search
Back to results

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

Primary Purpose

Hypercholesterolemia

Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
colesevelam HCl
placebo
Sponsored by
Daiichi Sankyo, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hypercholesterolemia focused on measuring Pediatric, hypercholesterolemia, colesevelam

Eligibility Criteria

10 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Male or female patients Ages 10 to 17 years inclusive Diagnosis of heterozygous familial hypercholesterolemia On a stable dose of statin monotherapy or are treatment naive to lipid- lowering agents On a low-cholesterol diet Exclusion Criteria: Patients should not have serious concomitant conditions that could interfere with the analysis of the results or that could interfere with the well-being of the patients

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

high dose colesevelam

Low dose colesevelam

placebo

Arm Description

colesevelam HCl 3.750 g

Low dose colesevelam 1.875 g

placebo comparator

Outcomes

Primary Outcome Measures

Percent Change in Plasma Low Density Lipoprotein-cholesterol (LDL-C) From Day 1 (Study Baseline) to Week 8.
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.

Secondary Outcome Measures

Percent Change in Plasma Total Cholesterol (TC) From Day 1 (Study Baseline) to Week 8.
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.
Percent Change in Plasma Triglycerides (TG) From Day 1 (Study Baseline) to Week 8.
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.
Percent Change in Plasma High-density Lipoprotein-cholesterol (HDL-C) From Day 1 (Study Baseline) to Week 8.
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.
Percent Change in Plasma Non-high Density Lipoprotein-cholesterol (Non-HDL-C) From Day 1 (Study Baseline) to Week 8.
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.
Percent Change in Plasma Apolipoprotien A-I (Apo A-1) From Day 1 (Study Baseline) to Week 8.
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.
Percent Change in Plasma Apolipoprotein B (Apo B) From Day 1 (Study Baseline) to Week 8.
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.
Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Study Baseline (Day 1) to Week 26.
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.
Percent Change in Total Cholesterol From Study Baseline (Day 1) to Week 26.
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.
Percent Change in Triglycerides From Study Baseline (Day 1) to Week 26.
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.
Percent Change in High-density Lipoprotein Cholesterol (HDL-C) From Study Baseline (Day 1) to Week 26.
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.
Percent Change in Non-high-density Lipoprotein Cholesterol From Study Baseline (Day 1) to Week 26.
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.
Percent Change in Apolipoprotein A-I From Study Baseline (Day 1) to Week 26.
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.
Percent Change in Apolipoprotein B From Study Baseline (Day 1) to Week 26.
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.

Full Information

First Posted
September 1, 2005
Last Updated
April 8, 2010
Sponsor
Daiichi Sankyo, Inc.
search

1. Study Identification

Unique Protocol Identification Number
NCT00145574
Brief Title
Efficacy and Safety of Colesevelam in Pediatric Patients With Genetic High Cholesterol
Official Title
Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Colesevelam HCl Administered to Pediatric Patients With Heterozygous Familial Hypercholesterolemia on a Stable Dose of Statins or Treatment Naive to Lipid-lowering Therapy
Study Type
Interventional

2. Study Status

Record Verification Date
April 2010
Overall Recruitment Status
Completed
Study Start Date
November 2005 (undefined)
Primary Completion Date
December 2007 (Actual)
Study Completion Date
December 2007 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Daiichi Sankyo, Inc.

4. Oversight

5. Study Description

Brief Summary
This study will evaluate the lipid-lowering effect and safety of colesevelam therapy administered to heterozygous familial pediatric patients 10 through 17 years of age who are on a stable dose of a pediatric-approved statin monotherapy (atorvastatin, lovastatin, simvastatin or pravastatin), or who are treatment naive to lipid-lowering therapy.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hypercholesterolemia
Keywords
Pediatric, hypercholesterolemia, colesevelam

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
194 (Actual)

8. Arms, Groups, and Interventions

Arm Title
high dose colesevelam
Arm Type
Experimental
Arm Description
colesevelam HCl 3.750 g
Arm Title
Low dose colesevelam
Arm Type
Experimental
Arm Description
Low dose colesevelam 1.875 g
Arm Title
placebo
Arm Type
Placebo Comparator
Arm Description
placebo comparator
Intervention Type
Drug
Intervention Name(s)
colesevelam HCl
Intervention Description
Tablets
Intervention Type
Drug
Intervention Name(s)
placebo
Intervention Description
Matching Tablets
Primary Outcome Measure Information:
Title
Percent Change in Plasma Low Density Lipoprotein-cholesterol (LDL-C) From Day 1 (Study Baseline) to Week 8.
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)
Secondary Outcome Measure Information:
Title
Percent Change in Plasma Total Cholesterol (TC) From Day 1 (Study Baseline) to Week 8.
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)
Title
Percent Change in Plasma Triglycerides (TG) From Day 1 (Study Baseline) to Week 8.
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)
Title
Percent Change in Plasma High-density Lipoprotein-cholesterol (HDL-C) From Day 1 (Study Baseline) to Week 8.
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)
Title
Percent Change in Plasma Non-high Density Lipoprotein-cholesterol (Non-HDL-C) From Day 1 (Study Baseline) to Week 8.
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)
Title
Percent Change in Plasma Apolipoprotien A-I (Apo A-1) From Day 1 (Study Baseline) to Week 8.
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)
Title
Percent Change in Plasma Apolipoprotein B (Apo B) From Day 1 (Study Baseline) to Week 8.
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)
Title
Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Study Baseline (Day 1) to Week 26.
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)
Title
Percent Change in Total Cholesterol From Study Baseline (Day 1) to Week 26.
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)
Title
Percent Change in Triglycerides From Study Baseline (Day 1) to Week 26.
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)
Title
Percent Change in High-density Lipoprotein Cholesterol (HDL-C) From Study Baseline (Day 1) to Week 26.
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)
Title
Percent Change in Non-high-density Lipoprotein Cholesterol From Study Baseline (Day 1) to Week 26.
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)
Title
Percent Change in Apolipoprotein A-I From Study Baseline (Day 1) to Week 26.
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)
Title
Percent Change in Apolipoprotein B From Study Baseline (Day 1) to Week 26.
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)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female patients Ages 10 to 17 years inclusive Diagnosis of heterozygous familial hypercholesterolemia On a stable dose of statin monotherapy or are treatment naive to lipid- lowering agents On a low-cholesterol diet Exclusion Criteria: Patients should not have serious concomitant conditions that could interfere with the analysis of the results or that could interfere with the well-being of the patients
Facility Information:
City
Washington
State/Province
District of Columbia
Country
United States
City
St. Louis
State/Province
Missouri
Country
United States
City
Bronx
State/Province
New York
Country
United States
City
New Hyde Park
State/Province
New York
Country
United States
City
New York
State/Province
New York
Country
United States
City
Cincinnati
State/Province
Ohio
Country
United States
City
Wexford
State/Province
Pennsylvania
Country
United States
City
Salt Lake City
State/Province
Utah
Country
United States
City
Camperdown NSW
Country
Australia
City
Vienna
Country
Austria
City
Vancouver
State/Province
British Columbia
Country
Canada
City
Toronto
State/Province
Ontario
Country
Canada
City
Chicoutimi
State/Province
Quebec
Country
Canada
City
Laval
State/Province
Quebec
Country
Canada
City
Stefoy
State/Province
Quebec
Country
Canada
City
Holon
Country
Israel
City
Jerusalem
Country
Israel
City
Kefer Saba
Country
Israel
City
Tel-Hashomer
Country
Israel
City
Amsterdam
Country
Netherlands
City
Rotterdam
Country
Netherlands
City
Oslo
Country
Norway
City
Observatory
Country
South Africa
City
Pretoria
Country
South Africa
City
Tygerberg
Country
South Africa

12. IPD Sharing Statement

Citations:
PubMed Identifier
19879596
Citation
Stein EA, Marais AD, Szamosi T, Raal FJ, Schurr D, Urbina EM, Hopkins PN, Karki S, Xu J, Misir S, Melino M. Colesevelam hydrochloride: efficacy and safety in pediatric subjects with heterozygous familial hypercholesterolemia. J Pediatr. 2010 Feb;156(2):231-6.e1-3. doi: 10.1016/j.jpeds.2009.08.037. Epub 2009 Oct 31.
Results Reference
derived

Learn more about this trial

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

We'll reach out to this number within 24 hrs