Ruxolitinib for Castleman's Disease

Overseen By Bridget Austin, MS

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Pennsylvania

Must be taking: Anti-IL-6 therapy

Must not be taking: Ruxolitinib, others

Disqualifiers: Pregnancy, Uncontrolled infection, HIV, 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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

14 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests ruxolitinib, a medication, to evaluate its effectiveness for individuals with idiopathic Multicentric Castleman Disease (iMCD). iMCD is a rare condition characterized by excessive lymph node growth, leading to symptoms such as fatigue, fever, and weight changes. The trial targets those whose iMCD hasn't improved with certain other treatments or who cannot take them. Individuals experiencing these symptoms and for whom treatments have been ineffective might find this trial suitable. Participants must be able to take pills and have active iMCD symptoms. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

Do I need to stop my current medications to join the trial?

The trial requires a 14-day period without taking any systemic therapies for Castleman's Disease, except for corticosteroids or anti-IL-6 therapy. If you're on anti-IL-6 therapy, you may need to stop it within the first 6 weeks after starting ruxolitinib.

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

Research has shown that ruxolitinib is generally well-tolerated by patients with idiopathic Multicentric Castleman Disease (iMCD). One study found that patients who took ruxolitinib experienced quick relief from their symptoms, and after a year, they had no severe symptoms. Another source mentions that the treatment is being tested to ensure manageable side effects for patients.

While specific side effects are not listed in the sources, the fact that ruxolitinib is in Phase 2 trials indicates it has passed initial safety tests. This suggests it was safe enough in earlier studies to be tested on more people.

Overall, the available data suggests that ruxolitinib is safe for people with iMCD, but like any treatment, there may be side effects. Anyone considering joining the trial should discuss potential risks with their healthcare provider. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Ruxolitinib is unique because it targets the JAK1 and JAK2 pathways, which play a role in the inflammatory process of Castleman's Disease. While current treatments often focus on chemotherapy or immunomodulatory therapies, ruxolitinib offers a targeted approach that directly interferes with the signaling pathways contributing to the disease. Researchers are excited about this treatment because it has the potential to reduce symptoms more effectively by specifically addressing the underlying mechanisms of the condition.

What evidence suggests that ruxolitinib might be an effective treatment for idiopathic Multicentric Castleman Disease?

Research has shown that ruxolitinib can help treat idiopathic multicentric Castleman disease (iMCD), especially when treatments like siltuximab or tocilizumab have failed. In one study, patients taking ruxolitinib improved quickly and were symptom-free after 12 months. Another report noted that patients felt better overall and had lower levels of CRP, a marker of inflammation. These findings suggest that ruxolitinib may help manage symptoms and improve health in those with iMCD that doesn't respond to other treatments. In this trial, all participants will receive ruxolitinib to further evaluate its effectiveness for iMCD. ^{1 2 3 4 6}

Who Is on the Research Team?

David Fajgenbaum, MD, MBA, MSc

Principal Investigator

University of Pennsylvania

Are You a Good Fit for This Trial?

Adults with idiopathic Multicentric Castleman Disease (iMCD) who haven't improved after using siltuximab or tocilizumab, or can't take those medications. Specific eligibility details are not provided.

Inclusion Criteria

My condition meets the criteria for iMCD.

My condition is confirmed by a pathologist to match iMCD criteria or I have a CAS grade of 3 or higher.

My condition did not improve with anti-IL-6 therapy, it returned, or I couldn't tolerate the treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ruxolitinib to assess its effects on iMCD symptoms

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 73 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ruxolitinib

Trial Overview The trial is studying the effects of a medication called Ruxolitinib on iMCD in patients who have previously been treated without success or cannot use standard treatments like siltuximab and tocilizumab.

How Is the Trial Designed?

Group I: Ruxolitinib Experimental Treatment 1 Intervention

This is an open-label study so all patients will be assigned to the same interventional arm and given ruxolitinib

Who Is Running the Clinical Trial?

University of Pennsylvania

Lead Sponsor

Trials

2,118

Recruited

45,270,000+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3,987

Recruited

47,860,000+

Incyte Corporation

Industry Sponsor

Trials

408

Recruited

66,800+

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10918405/

Ruxolitinib for the Treatment of Refractory Idiopathic ... Ruxolitinib (10 mg/day) was started as a third-line treatment with a rapid response (Table 1). After 12 months, the patient was free of constitutional symptoms.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07085039

Ruxolitinib in Previously Treated Idiopathic Multicentric ... The Overall Symptom Score, comprising 34 outcome measures (fatigue, weight change, night sweats, etc.), will be used to evaluate the broader impact of ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S0268960X23001315

Idiopathic multicentric Castleman disease: An update in ... At 5 years, the PFS was 43% and the overall survival was 81%. However, rituximab is most effective in the treatment of HHV8-associated MCD and has limited ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36482523/

Treatment-resistant idiopathic multicentric Castleman ... Outcomes: Each patient received ruxolitinib, the general condition improved, and CRP levels decreased. Lessons: These cases showed that ruxolitinib was ...

5. researchgate.net researchgate.net/publication/378220040_Ruxolitinib_for_the_Treatment_of_Refractory_Idiopathic_Multicentric_Castleman_Disease_A_Case_Report

(PDF) Ruxolitinib for the Treatment of Refractory Idiopathic ... No grade 3 or higher adverse events occurred during the median time to the next treatment of 29.0 (range: 15.0–36.2) months. No patient ...

6. withpower.com withpower.com/trial/phase-2-castleman-disease-8-2025-62cf2

Ruxolitinib for Castleman's Disease This Phase 2 medical study run by University of Pennsylvania is evaluating whether Ruxolitinib will have tolerable side effects & efficacy for patients with ...