Kawasaki DiseasePhase 1/2NCT01431105
Pharmacokinetics (PK)/Safety Study of Atorvastatin in Children With Kawasaki Disease and Coronary Artery Abnormalities
A Phase 1/2 study testing Atorvastatin in people with Kawasaki Disease, run by University of California, San Diego at 1 site in United States.
Trial summary
Kawasaki disease (KD) is the leading cause of acquired heart disease in children in the developed world. Despite available treatment, 25% of children in San Diego County appropriately treated for KD develop coronary artery abnormalities that could lead to complications later in life, including heart attack. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Kawasaki Disease
- What is being tested?
- Atorvastatin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2012to Jul 2018
- Phase
- Phase 1/2
- 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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age ≥ 2 years to 17 years old
- Meets clinical criteria for KD according to American Heart Association guidelines (Table 2): Fever (T≥38oC or 100.4oC) ≥ 3 days and ≥ 2 clinical criteria with left anterior descending coronary artery/right coronary artery z-score ≥ 2.5 or an aneurysm (≥ 1.5 x the adjacent segment) of one of the coronary arteries
- Patient presents within the first 20 days after fever onset
- Parent or legal guardian able and willing to provide informed consent and subject willing and able to provide assent when appropriate.
- Post-menarchal females: Negative pregnancy test at screening and willing to use two forms of contraception during the study
- Males engaging in sexual activity that could lead to pregnancy must use a condom.
Who cannot join
- Use of a statin, fibrate, or niacin within the 3 months prior to enrollment
- Have any chronic disease, except asthma, atopic dermatitis, autism or controlled seizure disorder
- Screening creatine phosphokinase (CK) ≥ 3x upper limit of normal for age
- Patient taking a CYP3A4 inhibitor (ie. cyclosporine or clarithromycin) in the last 7 days
- Patient has a history of allergy to atorvastatin or its derivatives
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California San Diego
San Diego, California, 92093
About this study
Official title: Phase I/IIa Study of Pharmacokinetics and Safety of Atorvastatin in Children With Coronary Artery Abnormalities Secondary to Kawasaki Disease
Kawasaki disease (KD) is the leading cause of acquired heart disease in children in the developed world. Despite available treatment, 25% of children in San Diego County appropriately treated for KD develop coronary artery abnormalities that could lead to complications later in life, including heart attack. Although investigators can identify children with KD that have these coronary artery abnormalities, there is no approved additional treatment to decrease coronary artery inflammation and arrest or prevent damage to the coronary arteries. Inflammation and damage to the arterial wall is central to these coronary artery abnormalities. Statins, a class of drugs that is known for lowering cholesterol, have also been shown to decrease inflammation in general as well as at the level of the vessel wall. Therefore, the investigators propose to study the safety of the drug atorvastatin in children with coronary artery abnormalities from KD.
- Conditions
- Kawasaki Disease
- Being tested
- Atorvastatin
- Keywords
- Kawasaki disease
- Medical subject headings
- Mucocutaneous Lymph Node Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AtorvastatinDrug
Also called Lipitor
Atorvastatin dose titration to maximum tolerated dose (once daily for 6 weeks)
| Study group | Role | What they receive |
|---|---|---|
| Atorvasatin | Experimental | Atorvastatin dose titration to maximum tolerated dose Receives: Drug: Atorvastatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With SAE
Number of participants who experienced an SAE within the 6 week study period
Measured: At 6 weeks after initiation of study drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2011 (estimated)
- Study startJul 2012
- Primary completionJan 2018
- Study completionJul 2018
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 Kawasaki DiseaseStudies of the same condition, open and under way.
- More trials run by University of California, San DiegoThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Diego
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Hospital Colorado, University of Colorado, Denver
- Responsible party
- Principal investigator, Jane C. Burns MD, University of California, San Diego
- Investigators
- Jane C Burns, MD, Principal investigator, University of California, San Diego
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01431105
- Sponsor's protocol ID
- KD-Atorvastatin
- Status last verified
- May 2020
- First submitted
- 6 Sep 2011
- Last updated
- 6 May 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
He M, Chen Z, Martin M, Zhang J, Sangwung P, Woo B, Tremoulet AH, Shimizu C, Jain MK, Burns JC, Shyy JY. miR-483 Targeting of CTGF Suppresses Endothelial-to-Mesenchymal Transition: Therapeutic Implications in Kawasaki Disease. Circ Res. 2017 Jan 20;120(2):354-365. doi: 10.1161/CIRCRESAHA.116.310233. Epub 2016 Dec 6.
PubMed 27923814
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/IIa Study of Pharmacokinetics and Safety of Atorvastatin in Children With Coronary Artery Abnormalities Secondary to Kawasaki Disease
- ClinicalTrials.gov ID
- NCT01431105
- Acronym / sponsor ID
- KD-Atorvastatin
- Conditions
- Kawasaki Disease
- Keywords
- Kawasaki disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- University of California, San DiegoUniversity, hospital or other
- Collaborators
- Children's Hospital Colorado, University of Colorado, Denver
- Study start
- Jul 2012
- Primary completion
- Jan 2018 (actual)
- Study completion
- Jul 2018 (actual)
- First posted
- 9 Sep 2011 (estimated)
- Results
- Posted 4 Jun 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Kawasaki disease (KD) is the leading cause of acquired heart disease in children in the developed world. Despite available treatment, 25% of children in San Diego County appropriately treated for KD develop coronary artery abnormalities that could lead to complications later in life, including heart attack. Although investigators can identify children with KD that have these coronary artery abnormalities, there is no approved additional treatment to decrease coronary artery inflammation and arrest or prevent damage to the coronary arteries. Inflammation and damage to the arterial wall is central to these coronary artery abnormalities. Statins, a class of drugs that is known for lowering cholesterol, have also been shown to decrease inflammation in general as well as at the level of the vessel wall. Therefore, the investigators propose to study the safety of the drug atorvastatin in children with coronary artery abnormalities from KD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Atorvasatin | Experimental | Drug: AtorvastatinAtorvastatin dose titration to maximum tolerated dose |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Age ≥ 2 years to 17 years old
- Meets clinical criteria for KD according to American Heart Association guidelines (Table 2): Fever (T≥38oC or 100.4oC) ≥ 3 days and ≥ 2 clinical criteria with left anterior descending coronary artery/right coronary artery z-score ≥ 2.5 or an aneurysm (≥ 1.5 x the adjacent segment) of one of the coronary arteries
- Patient presents within the first 20 days after fever onset
- Parent or legal guardian able and willing to provide informed consent and subject willing and able to provide assent when appropriate.
- Post-menarchal females: Negative pregnancy test at screening and willing to use two forms of contraception during the study
Key exclusion criteria (5 listed)
- Use of a statin, fibrate, or niacin within the 3 months prior to enrollment
- Have any chronic disease, except asthma, atopic dermatitis, autism or controlled seizure disorder
- Screening creatine phosphokinase (CK) ≥ 3x upper limit of normal for age
- Patient taking a CYP3A4 inhibitor (ie. cyclosporine or clarithromycin) in the last 7 days
- Patient has a history of allergy to atorvastatin or its derivatives
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 |
|---|---|---|
| AtorvastatinAlso: LipitorAtorvastatin dose titration to maximum tolerated dose (once daily for 6 weeks) | Drug | Atorvasatin |
Primary outcome
Number of Participants With SAE
Time frame: At 6 weeks after initiation of study drug
Number of participants who experienced an SAE within the 6 week study period
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 27923814He M, Chen Z, Martin M, Zhang J, Sangwung P, Woo B, Tremoulet AH, Shimizu C, Jain MK, Burns JC, Shyy JY. miR-483 Targeting of CTGF Suppresses Endothelial-to-Mesenchymal Transition: Therapeutic Implications in Kawasaki Disease. Circ Res. 2017 Jan 20;120(2):354-365. doi: 10.1161/CIRCRESAHA.116.310233. Epub 2016 Dec 6.
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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.
Jane C Burns, MD principal investigator
University of California, San Diego
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Atorvastatin in people with Kawasaki Disease, run by University of California, San Diego at 1 site in United States.
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 6 inclusion and 5 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 6 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 4 Jun 2019). 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?
Number of Participants With SAE (time frame: At 6 weeks after initiation of study drug). 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 NCT01431105, 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 6 May 2020.