MedPath

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

Phase 4
Completed
Conditions
Hypercholesterolemia
Interventions
Registration Number
NCT00145574
Lead Sponsor
Daiichi Sankyo
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
194
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
high dose colesevelamcolesevelam HClcolesevelam HCl 3.750 g
Low dose colesevelamcolesevelam HClLow dose colesevelam 1.875 g
placeboplaceboplacebo comparator
Primary Outcome Measures
NameTimeMethod
Percent Change in Plasma Low Density Lipoprotein-cholesterol (LDL-C) From Day 1 (Study Baseline) to Week 8.8 weeks (week 8 - day 1)

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
NameTimeMethod
Percent Change in Plasma Total Cholesterol (TC) From Day 1 (Study Baseline) to Week 8.8 weeks (week 8 - day 1)

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 8.8 weeks (week 8 - day 1)

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 8.8 weeks (week 8 - day 1)

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 8.8 weeks (week 8 - day 1)

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 8.8 weeks (week 8 - day 1)

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 8.8 weeks (week 8 - day 1)

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 26.26 weeks (week 26 - day 1)

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 26.26 weeks (week 26 - day 1)

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 26.26 weeks (week 26 - day 1)

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 26.26 weeks (week 26 - day 1)

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 26.26 weeks (week 26 - day 1)

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 26.26 weeks (week 26 - day 1)

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 26.26 weeks (week 26 - day 1)

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.

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