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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Hypercholesterolemia

Intervention / Treatment
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* Drug: colesevelam HCl
* Drug: placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 10 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: high dose colesevelam<br>colesevelam HCl 3.750 g | Drug: colesevelam HCl<br>* Tablets<br>|
| Experimental: Low dose colesevelam<br>Low dose colesevelam 1.875 g | Drug: colesevelam HCl<br>* Tablets<br>|
| Placebo Comparator: placebo<br>placebo comparator | Drug: placebo<br>* Matching Tablets<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 8 weeks (week 8 - day 1) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 8 weeks (week 8 - day 1) | 
| 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. | 8 weeks (week 8 - day 1) | 
| 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. | 8 weeks (week 8 - day 1) | 
| 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. | 8 weeks (week 8 - day 1) | 
| 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. | 8 weeks (week 8 - day 1) | 
| 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. | 8 weeks (week 8 - day 1) | 
| 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. | 26 weeks (week 26 - day 1) | 
| 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. | 26 weeks (week 26 - day 1) | 
| 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. | 26 weeks (week 26 - day 1) | 
| 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. | 26 weeks (week 26 - day 1) | 
| 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. | 26 weeks (week 26 - day 1) | 
| 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. | 26 weeks (week 26 - day 1) | 
| 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. | 26 weeks (week 26 - day 1) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pediatric, hypercholesterolemia, colesevelam