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Efficacy and Safety of Colesevelam in Pediatric Patients With Genetic High Cholesterol

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00145574
Recruitment Status : Completed
First Posted : September 5, 2005
Results First Posted : January 26, 2010
Last Update Posted : April 15, 2010
Sponsor:
Information provided by:
Daiichi Sankyo, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Hypercholesterolemia
Interventions Drug: colesevelam HCl
Drug: placebo
Enrollment 194
Recruitment Details Study was conducted at 41 clinical sites; Australia (1 site), Austria (1 site), Canada (5 sites), Hungary (1 site), Israel (5 sites), New Zealand (1 site), Norway (2 sites), Slovakia (3 sites), South Africa (4 sites), Czech Republic (3 sites), Netherlands (2 sites), and USA (13 sites). Study initiated November 5, 2005 completed December 18, 2007.
Pre-assignment Details Period I Run-In (4 weeks): Period I was a single-blind stabilization period prior to randomization. All subjects received 6 placebo tablets daily. Objective was to evaluate dosing compliance and tolerability to the tablets prior to randomization. Subjects could be on stable pediatric approved statin regimen and low cholesterol diet for 6 weeks.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam
Hide Arm/Group Description Placebo similar to active Low dose colesevelam 1.9 grams per day High dose colesevelam 3.8 grams per day
Period Title: Double Blind
Started 65 65 64
Completed 64 60 62
Not Completed 1 5 2
Reason Not Completed
Adverse Event             0             3             1
Withdrawal by Subject             1             1             1
required restricted medicine             0             1             0
Period Title: Open Label Long-term Extension
Started 0 0 184 [1]
Completed 0 0 173
Not Completed 0 0 11
Reason Not Completed
Adverse Event             0             0             5
Lost to Follow-up             0             0             1
Withdrawal by Subject             0             0             4
required restricted medicine             0             0             1
[1]
2 participants discontinued after completing double-blind but before starting open label.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam Total
Hide Arm/Group Description Placebo similar to active Low dose colesevelam 1.9 grams per day High dose colesevelam 3.8 grams per day Total of all reporting groups
Overall Number of Baseline Participants 65 65 64 194
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 65 participants 65 participants 64 participants 194 participants
<=18 years
65
 100.0%
65
 100.0%
64
 100.0%
194
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 65 participants 65 participants 64 participants 194 participants
14.3  (1.74) 14.1  (2.19) 13.9  (2.0) 14.1  (1.98)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 65 participants 65 participants 64 participants 194 participants
Female
21
  32.3%
26
  40.0%
24
  37.5%
71
  36.6%
Male
44
  67.7%
39
  60.0%
40
  62.5%
123
  63.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 65 participants 65 participants 64 participants 194 participants
United States 16 14 15 45
Canada 7 9 7 23
Austria 0 1 0 1
South Africa 18 16 17 51
Israel 6 6 4 16
Norway 3 3 4 10
Netherlands 3 3 4 10
Hungary 6 7 6 19
New Zealand 2 1 2 5
Slovakia 2 0 2 4
Czech Republic 2 5 3 10
Statin Status   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 65 participants 65 participants 64 participants 194 participants
non-naive 48 50 49 147
naive 17 15 15 47
[1]
Measure Description: Non-naive refers to the number of participants who are taking statins. Naive refers to the number of participants who are not taking statins.
Tanner Stage   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 65 participants 65 participants 64 participants 194 participants
I 0 0 0 0
II 9 15 15 39
III 20 16 16 52
IV 23 19 20 62
V 13 15 13 41
[1]
Measure Description: Tanner Stage represents a scale measuring physical development in children based on primary and secondary sexual characteristics. The scale has five stages, with Stage I representing prepuberty and Stage 5 representing adult physical development. Please refer to Tanner, JM; Growth at Adolescence, @nd ed. Oxford, England, Blackwell Scientific Publications, 1962 for complete definitions of each Tanner Stage of development.
Body Mass Index   [1] 
Mean (Standard Deviation)
Unit of measure:  Kg/m2
Number Analyzed 65 participants 65 participants 64 participants 194 participants
21.9  (4.30) 23.4  (6.14) 22.2  (4.75) 22.5  (5.14)
[1]
Measure Description: Body Mass Index (BMI) kg/m2
height   [1] 
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 65 participants 65 participants 64 participants 194 participants
164.8  (10.35) 160.7  (10.90) 162.1  (11.94) 162.5  (11.16)
[1]
Measure Description: height in centimeters (cm)
weight   [1] 
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 65 participants 65 participants 64 participants 194 participants
60.3  (15.32) 61.5  (20.77) 59.0  (16.81) 60.3  (17.72)
[1]
Measure Description: mean weight in kilograms (kg) and standard deviation (SD)
1.Primary Outcome
Title Percent Change in Plasma Low Density Lipoprotein-cholesterol (LDL-C) From Day 1 (Study Baseline) to Week 8.
Hide Description Percent change in LDL-C (mg/dL) and standard deviation (SD) from Day 1 (Study Baseline)to Week 8 (last observation carried forward - LOCF)- Intent-to-Treat ITT population.
Time Frame 8 weeks (week 8 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population for Double blind Period. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam
Hide Arm/Group Description:
Placebo similar to active
Low dose colesevelam 1.9 grams per day
High dose colesevelam 3.8 grams per day
Overall Number of Participants Analyzed 65 63 63
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
2.9  (16.46) -3.7  (18.36) -10.6  (19.36)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose Colesevelam
Comments The primary null hypotheses were tested sequentially in the following order: 1) no difference between the high-dose colesevelam HCl and placebo for percent change in LDL-C from study baseline to Week 8 endpoint with the last observation carried forward (LOCF) and 2) no difference between the low-dose colesevelam HCl and placebo for percent change in LDL-C from study baseline to Week 8 endpoint with LOCF. The hypotheses were tested at a 2-sided significance level of 5%.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1122
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
2.Secondary Outcome
Title Percent Change in Plasma Total Cholesterol (TC) From Day 1 (Study Baseline) to Week 8.
Hide Description Percent change in total cholesterol (mg/dL) and standard deviation (SD) from Day 1 (Study Baseline) to Week 8 (last observation carried forward - LOCF)- Intent-to-Treat ITT population.
Time Frame 8 weeks (week 8 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population for Double blind Period. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam
Hide Arm/Group Description:
Placebo similar to active
Low dose colesevelam 1.9 grams per day
High dose colesevelam 3.8 grams per day
Overall Number of Participants Analyzed 65 63 63
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
2.9  (13.29) -1.1  (14.22) -5.4  (15.8)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5260
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0085
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
3.Secondary Outcome
Title Percent Change in Plasma Triglycerides (TG) From Day 1 (Study Baseline) to Week 8.
Hide Description Percent change in triglycerides (mg/dL) and standard deviation (SD) from Day 1 (Study Baseline) to Week 8 (last observation carried forward - LOCF)- Intent-to-Treat ITT population.
Time Frame 8 weeks (week 8 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population for Double blind Period. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam
Hide Arm/Group Description:
Placebo similar to active
Low dose colesevelam 1.9 grams per day
High dose colesevelam 3.8 grams per day
Overall Number of Participants Analyzed 65 63 63
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
12.5  (40.8) 16.9  (35.7) 12.5  (53.2)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0008
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
4.Secondary Outcome
Title Percent Change in Plasma High-density Lipoprotein-cholesterol (HDL-C) From Day 1 (Study Baseline) to Week 8.
Hide Description Percent change in HDL-C (mg/dL) and standard deviation (SD) from Day 1 (Study Baseline) to Week 8 (last observation carried forward - LOCF)- Intent-to-Treat ITT population.
Time Frame 8 weeks (week 8 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population for Double blind Period. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam
Hide Arm/Group Description:
Placebo similar to active
Low dose colesevelam 1.9 grams per day
High dose colesevelam 3.8 grams per day
Overall Number of Participants Analyzed 65 63 63
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
2.5  (12.52) 3.9  (12.45) 8.5  (14.72)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0155
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
5.Secondary Outcome
Title Percent Change in Plasma Non-high Density Lipoprotein-cholesterol (Non-HDL-C) From Day 1 (Study Baseline) to Week 8.
Hide Description Percent change in non-HDL-C (mg/dL) and standard deviation (SD) from Day 1 (Study Baseline) to Week 8 (last observation carried forward - LOCF)- Intent-to-Treat ITT population.
Time Frame 8 weeks (week 8 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population for Double blind Period. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam
Hide Arm/Group Description:
Placebo similar to active
Low dose colesevelam 1.9 grams per day
High dose colesevelam 3.8 grams per day
Overall Number of Participants Analyzed 65 63 63
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
3.4  (16.01) -2.1  (17.32) -8.4  (18.28)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3482
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0006
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
6.Secondary Outcome
Title Percent Change in Plasma Apolipoprotien A-I (Apo A-1) From Day 1 (Study Baseline) to Week 8.
Hide Description Percent change in Apolipoprotien A-I (Apo A-1) (mg/dL) and standard deviation (SD) from Day 1 (Study Baseline) to Week 8 (last observation carried forward - LOCF)- Intent-to-Treat ITT population.
Time Frame 8 weeks (week 8 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population in Double blind Period. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam
Hide Arm/Group Description:
Placebo similar to active
Low dose colesevelam 1.9 grams per day
High dose colesevelam 3.8 grams per day
Overall Number of Participants Analyzed 63 62 61
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
4.4  (14.62) 7.0  (13.96) 11.2  (16.79)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
7.Secondary Outcome
Title Percent Change in Plasma Apolipoprotein B (Apo B) From Day 1 (Study Baseline) to Week 8.
Hide Description Percent change in Apo B (mg/dL) and standard deviation (SD) from Day 1 (Study Baseline) to Week 8 (last observation carried forward - LOCF)- Intent-to-Treat ITT population.
Time Frame 8 weeks (week 8 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population in Double blind Period. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam
Hide Arm/Group Description:
Placebo similar to active
Low dose colesevelam 1.9 grams per day
High dose colesevelam 3.8 grams per day
Overall Number of Participants Analyzed 63 62 61
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
2.3  (14.78) -0.7  (16.52) -7.0  (14.45)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7433
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose Colesevelam
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0003
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
8.Secondary Outcome
Title Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Study Baseline (Day 1) to Week 26.
Hide Description Percent change in low-density lipoprotein cholesterol from baseline to Week 26 was calculated for subpopulations representing each assigned treatment group during the Double blind Period, and for the overall population in the Open Label Extension Period.
Time Frame 26 weeks (week 26 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam All High Dose Colesevelam in Open Label Extension
Hide Arm/Group Description:
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to placebo treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to low dose colesevelam 1.9 grams per day treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to high dose colesevelam 3.8 grams per day treatment during the Double blind Period.
Open Label Extension was an 18 week open-label treatment period. All participants were treated with 3750 mg colesevelam (high-dose).
Overall Number of Participants Analyzed 62 56 60 178
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
-11.9  (22.39) -16.8  (19.85) -13.5  (21.58) -14.0  (21.32)
9.Secondary Outcome
Title Percent Change in Total Cholesterol From Study Baseline (Day 1) to Week 26.
Hide Description Percent change in total cholesterol (TC) from baseline to Week 26 was calculated for subpopulations representing each assigned treatment group during the Double blind Period, and for the overall population in the Open Label Extension Period.
Time Frame 26 weeks (week 26 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam All High Dose Colesevelam in Open Label Extension
Hide Arm/Group Description:
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to placebo treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to low dose colesevelam 1.9 grams per day treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to high dose colesevelam 3.8 grams per day treatment during the Double blind Period.
Open Label Extension was an 18 week open-label treatment period. All participants were treated with 3750 mg colesevelam (high-dose).
Overall Number of Participants Analyzed 62 56 60 178
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
-7.5  (17.21) -9.1  (16.91) -7.5  (17.55) -8.0  (17.15)
10.Secondary Outcome
Title Percent Change in Triglycerides From Study Baseline (Day 1) to Week 26.
Hide Description Percent change in triglycerides from baseline to Week 26 was calculated for subpopulations representing each assigned treatment group during the Double blind Period, and for the overall population in the Open Label Extension Period.
Time Frame 26 weeks (week 26 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam All High Dose Colesevelam in Open Label Extension
Hide Arm/Group Description:
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to placebo treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to low dose colesevelam 1.9 grams per day treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to high dose colesevelam 3.8 grams per day treatment during the Double blind Period.
Open Label Extension was an 18 week open-label treatment period. All participants were treated with 3750 mg colesevelam (high-dose).
Overall Number of Participants Analyzed 62 56 60 178
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
-5.3  (59.1) 19.5  (58.0) 14.2  (64.5) 11.5  (61.8)
11.Secondary Outcome
Title Percent Change in High-density Lipoprotein Cholesterol (HDL-C) From Study Baseline (Day 1) to Week 26.
Hide Description Percent change in high-density lipoprotein cholesterol from baseline to Week 26 was calculated for subpopulations representing each assigned treatment group during the Double blind Period, and for the overall population in the Open Label Extension Period.
Time Frame 26 weeks (week 26 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam All High Dose Colesevelam in Open Label Extension
Hide Arm/Group Description:
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to placebo treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to low dose colesevelam 1.9 grams per day treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to high dose colesevelam 3.8 grams per day treatment during the Double blind Period.
Open Label Extension was an 18 week open-label treatment period. All participants were treated with 3750 mg colesevelam (high-dose).
Overall Number of Participants Analyzed 62 56 60 178
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
6.6  (13.64) 8.5  (20.33) 9.3  (19.37) 8.1  (17.86)
12.Secondary Outcome
Title Percent Change in Non-high-density Lipoprotein Cholesterol From Study Baseline (Day 1) to Week 26.
Hide Description Percent change in non-high-density lipoprotein cholesterol from baseline to Week 26 was calculated for subpopulations representing each assigned treatment group during the Double blind Period, and for the overall population in the Open Label Extension Period.
Time Frame 26 weeks (week 26 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam All High Dose Colesevelam in Open Label Extension
Hide Arm/Group Description:
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to placebo treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to low dose colesevelam 1.9 grams per day treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to high dose colesevelam 3.8 grams per day treatment during the Double blind Period.
Open Label Extension was an 18 week open-label treatment period. All participants were treated with 3750 mg colesevelam (high-dose).
Overall Number of Participants Analyzed 61 56 60 177
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
-10.0  (21.04) -13.2  (19.9) -11.0  (20.80) -11.3  (20.53)
13.Secondary Outcome
Title Percent Change in Apolipoprotein A-I From Study Baseline (Day 1) to Week 26.
Hide Description Percent change in apolipoprotein A-I from baseline to Week 26 was calculated for subpopulations representing each assigned treatment group during the Double blind Period, and for the overall population in the Open Label Extension Period.
Time Frame 26 weeks (week 26 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam All High Dose Colesevelam in Open Label Extension
Hide Arm/Group Description:
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to placebo treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to low dose colesevelam 1.9 grams per day treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to high dose colesevelam 3.8 grams per day treatment during the Double blind Period.
Open Label Extension was an 18 week open-label treatment period. All participants were treated with 3750 mg colesevelam (high-dose).
Overall Number of Participants Analyzed 52 52 57 161
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
4.7  (12.95) 4.9  (14.86) 7.2  (15.34) 5.6  (14.40)
14.Secondary Outcome
Title Percent Change in Apolipoprotein B From Study Baseline (Day 1) to Week 26.
Hide Description Percent change in apolipoprotein B from baseline to Week 26 was calculated for subpopulations representing each assigned treatment group during the Double blind Period, and for the overall population in the Open Label Extension Period.
Time Frame 26 weeks (week 26 - day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Last Observation Carried Forward.
Arm/Group Title Placebo Low Dose Colesevelam High Dose Colesevelam All High Dose Colesevelam in Open Label Extension
Hide Arm/Group Description:
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to placebo treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to low dose colesevelam 1.9 grams per day treatment during the Double blind Period.
A subpopulation of the All High Dose treatment regimen of the Open Label Extension period of participants who were randomized to high dose colesevelam 3.8 grams per day treatment during the Double blind Period.
Open Label Extension was an 18 week open-label treatment period. All participants were treated with 3750 mg colesevelam (high-dose).
Overall Number of Participants Analyzed 52 52 57 161
Mean (Standard Deviation)
Unit of Measure: percent change from baseline
-11.4  (16.86) -11.3  (18.71) -11.2  (17.86) -11.3  (17.72)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Double Blind Period Low Dose Colesevelam Double Blind Period High Dose Colesevelam Double Blind Period High Dose Colesevelam Open Label Extension
Hide Arm/Group Description Placebo taken from day 1 to week 8. Low dose colesevelam 1.9 grams per day taken from day 1 to week 8. High dose colesevelam 3.8 grams per day taken from day 1 to week 8. All participants of the Open Label Extension (weeks 8-26) took colesevelam 3.8 grams per day.
All-Cause Mortality
Placebo Double Blind Period Low Dose Colesevelam Double Blind Period High Dose Colesevelam Double Blind Period High Dose Colesevelam Open Label Extension
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--    
Hide Serious Adverse Events
Placebo Double Blind Period Low Dose Colesevelam Double Blind Period High Dose Colesevelam Double Blind Period High Dose Colesevelam Open Label Extension
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/65 (4.62%)      3/65 (4.62%)      0/64 (0.00%)      0/184 (0.00%)    
Blood and lymphatic system disorders         
idiopathic thrombocytopenic purpura   1/65 (1.54%)  1 0/65 (0.00%)  0 0/64 (0.00%)  0 0/184 (0.00%)  0
Congenital, familial and genetic disorders         
renal hypoplasia   0/65 (0.00%)  0 1/65 (1.54%)  1 0/64 (0.00%)  0 0/184 (0.00%)  0
Gastrointestinal disorders         
gastroesophageal reflux disease   1/65 (1.54%)  1 0/65 (0.00%)  0 0/64 (0.00%)  0 0/184 (0.00%)  0
Injury, poisoning and procedural complications         
poisoning deliberate   1/65 (1.54%)  1 0/65 (0.00%)  0 0/64 (0.00%)  0 0/184 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
nasopharyngeal cancer NOS   0/65 (0.00%)  0 1/65 (1.54%)  1 0/64 (0.00%)  0 0/184 (0.00%)  0
Skin and subcutaneous tissue disorders         
contusion   0/65 (0.00%)  0 1/65 (1.54%)  1 0/64 (0.00%)  0 0/184 (0.00%)  0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 3%
Placebo Double Blind Period Low Dose Colesevelam Double Blind Period High Dose Colesevelam Double Blind Period High Dose Colesevelam Open Label Extension
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   20/65 (30.77%)      25/65 (38.46%)      14/64 (21.88%)      71/184 (38.59%)    
Gastrointestinal disorders         
vomiting NOS   1/65 (1.54%)  2/65 (3.08%)  1/64 (1.56%)  0/184 (0.00%) 
diarrhea NOS   2/65 (3.08%)  0/65 (0.00%)  1/64 (1.56%)  2/184 (1.09%) 
nausea   1/65 (1.54%)  2/65 (3.08%)  0/64 (0.00%)  7/184 (3.80%) 
abdominal pain NOS   0/65 (0.00%)  0/65 (0.00%)  0/64 (0.00%)  6/184 (3.26%) 
General disorders         
fatigue   1/65 (1.54%)  3/65 (4.62%)  2/64 (3.13%)  0/184 (0.00%) 
Infections and infestations         
nasopharyngitis   3/65 (4.62%)  4/65 (6.15%)  4/64 (6.25%)  10/184 (5.43%) 
upper respiratory infections NOS   3/65 (4.62%)  1/65 (1.54%)  1/64 (1.56%)  9/184 (4.89%) 
ear infection NOS   3/65 (4.62%)  1/65 (1.54%)  0/64 (0.00%)  3/184 (1.63%) 
gastrointestinal viral NOS   2/65 (3.08%)  0/65 (0.00%)  0/64 (0.00%)  4/184 (2.17%) 
influenza   0/65 (0.00%)  0/65 (0.00%)  2/64 (3.13%)  7/184 (3.80%) 
Investigations         
blood creatine phosphokinase increased   0/65 (0.00%)  2/65 (3.08%)  1/64 (1.56%)  0/184 (0.00%) 
Musculoskeletal and connective tissue disorders         
myalgia   0/65 (0.00%)  2/65 (3.08%)  0/64 (0.00%)  0/184 (0.00%) 
Nervous system disorders         
headache   2/65 (3.08%)  3/65 (4.62%)  2/64 (3.13%)  14/184 (7.61%) 
dizziness   2/65 (3.08%)  0/65 (0.00%)  0/64 (0.00%)  0/184 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
rhinitis NOS   0/65 (0.00%)  3/65 (4.62%)  0/64 (0.00%)  3/184 (1.63%) 
pharyngolaryngeal pain   0/65 (0.00%)  2/65 (3.08%)  0/64 (0.00%)  6/184 (3.26%) 
Indicates events were collected by systematic assessment
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.
Sponsor reflects the following restrictive language in the Clinical Study Agreements: "If identified by Daiichi Sankyo,Inc. (DSI), any of DSI's confidential information as defined herein shall be deleted…Nothing in this publication section shall be taken as giving DSI any right of editorial control over any publication prepared by the Study Site."
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Howard Kessler
Organization: Daiichi Sankyo
Phone: 732-590-5032
EMail: hmkessler@dsi.com
Layout table for additonal information
Responsible Party: Michael Melino, Daiichi Sankyo
ClinicalTrials.gov Identifier: NCT00145574    
Other Study ID Numbers: WEL-410
First Submitted: September 1, 2005
First Posted: September 5, 2005
Results First Submitted: November 6, 2009
Results First Posted: January 26, 2010
Last Update Posted: April 15, 2010