Atorvastatin Three Year Pediatric Study

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00827606
First received: January 21, 2009
Last updated: June 1, 2015
Last verified: June 2015
Results First Received: September 11, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Familial Hypercholesterolemia
Intervention: Drug: atorvastatin

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Atorvastatin (5-80 mg): Tanner_Stage 1 Participants aged 6 to less than (<)10 years, at Tanner_Stage 1 received an initial dose of atorvastatin tablets, 5 milligrams per day (mg/day), orally (PO), through Week 4; after Week 4, dose may have been doubled to 10 mg/day, with subsequent doubling to 20 mg/day (as necessary; maximum dose was 80 mg/day), PO, if target low-density lipoprotein cholesterol (LDL-C) (<3.35 millimoles per liter [mmol/L]) was not attained.
Atorvastatin (10-80 mg): Tanner_Stage 2+ Participants aged greater than or equal to (≥) 10 to 15 years, at Tanner_Stage 2+ received an initial dose of atorvastatin tablets, 10 mg/day, PO, through Week 4; after Week 4, dose may have been doubled to 20 mg/day, with subsequent doubling to 40 mg/day (as necessary; maximum dose was 80 mg/day), PO, if target LDL-C (<3.35 mmol/L) was not attained.

Participant Flow:   Overall Study
    Atorvastatin (5-80 mg): Tanner_Stage 1     Atorvastatin (10-80 mg): Tanner_Stage 2+  
STARTED     139     132  
COMPLETED     112     94  
NOT COMPLETED     27     38  
Protocol Violation                 4                 3  
Lost to Follow-up                 3                 1  
Withdrawal by Subject                 7                 6  
Pregnancy                 0                 1  
Not specified                 5                 13  
LDL below 2.59 mmol/L                 4                 12  
Adverse Event                 4                 2  



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Full Analysis Set (FAS): all participants who took at least one dose of study drug.

Reporting Groups
  Description
Atorvastatin (5-80 mg): Tanner_Stage 1 Participants aged 6 to <10 years, at Tanner_Stage 1 received an initial dose of atorvastatin tablets, 5 mg/day, PO, through Week 4; after Week 4, dose may have been doubled to 10 mg/day, with subsequent doubling to 20 mg/day (as necessary; maximum dose was 80 mg/day), PO, if target LDL-C (<3.35 mmol/L) was not attained.
Atorvastatin (10-80 mg): Tanner_Stage 2+ Participants aged ≥10 to 15 years, at Tanner_Stage 2+ received an initial dose of atorvastatin tablets, 10 mg/day, PO, through Week 4; after Week 4, dose may have been doubled to 20 mg/day, with subsequent doubling to 40 mg/day (as necessary; maximum dose was 80 mg/day), PO, if target LDL-C (<3.35 mmol/L) was not attained.
Total Total of all reporting groups

Baseline Measures
    Atorvastatin (5-80 mg): Tanner_Stage 1     Atorvastatin (10-80 mg): Tanner_Stage 2+     Total  
Number of Participants  
[units: participants]
  139     132     271  
Age  
[units: years]
Mean (Standard Deviation)
  8.5  (1.86)     12.0  (1.68)     10.2  (2.48)  
Gender  
[units: participants]
     
Female     46     79     125  
Male     93     53     146  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   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]) ]

2.  Primary:   Percent Change From Baseline in LDL-C   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or ET) ]

3.  Primary:   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 ]

4.  Primary:   Percent Change From Baseline in HDL-C   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

5.  Primary:   Total Cholesterol (mMol/L) During the Study   [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

6.  Primary:   Percent Change From Baseline in Total Cholesterol   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

7.  Primary:   Trigylcerides (mMol/L) During the Study   [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

8.  Primary:   Percent Change From Baseline in Trigylcerides   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

9.  Primary:   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 ]

10.  Primary:   Percent Change From Baseline in VLDL   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

11.  Primary:   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 ]

12.  Primary:   Percent Change From Baseline in Apo A-1   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

13.  Primary:   Apoliprotein B (Apo B; g/L) During the Study   [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

14.  Primary:   Percent Change From Baseline in Apo B   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

15.  Primary:   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 ]

16.  Primary:   Height (Centimeters [cm]) During the Study: Males   [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

17.  Primary:   Percent Change From Baseline in Height: Males   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

18.  Primary:   Height (cm) During the Study: Females   [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

19.  Primary:   Percent Change From Baseline in Height: Females   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

20.  Primary:   Weight (Kilograms [kg]) During the Study: Males   [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

21.  Primary:   Percent Change From Baseline in Weight: Males   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

22.  Primary:   Weight (kg) During the Study: Females   [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

23.  Primary:   Percent Change From Baseline in Weight: Females   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

24.  Primary:   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 ]

25.  Primary:   Percent Change From Baseline in BMI: Males   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

26.  Primary:   BMI (kg/m^2) During the Study: Females   [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

27.  Primary:   Percent Change From Baseline in BMI: Females   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

28.  Primary:   Age (Years) During the Study: Males   [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

29.  Primary:   Percent Change From Baseline in Age: Males   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

30.  Primary:   Age (Years) During the Study: Females   [ Time Frame: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

31.  Primary:   Percent Change From Baseline in Age: Females   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET ]

32.  Primary:   Flow-Mediated Dilatation (FMD) During the Study   [ Time Frame: Baseline, Months 6, 12, 18, 24, 30 and 36/ET ]

33.  Primary:   Percent Change From Baseline in FMD   [ Time Frame: Months 6, 12, 18, 24, 30 and 36/ET ]

34.  Secondary:   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) ]

35.  Secondary:   Percentage of Participants by Study Drug Compliance Category   [ Time Frame: Months 1, 2, 3, 6, 12, 18, 24, 30, and 36 (or early termination) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
  Hide More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
phone: 1-800-718-1021
e-mail: ClinicalTrials.gov_Inquiries@pfizer.com


No publications provided


Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00827606     History of Changes
Other Study ID Numbers: A2581173, 2008-006130-95
Study First Received: January 21, 2009
Results First Received: September 11, 2014
Last Updated: June 1, 2015
Health Authority: United States: Food and Drug Administration