Phase 3
N=271
Atorvastatin Three Year Pediatric Study
Familial Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT00827606 ↗Enrolled (actual)
271
Serious AEs
7.8%
Results posted
Jun 2015
Primary outcome: Primary: Low Density Lipoprotein Cholesterol (LDL-C; Millimoles Per Liter [mMol/L]) During the Study — 6.304; 5.921; 4.087; 3.675 mMol/L
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- atorvastatin (Drug)
- Age
- Pediatric · 6+ yrs
- Sex
- All
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
- Primary completion
- Oct 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Low Density Lipoprotein Cholesterol (LDL-C; Millimoles Per Liter [mMol/L]) During the Study |
6.304; 5.921; 4.087; 3.675; -2.214; -2.233 | — |
| PRIMARY Percent Change From Baseline in LDL-C |
-34.995; -37.516; -40.371; -41.939; -43.568; -44.887 | — |
| PRIMARY High-Density Lipoprotein Cholesterol (HDL-C; mMol/L) During the Study |
1.349; 1.277; 1.360; 1.292; 0.010; 0.016 | — |
| PRIMARY Percent Change From Baseline in HDL-C |
1.526; 2.192; 2.195; 2.544; 1.929; 1.674 | — |
| PRIMARY Total Cholesterol (mMol/L) During the Study |
8.056; 7.647; 5.785; 5.352; -2.266; -2.297 | — |
| PRIMARY Percent Change From Baseline in Total Cholesterol |
-27.879; -29.653; -31.900; -33.726; -34.784; -36.126 | — |
| PRIMARY Trigylcerides (mMol/L) During the Study |
0.880; 0.980; 0.759; 0.815; -0.121; -0.166 | — |
| PRIMARY Percent Change From Baseline in Trigylcerides |
-5.950; -5.927; -3.260; -14.832; -8.760; -14.415 | — |
| PRIMARY Very Low-Density Lipoprotein (VLDL; mMol/L) During the Study |
0.403; 0.449; 0.348; 0.374; -0.056; -0.076 | — |
| PRIMARY Percent Change From Baseline in VLDL |
-6.060; -5.805; -3.097; -14.694; -8.711; -14.263 | — |
| PRIMARY Apoliprotein A-1 (Apo A-1; Grams Per Liter [g/L]) During the Study |
1.396; 1.308; 1.419; 1.337; 0.025; 0.032 | — |
| PRIMARY Percent Change From Baseline in Apo A-1 |
2.229; 3.301; 1.701; 2.499; 1.041; 1.192 | — |
| PRIMARY Apoliprotein B (Apo B; g/L) During the Study |
1.454; 1.381; 1.062; 0.967; -0.395; -0.415 | — |
| PRIMARY Percent Change From Baseline in Apo B |
-26.933; -29.851; -31.867; -33.959; -35.163; -35.849 | — |
| PRIMARY Number of Participants With Shift From Baseline in Tanner_Stage by Timepoint and Baseline Tanner_Stage |
107; 0; 0; 0; 0; 14 | — |
| PRIMARY Height (Centimeters [cm]) During the Study: Males |
144.36; 145.21; 0.68; 145.93; 1.27; 146.00 | — |
| PRIMARY Percent Change From Baseline in Height: Males |
0.47; 0.88; 1.19; 2.25; 4.66; 6.53 | — |
| PRIMARY Height (cm) During the Study: Females |
145.28; 146.06; 0.71; 146.57; 1.19; 146.81 | — |
| PRIMARY Percent Change From Baseline in Height: Females |
0.51; 0.84; 1.20; 1.98; 3.93; 5.30 | — |
| PRIMARY Weight (Kilograms [kg]) During the Study: Males |
40.79; 41.18; 0.36; 41.83; 0.80; 41.68 | — |
| PRIMARY Percent Change From Baseline in Weight: Males |
0.94; 2.04; 2.73; 5.09; 11.85; 18.03 | — |
| PRIMARY Weight (kg) During the Study: Females |
42.14; 42.50; 0.36; 42.86; 0.67; 43.34 | — |
| PRIMARY Percent Change From Baseline in Weight: Females |
0.97; 1.85; 3.16; 5.37; 11.65; 16.79 | — |
| PRIMARY Body Mass Index (BMI in kg Per Square Meter [kg/m^2]) During the Study: Males |
18.97; 18.94; 0.01; 19.06; 0.06; 18.99 | — |
| PRIMARY Percent Change From Baseline in BMI: Males |
0.03; 0.31; 0.39; 0.55; 2.10; 3.98 | — |
| PRIMARY BMI (kg/m^2) During the Study: Females |
19.44; 19.45; -0.02; 19.47; 0.00; 19.54 | — |
| PRIMARY Percent Change From Baseline in BMI: Females |
-0.06; 0.16; 0.73; 1.26; 3.33; 5.14 | — |
| PRIMARY Age (Years) During the Study: Males |
9.94; 10.05; 0.11; 10.14; 0.18; 10.21 | — |
| PRIMARY Percent Change From Baseline in Age: Males |
1.09; 1.86; 3.16; 6.06; 11.13; 16.82 | — |
| PRIMARY Age (Years) During the Study: Females |
10.55; 10.66; 0.11; 10.78; 0.20; 10.75 | — |
| PRIMARY Percent Change From Baseline in Age: Females |
1.10; 2.00; 2.74; 5.18; 10.00; 15.20 | — |
| PRIMARY Flow-Mediated Dilatation (FMD) During the Study |
5.523; 6.651; 5.749; 6.520; -0.063; -0.759 | — |
| PRIMARY Percent Change From Baseline in FMD |
14.867; -0.205; -3.880; 15.444; -4.598; -24.387 | — |
| SECONDARY Percentage of Participants With Overall Expected Maturation and Development Consistent With Expectations as Assessed by the Investigator |
99.3; 100.0; 100.0; 100.0; 100.0; 100.0 | — |
| SECONDARY Percentage of Participants by Study Drug Compliance Category |
3.0; 6.8; 97.0; 93.2; 0; 0 | — |
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
Data sourced from ClinicalTrials.gov (NCT00827606). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.