XXXX : efficacy and safety in pediatric subjects with heterozygous @entity753 .

@entity1 @entity35277 @entity753 @entity1674 @entity755 @entity1675 @entity5209 @entity165
OBJECTIVE: Evaluate the efficacy and safety of colesevelam @entity5209 in @entity1 with heterozygous @entity753 (heFH). STUDY DESIGN: This was a randomized, double-blind, 41-site study in 194 @entity1 aged 10 to 17 years (inclusive) with heFH (statin-na  ve or on a stable statin regimen). After a 4-week stabilization period (period I), subjects were randomized 1:1:1 to placebo, colesevelam 1.875 g/d, or colesevelam 3.75 g/d for 8 weeks (period II). All then received open-label colesevelam 3.75 g/d for 18 weeks (period III), with follow-up 2 weeks later. The primary endpoint was percent change in low-density lipoprotein (LDL)- @entity165 from baseline to week 8. Secondary endpoints included percent change in other lipoprotein variables, including non-high-density lipoprotein (non-HDL)- @entity165 . Adverse events were also evaluated. RESULTS: At week 8, a significant difference from baseline in LDL- @entity165 was reported with colesevelam 1.875 g/d (-6.3%; P = .031) and colesevelam 3.75 g/d (-12.5%; P < .001) compared with placebo. Significant treatment effects were also reported for total @entity165 (-7.4%), non-HDL- @entity165 (-10.9%), HDL- @entity165 (+6.1%), @entity1675 (+6.9%), and @entity755 (-8.3%) and a nonsignificant effect for @entity1674 (+5.1%) with colesevelam 3.75 g/d compared with placebo at week 8. These treatment effects were maintained during period III. CONCLUSIONS: @entity35277 significantly lowered LDL- @entity165 levels in @entity1 with heFH.
