterol (LDL-C; Millimoles Per Liter [mMol/L]) During the Study
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination [ET])
measure: Percent Change From Baseline in LDL-C
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or ET)
measure: High-Density Lipoprotein Cholesterol (HDL-C; mMol/L) During the Study
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in HDL-C
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Total Cholesterol (mMol/L) During the Study
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in Total Cholesterol
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Trigylcerides (mMol/L) During the Study
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in Trigylcerides
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Very Low-Density Lipoprotein (VLDL; mMol/L) During the Study
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in VLDL
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Apoliprotein A-1 (Apo A-1; Grams Per Liter [g/L]) During the Study
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in Apo A-1
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Apoliprotein B (Apo B; g/L) During the Study
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in Apo B
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Number of Participants With Shift From Baseline in Tanner_Stage by Timepoint and Baseline Tanner_Stage
timeFrame: Baseline, Months 6, 12, 18, 24, 30, and 36/ET
measure: Height (Centimeters [cm]) During the Study: Males
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in Height: Males
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Height (cm) During the Study: Females
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in Height: Females
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Weight (Kilograms [kg]) During the Study: Males
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in Weight: Males
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Weight (kg) During the Study: Females
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in Weight: Females
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Body Mass Index (BMI in kg Per Square Meter [kg/m^2]) During the Study: Males
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in BMI: Males
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: BMI (kg/m^2) During the Study: Females
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in BMI: Females
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Age (Years) During the Study: Males
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in Age: Males
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Age (Years) During the Study: Females
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in Age: Females
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
measure: Flow-Mediated Dilatation (FMD) During the Study
timeFrame: Baseline, Months 6, 12, 18, 24, 30 and 36/ET
measure: Percent Change From Baseline in FMD
timeFrame: Months 6, 12, 18, 24, 30 and 36/ET
Secondary outcomes:
measure: Percentage of Participants With Overall Expected Maturation and Development Consistent With Expectations as Assessed by the Investigator
timeFrame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination)
measure: Percentage of Participants by Study Drug Compliance Category
timeFrame: Months 1, 2, 3, 6, 12, 18, 24, 30, and 36 (or early termination)
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins
state: Maryland
city: Cincinnati
country: United States
facility: MEDPACE Clinical Pharmacology Unit
state: Ohio
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Bruxelles
country: Belgium
facility: Cliniques Universitaires Saint-Luc / Pediatrie generale
city: Leuven
country: Belgium
facility: Universitaire Ziekenhuizen Leuven / Center for Metabolic Diseases
city: Sainte-Foy,
country: Canada
facility: Clinique des maladies lipidiques de Quebec Inc
state: Quebec
city: Quebec
country: Canada
facility: Clinique des Maladies Lipidiques de Quebec
city: Berlin
country: Germany
facility: Charite Campus Virchow-Klinikum
city: Freiburg
country: Germany
facility: Augenaerztliche Gemeinschaftspraxis
city: Athens
country: Greece
facility: 1st Pediatrics Clinic , University of Athens, Agia Sofia Hospital
city: Budapest
country: Hungary
facility: Semmelweis Medical University
city: Szekesfehervar
country: Hungary
facility: Fejer Megyei Szent Gyorgy Korhaz
city: Palermo
country: Italy
facility: Dipartimento di Medicina Clinica e delle Patologie Emergenti-University Hospital of Palermo
city: Roma
country: Italy
facility: Dipartimento di Clinica e Terapia Medica, Università degli Studi di Roma La Sapienza
city: Oslo
country: Norway
facility: Rikshospitalet Lipidklinikken
city: Bydgoszcz
country: Poland
facility: Poradnia Chorob Metabolicznych
city: Warszawa
country: Poland
facility: Klinika Chorob Metabolicznych
city: San German
country: Puerto Rico
facility: Hospital de la Concepcion
city: Moscow
country: Russian Federation
facility: Moscow State Healthcare Institution 'Morozovskaya Children's City Clinical Hospital'
city: Saint-Petersburg
country: Russian Federation
facility: Saint-Petersburg State Pediatric Medical Academy
city: Saint-Petersburg
country: Russian Federation
facility: Saint-Petersburg State Healthcare Institution "Children's Polyclinic #35"
city: St Petersburg
country: Russian Federation
facility: Autonomous Non-Profit Organization Medical Center 21 Century
city: Bratislava
country: Slovakia
facility: Metabolicka ambulancia, 2. Detská klinika Lekárskej fakulty Univerzity Komenského
city: Kosice
country: Slovakia
facility: Detska fakultna nemocnica Kosice-old
city: Kosice
country: Slovakia
facility: Detska fakultna nemocnica Kosice
city: Poprad
country: Slovakia
facility: Lipmet, s.r.o.
city: Trencin
country: Slovakia
facility: Fakultna nemocnica Trencin
city: Elche
country: Spain
facility: Hospital General Universitario de Elche
state: Alicante
city: Merida
country: Spain
facility: Hospital de Merida
state: Badajoz
city: Santander
country: Spain
facility: Hospital Universitario Marques de Valdecilla
state: Cantabria
city: Barcelona
country: Spain
facility: Hospital Santa Creu I Sant Pau
city: Esplugues de Llobregat
country: Spain
facility: Hospital Sant Joan de Deu
city: Ch-3010 Bern
country: Switzerland
facility: Dr. med. Jean-Marc Nuoffer
city: Ankara
country: Turkey
facility: Hacettepe University Medical Faculty. Department of Pediatrics. Nutrition and Metabolism Unit
city: Ankara
country: Turkey
facility: Gazi University, Medical Faculty
city: Izmir
country: Turkey
facility: Ege University Medical Faculty
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: 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.