Clinical trial NCT00145574: 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
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hypercholesterolemia
Keywords: Pediatric
hypercholesterolemia
colesevelam
Lead sponsor: Daiichi Sankyo
Sponsor class: INDUSTRY
First posted: 2005-09-05
Enrollment: 194
Sex: ALL
Minimum age (years): 10
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Percent Change in Plasma Low Density Lipoprotein-cholesterol (LDL-C) From Day 1 (Study Baseline) to Week 8.
timeFrame: 8 weeks (week 8 - day 1)
Secondary outcomes:
measure: Percent Change in Plasma Total Cholesterol (TC) From Day 1 (Study Baseline) to Week 8.
timeFrame: 8 weeks (week 8 - day 1)
measure: Percent Change in Plasma Triglycerides (TG) From Day 1 (Study Baseline) to Week 8.
timeFrame: 8 weeks (week 8 - day 1)
measure: Percent Change in Plasma High-density Lipoprotein-cholesterol (HDL-C) From Day 1 (Study Baseline) to Week 8.
timeFrame: 8 weeks (week 8 - day 1)
measure: Percent Change in Plasma Non-high Density Lipoprotein-cholesterol (Non-HDL-C) From Day 1 (Study Baseline) to Week 8.
timeFrame: 8 weeks (week 8 - day 1)
measure: Percent Change in Plasma Apolipoprotien A-I (Apo A-1) From Day 1 (Study Baseline) to Week 8.
timeFrame: 8 weeks (week 8 - day 1)
measure: Percent Change in Plasma Apolipoprotein B (Apo B) From Day 1 (Study Baseline) to Week 8.
timeFrame: 8 weeks (week 8 - day 1)
measure: Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Study Baseline (Day 1) to Week 26.
timeFrame: 26 weeks (week 26 - day 1)
measure: Percent Change in Total Cholesterol From Study Baseline (Day 1) to Week 26.
timeFrame: 26 weeks (week 26 - day 1)
measure: Percent Change in Triglycerides From Study Baseline (Day 1) to Week 26.
timeFrame: 26 weeks (week 26 - day 1)
measure: Percent Change in High-density Lipoprotein Cholesterol (HDL-C) From Study Baseline (Day 1) to Week 26.
timeFrame: 26 weeks (week 26 - day 1)
measure: Percent Change in Non-high-density Lipoprotein Cholesterol From Study Baseline (Day 1) to Week 26.
timeFrame: 26 weeks (week 26 - day 1)
measure: Percent Change in Apolipoprotein A-I From Study Baseline (Day 1) to Week 26.
timeFrame: 26 weeks (week 26 - day 1)
measure: Percent Change in Apolipoprotein B From Study Baseline (Day 1) to Week 26.
timeFrame: 26 weeks (week 26 - day 1)
Other outcomes:
Locations:
city: Washington
country: United States
state: District of Columbia
city: St. Louis
country: United States
state: Missouri
city: Bronx
country: United States
state: New York
city: New Hyde Park
country: United States
state: New York
city: New York
country: United States
state: New York
city: Cincinnati
country: United States
state: Ohio
city: Wexford
country: United States
state: Pennsylvania
city: Salt Lake City
country: United States
state: Utah
city: Camperdown NSW
country: Australia
city: Vienna
country: Austria
city: Vancouver
country: Canada
state: British Columbia
city: Toronto
country: Canada
state: Ontario
city: Chicoutimi
country: Canada
state: Quebec
city: Laval
country: Canada
state: Quebec
city: Stefoy
country: Canada
state: Quebec
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
Study officials:
References:
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.