HypercholesterolemiaPhase 4NCT00145574

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

A Phase 4 study testing colesevelam HCl in people with Hypercholesterolemia, run by Daiichi Sankyo at 25 sites in 8 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial 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… Read more about this study

Who is it for?: Ages 10 to 17 years
Conditions the study is for: Hypercholesterolemia
What is being tested?: colesevelam HCl
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 194 participants
Where: 25 sitesin 8 countries
Study dates: Nov 2005to Dec 2007
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 10 to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

25 sites in 8 countries.

Country

City, postcode or hospital

25 sites in 8 countries

United States8 sites

Study site
Cincinnati, Ohio
Study site
New Hyde Park, New York
Study site
New York, New York
Study site
Salt Lake City, Utah
Study site
St Louis, Missouri
Study site
The Bronx, New York
Study site
Washington D.C., District of Columbia
Study site
Wexford, Pennsylvania

Other trials in United States

Canada5 sites

Study site
Chicoutimi, Quebec
Study site
Laval, Quebec
Study site
Stefoy, Quebec
Study site
Toronto, Ontario
Study site
Vancouver, British Columbia

Other trials in Canada

Israel4 sites

Study site
Holon
Study site
Jerusalem
Study site
Kefer Saba
Study site
Tel HaShomer

Other trials in Israel

South Africa3 sites

Study site
Observatory
Study site
Pretoria
Study site
Tygerberg

Other trials in South Africa

Netherlands2 sites

Study site
Amsterdam
Study site
Rotterdam

Other trials in Netherlands

Australia1 site

Study site
Camperdown NSW

Other trials in Australia

Austria1 site

Study site
Vienna

Other trials in Austria

Norway1 site

Study site
Oslo

Other trials in Norway

About this study

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

Conditions: Hypercholesterolemia
Being tested: colesevelam HCl, placebo
Keywords: Pediatric, hypercholesterolemia, colesevelam
Medical subject headings: Hypercholesterolemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

colesevelam HClDrug
Tablets
placeboDrug
Matching Tablets

Study groups in NCT00145574
Study group	Role	What they receive
high dose colesevelam	Experimental	colesevelam HCl 3.750 g Receives: Drug: colesevelam HCl
Low dose colesevelam	Experimental	Low dose colesevelam 1.875 g Receives: Drug: colesevelam HCl
placebo	Placebo comparator	placebo comparator Receives: Drug: placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 8 weeks (week 8 - day 1)

Secondary outcomes (13)

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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 26 weeks (week 26 - day 1)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Nov 2005Dec 2007

First posted5 Sep 2005 (estimated)
Study startNov 2005
Primary completionDec 2007
Study completionDec 2007

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for HypercholesterolemiaStudies of the same condition, open and under way.
More trials run by Daiichi SankyoThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 194 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00145574
Sponsor's protocol ID: WEL-410
Status last verified: Apr 2010
First submitted: 1 Sep 2005
Last updated: 15 Apr 2010
Results posted: Yes, on ClinicalTrials.gov

Publications

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.
PubMed 19879596

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
Study Evaluating LDL-C Change and Adherence to Inclisiran Lipid-lowering Therapy in ASCVD
Primary Hypercholesterolemia, Mixed Dyslipidemia
Active, not recruitingPhase 3
A Study of Inclisiran to Prevent Cardiovascular Events in High-risk Primary Prevention Patients.
Primary Prevention of Atherosclerotic Cardiovascular Disease
RecruitingObservational study
Causes and Natural History of Dyslipidemias
Hypercholesterolemia, Atherosclerosis
Active, not recruitingObservational study
Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
Familial Hypercholesterolaemia
Not yet recruiting
Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia

All Hypercholesterolemia trials

Study at a glance

From the registry record, last updated by the sponsor on 15 Apr 2010. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT00145574
Acronym / sponsor ID: WEL-410
Conditions: Hypercholesterolemia
Keywords: Pediatric, hypercholesterolemia, colesevelam
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 194 participants (actual)
Lead sponsor: Daiichi SankyoIndustry (pharma, biotech, devices)
Study start: Nov 2005
Primary completion: Dec 2007 (actual)
Study completion: Dec 2007 (actual)
First posted: 5 Sep 2005 (estimated)
Results: Posted 26 Jan 2010

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 3

Arm	Type	Receives
high dose colesevelam	Experimental	Drug: colesevelam HClcolesevelam HCl 3.750 g
Low dose colesevelam	Experimental	Drug: colesevelam HClLow dose colesevelam 1.875 g
placebo	Placebo comparator	Drug: placeboplacebo comparator

Who may qualify

Ages: 10 to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (5 listed)

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

Key exclusion criteria (1 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
colesevelam HClTablets	Drug	Low dose colesevelam, high dose colesevelam
placeboMatching Tablets	Drug	placebo

Primary outcome

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)
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.

13 secondary outcomes

Percent Change in Plasma Total Cholesterol (TC) From Day 1 (Study Baseline) to Week 8.
Time frame: 8 weeks (week 8 - day 1)
Percent Change in Plasma Triglycerides (TG) From Day 1 (Study Baseline) to Week 8.
Time frame: 8 weeks (week 8 - day 1)
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)
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)
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)
Percent Change in Plasma Apolipoprotein B (Apo B) From Day 1 (Study Baseline) to Week 8.
Time frame: 8 weeks (week 8 - day 1)
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)
Percent Change in Total Cholesterol From Study Baseline (Day 1) to Week 26.
Time frame: 26 weeks (week 26 - day 1)
Percent Change in Triglycerides From Study Baseline (Day 1) to Week 26.
Time frame: 26 weeks (week 26 - day 1)
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)
Percent Change in Non-high-density Lipoprotein Cholesterol From Study Baseline (Day 1) to Week 26.
Time frame: 26 weeks (week 26 - day 1)
Percent Change in Apolipoprotein A-I From Study Baseline (Day 1) to Week 26.
Time frame: 26 weeks (week 26 - day 1)
Percent Change in Apolipoprotein B From Study Baseline (Day 1) to Week 26.
Time frame: 26 weeks (week 26 - day 1)

Results and safety

Results posted to the registry on 26 Jan 2010, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	65	64
Low Dose Colesevelam	65	60
High Dose Colesevelam	64	62

Primary outcome: 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). Mean (Standard Deviation). Unit: percent change from baseline

Measure	Placebo n = 65	Low Dose Colesevelam n = 63	High Dose Colesevelam n = 63
Value	2.9 (16.46)	-3.7 (18.36)	-10.6 (19.36)

Placebo vs Low Dose Colesevelam: p = 0.1122, ANCOVA
Placebo vs High Dose Colesevelam: p <0.0001, ANCOVA

Population analysed: Intent-to-Treat Population for Double blind Period. Last Observation Carried Forward.

13 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 3% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo Double Blind Period	3 of 65 (4.6%)	20 of 65 (30.8%)	Not reported
Low Dose Colesevelam Double Blind Period	3 of 65 (4.6%)	25 of 65 (38.5%)	Not reported
High Dose Colesevelam Double Blind Period	0 of 64 (0%)	14 of 64 (21.9%)	Not reported
High Dose Colesevelam Open Label Extension	0 of 184 (0%)	71 of 184 (38.6%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2010.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 19879596Stein 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.

Related trials for Hypercholesterolemia

Active, not recruitingObservational study
Study Evaluating LDL-C Change and Adherence to Inclisiran Lipid-lowering Therapy in ASCVD
Primary Hypercholesterolemia, Mixed Dyslipidemia
Active, not recruitingPhase 3
A Study of Inclisiran to Prevent Cardiovascular Events in High-risk Primary Prevention Patients.
Primary Prevention of Atherosclerotic Cardiovascular Disease
RecruitingObservational study
Causes and Natural History of Dyslipidemias
Hypercholesterolemia, Atherosclerosis
Active, not recruitingObservational study
Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
Familial Hypercholesterolaemia
Not yet recruiting
Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

25 sites in 8 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing colesevelam HCl in people with Hypercholesterolemia, run by Daiichi Sankyo at 25 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Apr 2010. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 25 sites in United States, Canada, Israel, South Africa, Netherlands and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Jan 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00145574, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2010.