Atorvastatin + Anakinra for Kawasaki Disease

Overseen By Adriana Tremoulet

Age: < 18

Sex: Any

Trial Phase: Phase < 1

Sponsor: University of California, San Diego

Must not be taking: CYP3A4 drugs

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 6 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines a new combination treatment for Kawasaki disease, a condition that can lead to heart problems in children. Researchers are testing whether atorvastatin (a drug typically used to lower cholesterol) and anakinra (a drug that blocks inflammation) can reduce heart artery inflammation and prevent damage. Children who recently had Kawasaki disease, especially those showing signs of heart artery issues, might be suitable for this study. The goal is to find a better way to protect children's hearts from long-term damage. As an Early Phase 1 trial, this research focuses on understanding how the treatment works in children, offering participants a chance to contribute to groundbreaking medical advancements.

Will I have to stop taking my current medications?

If you are taking a medication that is metabolized by CYP3A4 (like cyclosporine), you will need to stop taking it to participate in this trial.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that both atorvastatin and anakinra are safe and well-tolerated in children with Kawasaki disease. Atorvastatin, commonly used to lower cholesterol, has been safely administered to children with this condition. Anakinra, which reduces inflammation, has also proven safe in similar cases. Together, these treatments reduce inflammation in the heart's blood vessels and prevent further damage. Previous studies indicate that these treatments generally do not cause serious side effects in children with Kawasaki disease. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about the combination of atorvastatin and anakinra for Kawasaki disease because it offers a fresh approach to treatment. Unlike the standard treatments like high-dose aspirin and intravenous immunoglobulin (IVIG), which mainly focus on reducing inflammation, this new combination targets inflammation through two different pathways. Anakinra blocks an inflammatory protein called interleukin-1, while atorvastatin is known to stabilize blood vessels and reduce inflammation. This dual-action approach could offer more comprehensive protection for the heart, potentially leading to better outcomes for children with Kawasaki disease.

What evidence suggests that atorvastatin and anakinra might be an effective treatment for Kawasaki disease?

Research has shown that atorvastatin and anakinra might help treat Kawasaki disease (KD). Atorvastatin, typically used to lower cholesterol, also reduces inflammation, a major issue in KD. Anakinra blocks a protein called interleukin 1 (IL1) that causes inflammation. In animal studies, anakinra prevented damage to the heart's arteries in KD. Both treatments are safe and well-tolerated in children with KD. While more research is needed, these findings suggest that using both medications together, as studied in this trial, may reduce inflammation and damage in the heart's arteries for KD patients. ^{1 3 4 5 6}

Who Is on the Research Team?

Adriana Tremoulet, MD

Principal Investigator

Professor

Are You a Good Fit for This Trial?

This trial is for children with Kawasaki Disease who have developed coronary artery abnormalities, specifically with a Z score of 3 or larger in certain arteries. Children cannot participate if they are taking drugs metabolized by the enzyme CYP3A4, like cyclosporine.

Inclusion Criteria

I have Kawasaki disease with significant heart artery enlargement.

Exclusion Criteria

I am currently taking medication that is processed by the liver.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive combination therapy with atorvastatin and anakinra for coronary artery abnormalities in Kawasaki disease

6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Atorvastatin and Anakinra

Trial Overview The study tests whether atorvastatin and anakinra can reduce inflammation and prevent damage to the coronary arteries in children with acute Kawasaki Disease who already show early signs of arterial issues.

How Is the Trial Designed?

Group I: Atorvastatin and anakinra Experimental Treatment 1 Intervention

Anakinra up to 8 mg/kg/day and atorvastatin at 0.75 mg/kg/day

Who Is Running the Clinical Trial?

University of California, San Diego

Lead Sponsor

Trials

1,215

Recruited

1,593,000+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3,987

Recruited

47,860,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04747847

NCT04747847 | Pilot Study of Atorvastatin and Anakinra in ... Both of these therapies have been demonstrated to be safe and well-tolerated in KD patients. Therefore, we propose to study the effects of combination therapy ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04747847?term=AREA%5BBasicSearch%5D(atorvastatin%20and%20children)&rank=4

Pilot Study of Atorvastatin and Anakinra in Children With ... Both of these therapies have been demonstrated to be safe and well-tolerated in KD patients. Therefore, we propose to study the effects of combination therapy ...

3. clinconnect.io clinconnect.io/trials/NCT04747847

Pilot Study of Atorvastatin and Anakinra in Children Participants will receive the combination of atorvastatin and anakinra, both of which have been shown to be safe for children with Kawasaki disease.

4. ahajournals.org ahajournals.org/doi/10.1161/CIR.0000000000001295

Update on Diagnosis and Management of Kawasaki Disease Additional data have accumulated showing clear benefit of anticoagulation plus antiplatelet therapy in patients with KD with large CAA, but ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6878161/

Phase I/IIa Trial of Atorvastatin in Patients With Acute ... Conclusions: Atorvastatin was safe and well-tolerated in children with acute KD and CAA. A Phase III efficacy trial is warranted in this patient ...

6. clinicaltrials.ucsd.edu clinicaltrials.ucsd.edu/trial/NCT04747847

Atorvastatin and Anakinra in Children With Coronary Artery ... Both of these therapies have been demonstrated to be safe and well-tolerated in KD patients. Therefore, we propose to study the effects of ...