Rilonacept for Cardiac Sarcoidosis

Recruiting at 1 trial location

Overseen By Lezlie Peterson, R.N.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Mayo Clinic

Must not be taking: TNF inhibitors, Anakinra, others

Disqualifiers: Pregnancy, TB, Cancer, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 12

Duration: 24 weeks

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called rilonacept to determine its effectiveness in reducing heart inflammation in individuals with cardiac sarcoidosis, a condition where clusters of immune cells cause heart problems. Participants will receive either standard treatment or standard treatment plus rilonacept to compare the effects. It suits adults diagnosed with cardiac sarcoidosis who continue to experience heart inflammation despite current treatments. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant medical advancements.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it does mention that you cannot start new immunosuppressive drugs or increase the dose of existing ones close to the trial's start. It's best to discuss your specific medications with the trial team.

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

Research has shown that rilonacept has been used safely in other conditions. For example, in studies involving people with recurrent pericarditis (a type of heart inflammation), rilonacept reduced pain and inflammation without major safety concerns. This suggests the treatment might be well-tolerated.

For cardiac sarcoidosis, research is ongoing to better understand rilonacept's safety. Since the trial is in Phase 2, the treatment has already demonstrated some safety in earlier tests. However, researchers continue to gather more information about its safety specifically for this condition.

Overall, while early results are promising, more information is needed to fully confirm rilonacept's safety in treating cardiac sarcoidosis. Trial participants are closely monitored to ensure any side effects are quickly addressed. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for cardiac sarcoidosis?

Rilonacept is unique because it targets the interleukin-1 (IL-1) pathway, which plays a significant role in inflammation. Unlike standard treatments for cardiac sarcoidosis that often involve corticosteroids and immunosuppressants, which can have broad effects on the immune system, Rilonacept specifically inhibits the activity of IL-1. This targeted approach may lead to fewer side effects and better control of inflammation associated with cardiac sarcoidosis. Researchers are excited because this could provide a more precise and effective treatment option, potentially improving outcomes for patients with this challenging condition.

What evidence suggests that rilonacept might be an effective treatment for cardiac sarcoidosis?

Research has shown that rilonacept might help reduce inflammation in people with cardiac sarcoidosis. In this trial, participants will receive either standard therapy alone or rilonacept combined with standard therapy. One study found that adding rilonacept to regular treatment lowered heart inflammation after 24 weeks. Rilonacept blocks a protein called interleukin-1, which causes inflammation. While primarily used for conditions like chronic recurrent pericarditis (inflammation of the heart's outer lining), its ability to reduce inflammation could also benefit those with cardiac sarcoidosis. Early evidence is promising, but further research is needed to confirm its effectiveness for this specific condition. ^{1 2 6 7 8}

Who Is on the Research Team?

Andrew Rosenbaum

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

This trial is for adults aged 18-80 with cardiac sarcoidosis, evidenced by active FDG uptake in the heart. Participants must understand and agree to study requirements, have completed vaccinations, and use effective contraception if applicable. Exclusions include pregnancy, weight over 380 pounds, other severe health conditions or treatments that could affect results.

Inclusion Criteria

Able to comprehend and willing to sign an ICF and to abide by the study restrictions and requirements

I am either postmenopausal, permanently sterile, or using effective birth control.

My PET scan shows active cancer in three or more areas recently.

Exclusion Criteria

Received or scheduled to receive mechanical circulatory support after randomization

My weight is over 380 pounds.

Unable or unwilling to provide informed consent

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive rilonacept added to standard therapy or standard therapy alone for 24 weeks

24 weeks

Visits at Baseline, Week 12, and Week 24

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Rilonacept

Trial Overview The study tests rilonacept's effectiveness on myocardial inflammation when added to standard therapy versus standard therapy alone in cardiac sarcoidosis patients over a period of 24 weeks. Participants will be monitored using an ambulatory cardiac rhythm monitor at certain times.

How Is the Trial Designed?

Group I: Rilonacept+Standard therapy Experimental Treatment 1 Intervention

Group II: Standard therapy Active Control 1 Intervention

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

Johns Hopkins University

Collaborator

Trials

2,366

Recruited

15,160,000+

Published Research Related to This Trial

Rituximab, an anti-CD20 monoclonal antibody, has shown promise in treating refractory sarcoidosis, particularly in cases involving the lungs, eyes, lymph nodes, and skin, as evidenced by three case reports.

While steroids are the standard first-line treatment for sarcoidosis, the use of Rituximab may offer an alternative for patients who do not respond to conventional therapies, highlighting its potential role in managing this challenging condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rituximab in refractory sarcoidosis: a single centre experience. Cinetto, F., Compagno, N., Scarpa, R., et al.[2020]

Cardiac involvement in sarcoidosis can range from 2% to 75% and may lead to serious issues like rhythm problems or heart failure, making diagnosis and treatment challenging.

Corticosteroids are likely effective for treating cardiac sarcoidosis, especially in cases of atrio-ventricular block or moderate heart failure, while TNF-α antagonists have shown success in treating refractory cases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Cardiac sarcoidosis: Diagnosis and therapeutic challenges]. Cohen Aubart, F., Nunes, H., Mathian, A., et al.[2017]

Corticosteroids are the first-line treatment for cardiac sarcoidosis (CS), especially in patients with serious heart issues like heart blocks or dysrhythmias, but there is a lack of large studies to guide treatment specifics.

Early use of second-line medications alongside corticosteroids may improve outcomes, although the effectiveness of anti-TNF agents as third-line treatments remains debated.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Controversies in the Treatment of Cardiac Sarcoidosis. Obi, ON., Lower, EE., Baughman, RP.[2022]

In a retrospective study of 4 patients with cardiac sarcoidosis (CS), TNFα antagonists (infliximab or adalimumab) were effective in treating refractory cases, showing promise for earlier intervention in treatment protocols.

All patients tolerated TNFα antagonists well, with no serious adverse events reported, and three patients were able to discontinue corticosteroid therapy without experiencing relapses or major cardiac issues during a follow-up period averaging 54.75 months.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Case Report: TNFα Antagonists Are an Effective Therapy in Cardiac Sarcoidosis. Stievenart, J., Le Guenno, G., Ruivard, M., et al.[2021]

In a study of 50 patients with cardiac sarcoidosis, treatment with tumor necrosis factor inhibitors (TNFi), particularly adalimumab, significantly reduced cardiac inflammation as measured by 18F-FDG PET/CT scans, showing better efficacy than traditional corticosteroid therapies.

TNFi was found to be safe and effective even in patients with severely reduced left ventricular ejection fraction (LVEF), with many patients achieving inactive or remitting disease status after treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of treatment response in cardiac sarcoidosis based on myocardial 18F-FDG uptake. Frischknecht, L., Schaab, J., Schmauch, E., et al.[2023]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06660732

Rilonacept in Subjects With Cardiac Sarcoidosis The primary objective of this study is to evaluate the effect of rilonacept, added to standard therapy and compared with standard therapy alone, on improvement ...

2. event.fourwaves.com event.fourwaves.com/wasog2025/abstracts/51109adf-1e6a-46ae-903c-280ae1e32a63

A Phase 2 Study of Rilonacept in Patients with Cardiac ... Results: The primary efficacy endpoint is change from baseline in number of segments with FDG uptake on cardiac FDG-PET scan at Week 24.

3. jacc.org jacc.org/doi/10.1016/S0735-1097%2823%2903816-0

A RARE CASE OF CARDIAC SARCOIDOSIS ... The use of IL-1 inhibitors in sarcoid has not been studied, however rilonacept was initiated due to its efficacy in treating chronic recurrent pericarditis ...

4. myocarditisfoundation.org myocarditisfoundation.org/location/johns-hopkins-university/

Johns Hopkins University Archives Brief Summary: The primary objective of this study is to evaluate the effect of rilonacept, added to standard therapy and compared with standard therapy alone, ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0167527321006586

Identification of a novel presumed cardiac sarcoidosis ... Clinical categorization of patients with presumed CS identified a high-risk cohort comparable to patients with histologic evidence of disease.

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/33229362/

Efficacy and safety of rilonacept for recurrent pericarditis Rilonacept led to rapid and sustained improvement in pain, inflammation (CRP and pericarditis manifestations) and HRQOL.

7. app.trialscreen.org app.trialscreen.org/trials/phase-2-rilonacept-subjects-cardiac-sarcoidosis-trial-nct06660732

Rilonacept in Subjects With Cardiac Sarcoidosis Participants in the group receiving rilonacept may experience a reduction in inflammation in their heart caused by sarcoidosis, which could potentially improve ...

8. centerwatch.com centerwatch.com/clinical-trials/listings/search/?q=cardiac%20sarcoidosis

cardiac sarcoidosis Clinical Research Trials Clinical outcomes will be defined as: 1) improvement of symptoms (FAS score, heart failure class, AV block, ventricular arrhythmias, and KSQ score) and 2) ...