Familial HypercholesterolemiaPhase 3NCT00827606

Atorvastatin Three Year Pediatric Study

A Phase 3 study testing atorvastatin in people with Familial Hypercholesterolemia, run by Pfizer's Upjohn has merged with Mylan to form Viatris Inc. at 40 sites in 15 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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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. Read more about this study

Who is it for?: Ages 6 to 15 years
Conditions the study is for: Familial Hypercholesterolemia
What is being tested?: atorvastatin
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 272 participants
Where: 40 sitesin 15 countries
Study dates: Mar 2009to Oct 2013
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 6 to 15 years
All sexes
No healthy volunteers
Child

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

40 sites in 15 countries.

Country

City, postcode or hospital

40 sites in 15 countries

United States7 sites

Johns Hopkins University
Baltimore, Maryland, 21287
Johns Hopkins
Baltimore, Maryland, 21287
MEDPACE Clinical Pharmacology Unit
Cincinnati, Ohio, 45212
University of Florida
Gainesville, Florida, 32608
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Children's National Medical Center
Washington D.C., District of Columbia, 20010

Other trials in United States

Slovakia5 sites

Metabolicka ambulancia, 2. Detská klinika Lekárskej fakulty Univerzity Komenského
Bratislava, 833 40
Detska fakultna nemocnica Kosice-old
Košice, 040 01
Detska fakultna nemocnica Kosice
Košice, 040 11
Lipmet, s.r.o.
Poprad, 05801
Fakultna nemocnica Trencin
Trenčín, 911 71

Other trials in Slovakia

Spain5 sites

Hospital Santa Creu I Sant Pau
Barcelona, 08041
Hospital General Universitario de Elche
Elche, Alicante, 03202
Hospital Sant Joan de Deu
Esplugues de Llobregat, 08950
Hospital de Merida
Mérida, Badajoz, 6800
Hospital Universitario Marques de Valdecilla
Santander, Cantabria, 39008

Other trials in Spain

Russia4 sites

Moscow State Healthcare Institution 'Morozovskaya Children's City Clinical Hospital'
Moscow, 119049
Autonomous Non-Profit Organization Medical Center 21 Century
Saint Petersburg, 194044
Saint-Petersburg State Pediatric Medical Academy
Saint Petersburg, 194100
Saint-Petersburg State Healthcare Institution "Children's Polyclinic #35"
Saint Petersburg, 196084

Other trials in Russia

Turkey (Türkiye)3 sites

Hacettepe University Medical Faculty. Department of Pediatrics. Nutrition and Metabolism Unit
Ankara, 06100
Gazi University, Medical Faculty
Ankara, 6500
Ege University Medical Faculty
Izmir, 35100

Other trials in Turkey (Türkiye)

Belgium2 sites

Cliniques Universitaires Saint-Luc / Pediatrie generale
Brussels, 1200
Universitaire Ziekenhuizen Leuven / Center for Metabolic Diseases
Leuven, 3000

Other trials in Belgium

Canada2 sites

Clinique des Maladies Lipidiques de Quebec
Québec, Quebec, G1V 4M6
Clinique des maladies lipidiques de Quebec Inc
Sainte-Foy, Quebec, G1V 4M6

Other trials in Canada

Germany2 sites

Charite Campus Virchow-Klinikum
Berlin, 13353
Augenaerztliche Gemeinschaftspraxis
Freiburg im Breisgau, 79098

Other trials in Germany

Hungary2 sites

Semmelweis Medical University
Budapest, H-1094
Fejer Megyei Szent Gyorgy Korhaz
Székesfehérvár, 8003

Other trials in Hungary

Italy2 sites

Dipartimento di Medicina Clinica e delle Patologie Emergenti-University Hospital of Palermo
Palermo, 90127
Dipartimento di Clinica e Terapia Medica, Università degli Studi di Roma La Sapienza
Roma, 00161

Other trials in Italy

Poland2 sites

Poradnia Chorob Metabolicznych
Bydgoszcz, 85-667
Klinika Chorob Metabolicznych
Warsaw, 04-730

Other trials in Poland

Greece1 site

1st Pediatrics Clinic , University of Athens, Agia Sofia Hospital
Athens, 115 27

Other trials in Greece

Norway1 site

Rikshospitalet Lipidklinikken
Oslo, 0027

Other trials in Norway

Puerto Rico1 site

Hospital de la Concepcion
San German, 683

Other trials in Puerto Rico

Switzerland1 site

Dr. med. Jean-Marc Nuoffer
Ch-3010 Bern

Other trials in Switzerland

About this 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

Conditions: Familial Hypercholesterolemia
Being tested: atorvastatin
Keywords: pediatric heterozygous familial hypercholesterolemia
Medical subject headings: Hyperlipoproteinemia Type II

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

atorvastatinDrug
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)

Study groups in NCT00827606
Study group	Role	What they receive
Atorvastatin	Experimental	All subjects will be treated with atorvastatin Receives: Drug: atorvastatin

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination)
Percentage of Participants by Study Drug Compliance Category
Compliance to study drug was categorized as <80%, 80% - 120%, and greater than (>) 120%.
Measured: Months 1, 2, 3, 6, 12, 18, 24, 30, and 36 (or early termination)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Mar 2009Oct 2013

First posted23 Jan 2009 (estimated)
Study startMar 2009
Primary completionOct 2013
Study completionOct 2013

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Familial HypercholesterolemiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 272 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00827606
Sponsor's protocol ID: A2581173
Other IDs: 2008-006130-95, 2008
Status last verified: Feb 2021
First submitted: 21 Jan 2009
Last updated: 21 Feb 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

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.
PubMed 27678432

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Not yet recruitingPhase 1/2
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
Heterozygous Familial Hypercholesterolemia (HeFH)
RecruitingPhase 3
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
RecruitingPhase 1
Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia
Hypercholesterolemia, Hypertriglyceridemia
Active, not recruiting
Clinical Decision Support for Familial Hypercholesterolemia
Hypercholesterolemia, Familial, Clinical Decision Support
Recruiting
The ORIGIN-FH Study
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH)

All Familial Hypercholesterolemia trials

Study at a glance

From the registry record, last updated by the sponsor on 21 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT00827606
Acronym / sponsor ID: A2581173
Conditions: Familial Hypercholesterolemia
Keywords: pediatric heterozygous familial hypercholesterolemia
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 272 participants (actual)
Lead sponsor: Pfizer's Upjohn has merged with Mylan to form Viatris Inc.Industry (pharma, biotech, devices)
Study start: Mar 2009
Primary completion: Oct 2013 (actual)
Study completion: Oct 2013 (actual)
First posted: 23 Jan 2009 (estimated)
Results: Posted 2 Jun 2015

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Atorvastatin	Experimental	Drug: atorvastatinAll subjects will be treated with atorvastatin

Who may qualify

Ages: 6 to 15 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (1 listed)

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

Key exclusion criteria (3 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
atorvastatinAtorvastatin 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)	Drug	Atorvastatin

Primary outcome

Low Density Lipoprotein Cholesterol (LDL-C; Millimoles Per Liter [mMol/L]) During the Study
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination [ET])
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in LDL-C
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or ET)
Assessments were performed in the fasting state (minimum 10-hour fast).
High-Density Lipoprotein Cholesterol (HDL-C; mMol/L) During the Study
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in HDL-C
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Total Cholesterol (mMol/L) During the Study
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in Total Cholesterol
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Trigylcerides (mMol/L) During the Study
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in Trigylcerides
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Very Low-Density Lipoprotein (VLDL; mMol/L) During the Study
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in VLDL
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Apoliprotein A-1 (Apo A-1; Grams Per Liter [g/L]) During the Study
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in Apo A-1
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Apoliprotein B (Apo B; g/L) During the Study
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in Apo B
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Number of Participants With Shift From Baseline in Tanner_Stage by Timepoint and Baseline Tanner_Stage
Time frame: Baseline, Months 6, 12, 18, 24, 30, and 36/ET
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.
Height (Centimeters [cm]) During the Study: Males
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of height changes during the study. Change from baseline was also determined.
Percent Change From Baseline in Height: Males
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of height changes during the study.
Height (cm) During the Study: Females
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of height changes during the study. Change from baseline was also determined.
Percent Change From Baseline in Height: Females
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of height changes during the study.
Weight (Kilograms [kg]) During the Study: Males
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of weight changes during the study. Change from baseline was also determined.
Percent Change From Baseline in Weight: Males
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of weight changes during the study.
Weight (kg) During the Study: Females
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of weight changes during the study. Change from baseline was also determined.
Percent Change From Baseline in Weight: Females
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of weight changes during the study.
Body Mass Index (BMI in kg Per Square Meter [kg/m^2]) During the Study: Males
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of BMI changes during the study. Change from baseline was also determined.
Percent Change From Baseline in BMI: Males
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of BMI changes during the study.
BMI (kg/m^2) During the Study: Females
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of BMI changes during the study. Change from baseline was also determined.
Percent Change From Baseline in BMI: Females
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of BMI changes during the study.
Age (Years) During the Study: Males
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of age during the study. Change from baseline was also determined.
Percent Change From Baseline in Age: Males
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of age during the study.
Age (Years) During the Study: Females
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of age during the study. Change from baseline was also determined.
Percent Change From Baseline in Age: Females
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of age during the study.
Flow-Mediated Dilatation (FMD) During the Study
Time frame: Baseline, Months 6, 12, 18, 24, 30 and 36/ET
Percent (%) FMD was calculated as (hyperemic diameter minus resting diameter) divided by the resting diameter multiplied by 100. Change from baseline was also determined.
Percent Change From Baseline in FMD
Time frame: Months 6, 12, 18, 24, 30 and 36/ET
Percent (%) FMD was calculated as (hyperemic diameter minus resting diameter) divided by the resting diameter multiplied by 100.

2 secondary outcomes

Percentage of Participants With Overall Expected Maturation and Development Consistent With Expectations as Assessed by the Investigator
Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination)
Percentage of Participants by Study Drug Compliance Category
Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30, and 36 (or early termination)

Results and safety

Results posted to the registry on 2 Jun 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Atorvastatin (5-80 mg): Tanner_Stage 1	139	112
Atorvastatin (10-80 mg): Tanner_Stage 2+	132	94

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

Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination [ET]). Mean (Standard Deviation). Unit: mMol/L

Measure	Atorvastatin (5-80 mg): Tanner_Stage 1 n = 139	Atorvastatin (10-80 mg): Tanner_Stage 2+ n = 132
Baseline (n=139,132)	6.304 (1.3130)	5.921 (1.1646)
Month 1 (n=131,130)	4.087 (1.0264)	3.675 (0.8749)
Change at Month 1 (n=131,130)	-2.214 (0.7568)	-2.233 (0.8009)
Month 2 (n=132,122)	3.719 (0.8346)	3.437 (0.7438)
Change at Month 2 (n=132,122)	-2.586 (0.9297)	-2.554 (0.9155)
Month 3 (n=126,117)	3.503 (0.7566)	3.270 (0.6533)
Change at Month 3 (n=126,117)	-2.798 (1.0251)	-2.795 (1.0835)
Month 6 (n=127,115)	3.366 (0.5787)	3.347 (0.5953)

Population analysed: FAS; n (number) equals (=) number of participants assessed for the specified parameter at a given visit.

Primary outcome: Percent Change From Baseline in LDL-C

Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or ET). Mean (Standard Deviation). Unit: percent change

Measure	Atorvastatin (5-80 mg): Tanner_Stage 1 n = 132	Atorvastatin (10-80 mg): Tanner_Stage 2+ n = 130
Month 1 (n=131,130)	-34.995 (9.7300)	-37.516 (10.0245)
Month 2 (n=132,122)	-40.371 (9.6468)	-41.939 (10.4816)
Month 3 (n=126,117)	-43.568 (10.2149)	-44.887 (12.7530)
Month 6 (n=127,115)	-45.647 (9.6969)	-43.697 (11.1836)
Month 12 (n=121,109)	-45.530 (10.5112)	-45.727 (13.5336)
Month 18 (n=116,101)	-47.101 (10.5592)	-44.818 (13.2623)
Month 24 (n=111,96)	-46.944 (11.9490)	-46.417 (13.8658)
Month 30 (n=112,94)	-46.631 (12.0206)	-47.734 (12.6696)

Population analysed: FAS; n=number of participants assessed for the specified parameter at a given visit.

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

Time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET. Mean (Standard Deviation). Unit: mMol/L

Measure	Atorvastatin (5-80 mg): Tanner_Stage 1 n = 139	Atorvastatin (10-80 mg): Tanner_Stage 2+ n = 132
Baseline (n=139,132)	1.349 (0.2732)	1.277 (0.2546)
Month 1 (n=131,130)	1.360 (0.2717)	1.292 (0.2650)
Change at Month 1 (n=131,130)	0.010 (0.1685)	0.016 (0.1740)
Month 2 (n=132,122)	1.364 (0.2805)	1.297 (0.2625)
Change at Month 2 (n=132,122)	0.014 (0.2238)	0.025 (0.1600)
Month 3 (n=126,117)	1.371 (0.2768)	1.274 (0.2606)
Change at Month 3 (n=126,117)	0.015 (0.1943)	0.011 (0.1837)
Month 6 (n=127,115)	1.337 (0.2834)	1.268 (0.2311)

Population analysed: FAS; n=number of participants assessed for the specified parameter at a given visit.

Primary outcome: Percent Change From Baseline in HDL-C

Time frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET. Mean (Standard Deviation). Unit: percent change

Measure	Atorvastatin (5-80 mg): Tanner_Stage 1 n = 132	Atorvastatin (10-80 mg): Tanner_Stage 2+ n = 130
Month 1 (n=131,130)	1.526 (12.7845)	2.192 (14.0769)
Month 2 (n=132,122)	2.195 (15.6882)	2.544 (12.7802)
Month 3 (n=126,117)	1.929 (14.7329)	1.674 (14.9525)
Month 6 (n=127,115)	-0.016 (14.8853)	1.877 (14.9113)
Month 12 (n=121,109)	-0.069 (15.2821)	-1.023 (14.1983)
Month 18 (n=116,102)	2.472 (14.8939)	-1.165 (14.3783)
Month 24 (n=111,96)	3.014 (14.9559)	1.737 (15.6924)
Month 30 (n=112,94)	3.345 (15.5080)	1.868 (17.2633)

Population analysed: FAS; n=number of participants assessed for the specified parameter at a given visit.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Baseline up through Month 36. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Atorvastatin (5-80 mg): Tanner_Stage 1	14 of 139 (10.1%)	93 of 139 (66.9%)	Not reported
Atorvastatin (10-80 mg): Tanner_Stage 2+	7 of 132 (5.3%)	86 of 132 (65.2%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 27678432Langslet 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.
To obtain contact information for a study center near you, click here.Link provided by the sponsor.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Pfizer CT.gov Call Center study director

Pfizer

40 sites in 15 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing atorvastatin in people with Familial Hypercholesterolemia, run by Pfizer's Upjohn has merged with Mylan to form Viatris Inc. at 40 sites in 15 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 21 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 40 sites in United States, Slovakia, Spain, Russia, Turkey (Türkiye) and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 2 Jun 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Low Density Lipoprotein Cholesterol (LDL-C; Millimoles Per Liter [mMol/L]) During the Study (time frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination [ET])). The study lists 33 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00827606, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Feb 2021.