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”

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.

Intervention(s) in this Clinical Trial

• Drug: colesevelam HCl
  • Tablets
• Drug: placebo
  • Matching Tablets

Arms, Groups and Cohorts in this Clinical Trial

• Experimental: high dose colesevelam
  • colesevelam HCl 3.750 g
• Experimental: Low dose colesevelam
  • Low dose colesevelam 1.875 g
• Placebo Comparator: placebo
  • placebo comparator

Primary Measures

• Percent Change in Plasma Low Density Lipoprotein-cholesterol (LDL-C) From Day 1 (Study Baseline) to Week 8.
  • Time Frame: 8 weeks (week 8 - day 1)
    Safety Issue?: No

Secondary Measures

• Percent Change in Plasma Total Cholesterol (TC) From Day 1 (Study Baseline) to Week 8.
  • Time Frame: 8 weeks (week 8 - day 1)
    Safety Issue?: No
• Percent Change in Plasma Triglycerides (TG) From Day 1 (Study Baseline) to Week 8.
  • Time Frame: 8 weeks (week 8 - day 1)
    Safety Issue?: No
• Percent Change in Plasma High-density Lipoprotein-cholesterol (HDL-C) From Day 1 (Study Baseline) to Week 8.
  • Time Frame: 8 weeks (week 8 - day 1)
    Safety Issue?: No
• Percent Change in Plasma Non-high Density Lipoprotein-cholesterol (Non-HDL-C) From Day 1 (Study Baseline) to Week 8.
  • Time Frame: 8 weeks (week 8 - day 1)
    Safety Issue?: No
• Percent Change in Plasma Apolipoprotien A-I (Apo A-1) From Day 1 (Study Baseline) to Week 8.
  • Time Frame: 8 weeks (week 8 - day 1)
    Safety Issue?: No
• Percent Change in Plasma Apolipoprotein B (Apo B) From Day 1 (Study Baseline) to Week 8.
  • Time Frame: 8 weeks (week 8 - day 1)
    Safety Issue?: No
• Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Study Baseline (Day 1) to Week 26.
  • Time Frame: 26 weeks (week 26 - day 1)
    Safety Issue?: No
• Percent Change in Total Cholesterol From Study Baseline (Day 1) to Week 26.
  • Time Frame: 26 weeks (week 26 - day 1)
    Safety Issue?: No
• Percent Change in Triglycerides From Study Baseline (Day 1) to Week 26.
  • Time Frame: 26 weeks (week 26 - day 1)
    Safety Issue?: No
• Percent Change in High-density Lipoprotein Cholesterol (HDL-C) From Study Baseline (Day 1) to Week 26.
  • Time Frame: 26 weeks (week 26 - day 1)
    Safety Issue?: No
• Percent Change in Non-high-density Lipoprotein Cholesterol From Study Baseline (Day 1) to Week 26.
  • Time Frame: 26 weeks (week 26 - day 1)
    Safety Issue?: No
• Percent Change in Apolipoprotein A-I From Study Baseline (Day 1) to Week 26.
  • Time Frame: 26 weeks (week 26 - day 1)
    Safety Issue?: No
• Percent Change in Apolipoprotein B From Study Baseline (Day 1) to Week 26.
  • Time Frame: 26 weeks (week 26 - day 1)
    Safety Issue?: No

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

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: 10 Years

Maximum Age for this Clinical Trial: 17 Years

Are Healthy Volunteers Accepted for this Clinical Trial?: No

Lead Investigator: Daiichi Sankyo Inc. Industry

Information obtained from ClinicalTrials.gov on February 12, 2012

Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00145574

Study ID Number: WEL-410

ClinicalTrials.gov Identifier: NCT00145574

Health Authority: United States: Food and Drug Administration