Familial HypercholesterolemiaPhase 3NCT00355615

PLUTO: Pediatric Lipid-redUction Trial of rOsuvastatin

A Phase 3 study testing Rosuvastatin in people with Familial Hypercholesterolemia, run by AstraZeneca at 20 sites in 5 countries.

Completed

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

Official record

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Trial summary

The primary objective of this study is to determine the efficacy of once-daily rosuvastatin in reducing LDL-C in children and adolescents aged 10-17 years with HeFH from baseline (Day 0) to the end of the 12-week double-blind treatment period. Read more about this study

Who is it for?: Ages 10 to 17 years
Conditions the study is for: Familial Hypercholesterolemia
What is being tested?: Rosuvastatin
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 173 participants
Where: 20 sitesin 5 countries
Study dates: Jul 2006to Jul 2008
Phase: Phase 3
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 (at least 1 year post-menarche) children and adolescents (aged 10 -17 years) with heterozygous familial hypercholesterolemia (HeFH)

Who cannot join

Certain medical conditions and lab test results
History of a reaction to rosuvastatin or other statin drugs
Use of specified disallowed medications

Trial locations and contacts

20 sites in 5 countries.

Country

City, postcode or hospital

20 sites in 5 countries

Netherlands7 sites

Research SIte
Amsterdam
Research Site
Eindhoven
Research Site
Groningen
Research Site
Hoorn
Research Site
Rotterdam
Research Site
Utrecht
Research SIte
Waalwijk

Other trials in Netherlands

Canada5 sites

Research Site
Chicoutimi, Quebec
Research Site
Hamilton, Ontario
Research Site
Laval, Quebec
Research Site
Sherbrook, Quebec
Research Site
Toronto, Ontario

Other trials in Canada

United States4 sites

Research Site
Cincinnati, Ohio
Research Site
Hyde Park, New York
Research Site
Los Angeles, California
Research Site
Wexford, Pennsylvania

Other trials in United States

Spain3 sites

Research Site
Córdoba
Research Site
Madrid
Research Site
Reus

Other trials in Spain

Norway1 site

Research Site
Oslo

Other trials in Norway

About this study

Official title: A Phase IIIb, Efficacy, and Safety Study of Rosuvastatin in Children 10-17 Years of Age With Heterozygous Familial Hypercholesterolemia: a 12-week, Double-blind, Randomized, Multicenter, Placebo-controlled Study With a 40-week, Open-label, Follow-up

Conditions: Familial Hypercholesterolemia
Being tested: Rosuvastatin, Placebo
Keywords: Heterozygous Familial Hypercholesterolemia
Medical subject headings: Hyperlipoproteinemia Type II

What participants receive

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

RosuvastatinDrug
Also called Nexium
oral
PlaceboDrug
oral

Study groups in NCT00355615
Study group	Role	What they receive
rosuva 5	Active comparator	rosuvastatin 5 mg Receives: Drug: Rosuvastatin
rosuva 10	Active comparator	rosuvastatin 10 mg Receives: Drug: Rosuvastatin
rosuva 20	Active comparator	rosuvastatin 20 mg Receives: Drug: Rosuvastatin
Placebo	Placebo comparator	Placebo Receives: Drug: Placebo
rosuva ol	Other	rosuvastatin open label Receives: Drug: Rosuvastatin

What is being measured

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

Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline (Day 0) to the End of the 12-week Double-blind Treatment Phase
Percent change in low-density lipoprotein cholesterol (LDL-C) = (final value - Baseline value)/Baseline value * 100
Measured: 12 weeks

Secondary outcomes (12)

Percent Change in LDL-C and Other Lipid Parameters From Baseline to Week 6, and at End of Double-blind Dose Treatment Phase (Week 12)
Percent change from baseline in LDL-C after six week of treatment
Measured: 6 weeks
Percent Control Rate Based on Achievement of LDL-C Target of <110 mg/dL During Double-blind Dose Treatment
Percent of patients achieving LDL-C < 110 mg/dL out of the total patients in each treatment group
Measured: 12 weeks
Percent Change in HDL-C
Percent change in high-density lipoprotein cholesterol (HDL-C) after 12 weeks of treatment
Measured: After 12 weeks of treatment
Percent Change in Non-HDL-C at 12 Weeks
Percent change in non-HDL-C at 12 weeks
Measured: After 12 weeks of treatment
Percent Change in Triglycerides (TG)
Percent change in tryglycerides (TG) after 12 weeks of treatment
Measured: After 12 weeks of treatment
Percent Change in Total Cholesterol (TC)
Percent change from baseline in total cholesteral after 12 weeks of treatment
Measured: After 12 weeks of treatment
Percent Change in Apolipoprotein A-1 (ApoA-1)
Percent change in ApoA-1 after 12 weeks of treatment
Measured: After 12 weeks of treatment
Percent Change in Apolipoprotein B (ApoB)
Percent change in ApoB after 12 weeks of treatment
Measured: After 12 weeks of treatment
Percent Change in ApoB/ApoA-1
Percent change in the ratio of ApoB/ApoA-1 after 12 weeks of treatment
Measured: After 12 weeks of treatment
Percent Change in LDL-C/HDL-C
Percent change in the ratio of LDL-C/HDL-C after 12 weeks of treatment
Measured: After 12 week of treatment
Percent Change in TC/HDL-C
Percent change in the ratio of TC/HDL-C after 12 weeks of treatment
Measured: After 12 weeks of treatment
Percent Change in Non-HDL-C/HDL-C
Percent change in the ratio of non-HDL-C/HDL-C after 12 weeks of treatment
Measured: After 12 weeks of treatment

Timeline

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

Jul 2006Jul 2008

First posted24 Jul 2006 (estimated)
Study startJul 2006
Primary completionJul 2008
Study completionJul 2008

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 Familial HypercholesterolemiaStudies of the same condition, open and under way.
More trials run by AstraZenecaThe 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 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 173 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00355615
Sponsor's protocol ID: D3561C00087
Other IDs: PLUTO
Status last verified: Aug 2011
First submitted: 20 Jul 2006
Last updated: 31 Aug 2011
Results posted: Yes, on ClinicalTrials.gov

Publications

Avis HJ, Hutten BA, Gagne C, Langslet G, McCrindle BW, Wiegman A, Hsia J, Kastelein JJ, Stein EA. Efficacy and safety of rosuvastatin therapy for children with familial hypercholesterolemia. J Am Coll Cardiol. 2010 Mar 16;55(11):1121-6. doi: 10.1016/j.jacc.2009.10.042.
PubMed 20223367

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
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
Not yet recruitingObservational study
An Observational Study to Evaluate Treatment Patterns in High, Very High and Extreme Cardiovascular Risk Patients With Hypercholesterolemia, Including Familial Hypercholesterolemia
Hypercholesterolaemia, Familial Hypercholesterolaemia
Active, not recruitingObservational study
Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia (HoFH), Major Adverse Cardiovascular Events (MACE)
Active, not recruitingObservational study
Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia

All Familial Hypercholesterolemia trials

Study at a glance

From the registry record, last updated by the sponsor on 31 Aug 2011. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase IIIb, Efficacy, and Safety Study of Rosuvastatin in Children 10-17 Years of Age With Heterozygous Familial Hypercholesterolemia: a 12-week, Double-blind, Randomized, Multicenter, Placebo-controlled Study With a 40-week, Open-label, Follow-up
ClinicalTrials.gov ID: NCT00355615
Acronym / sponsor ID: D3561C00087
Conditions: Familial Hypercholesterolemia
Keywords: Heterozygous Familial Hypercholesterolemia
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 173 participants (actual)
Lead sponsor: AstraZenecaIndustry (pharma, biotech, devices)
Study start: Jul 2006
Primary completion: Jul 2008 (actual)
Study completion: Jul 2008 (actual)
First posted: 24 Jul 2006 (estimated)
Results: Posted 20 Jul 2009

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: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 5

Arm	Type	Receives
rosuva 5	Active comparator	Drug: Rosuvastatinrosuvastatin 5 mg
rosuva 10	Active comparator	Drug: Rosuvastatinrosuvastatin 10 mg
rosuva 20	Active comparator	Drug: Rosuvastatinrosuvastatin 20 mg
Placebo	Placebo comparator	Drug: PlaceboPlacebo
rosuva ol	Other	Drug: Rosuvastatinrosuvastatin open label

Who may qualify

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

Key inclusion criteria (1 listed)

Male or female (at least 1 year post-menarche) children and adolescents (aged 10 -17 years) with heterozygous familial hypercholesterolemia (HeFH)

Key exclusion criteria (3 listed)

Certain medical conditions and lab test results
History of a reaction to rosuvastatin or other statin drugs
Use of specified disallowed medications

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
RosuvastatinAlso: Nexiumoral	Drug	rosuva 10, rosuva 20, rosuva 5, rosuva ol
Placebooral	Drug	Placebo

Primary outcome

Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline (Day 0) to the End of the 12-week Double-blind Treatment Phase
Time frame: 12 weeks
Percent change in low-density lipoprotein cholesterol (LDL-C) = (final value - Baseline value)/Baseline value * 100

12 secondary outcomes

Percent Change in LDL-C and Other Lipid Parameters From Baseline to Week 6, and at End of Double-blind Dose Treatment Phase (Week 12)
Time frame: 6 weeks
Percent Control Rate Based on Achievement of LDL-C Target of <110 mg/dL During Double-blind Dose Treatment
Time frame: 12 weeks
Percent Change in HDL-C
Time frame: After 12 weeks of treatment
Percent Change in Non-HDL-C at 12 Weeks
Time frame: After 12 weeks of treatment
Percent Change in Triglycerides (TG)
Time frame: After 12 weeks of treatment
Percent Change in Total Cholesterol (TC)
Time frame: After 12 weeks of treatment
Percent Change in Apolipoprotein A-1 (ApoA-1)
Time frame: After 12 weeks of treatment
Percent Change in Apolipoprotein B (ApoB)
Time frame: After 12 weeks of treatment
Percent Change in ApoB/ApoA-1
Time frame: After 12 weeks of treatment
Percent Change in LDL-C/HDL-C
Time frame: After 12 week of treatment
Percent Change in TC/HDL-C
Time frame: After 12 weeks of treatment
Percent Change in Non-HDL-C/HDL-C
Time frame: After 12 weeks of treatment

Results and safety

Results posted to the registry on 20 Jul 2009, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Rosuva 5	42	41
Rosuva 10	44	44
Rosuva 20	45	44
Placebo	46	45
Rosuva ol	0	0

Primary outcome: Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline (Day 0) to the End of the 12-week Double-blind Treatment Phase

Time frame: 12 weeks. Mean (Standard Deviation). Unit: percentage

Measure	Rosuva 5 n = 42	Rosuva 10 n = 44	Rosuva 20 n = 44	Placebo n = 46
Value	-38.5 (11.38)	-44.4 (12.15)	-50.2 (13.3)	-0.5 (13.18)

Population analysed: Intention-to-treat (ITT) analysis set included all randomized patients who took study medication and had both a baseline reading and at least 1 post-baseline reading for the variable being analyzed. Analyses were performed using the last-observation-carried-forward (LOCF) method on the ITT analysis set for all efficacy outcome variables.

12 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Rosuva 5	0 of ?	12 of ?	Not reported
Rosuva 10	0 of ?	14 of ?	Not reported
Rosuva 20	0 of ?	17 of ?	Not reported
Placebo	1 of ?	17 of ?	Not reported
Rosuva ol	2 of ?	75 of ?	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2011.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 20223367Avis HJ, Hutten BA, Gagne C, Langslet G, McCrindle BW, Wiegman A, Hsia J, Kastelein JJ, Stein EA. Efficacy and safety of rosuvastatin therapy for children with familial hypercholesterolemia. J Am Coll Cardiol. 2010 Mar 16;55(11):1121-6. doi: 10.1016/j.jacc.2009.10.042.

Related trials for Familial Hypercholesterolemia

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
Not yet recruitingObservational study
An Observational Study to Evaluate Treatment Patterns in High, Very High and Extreme Cardiovascular Risk Patients With Hypercholesterolemia, Including Familial Hypercholesterolemia
Hypercholesterolaemia, Familial Hypercholesterolaemia
Active, not recruitingObservational study
Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia (HoFH), Major Adverse Cardiovascular Events (MACE)
Active, not recruitingObservational study
Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia
Heterozygous 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.

Crestor Medical Science Director, MD study director

AstraZeneca

20 sites in 5 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Rosuvastatin in people with Familial Hypercholesterolemia, run by AstraZeneca at 20 sites in 5 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 1 inclusion and 3 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 31 Aug 2011. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites in Netherlands, Canada, United States, Spain, Norway. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 20 Jul 2009). 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 Low-density Lipoprotein Cholesterol (LDL-C) From Baseline (Day 0) to the End of the 12-week Double-blind Treatment Phase (time frame: 12 weeks). 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 NCT00355615, 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 31 Aug 2011.