Sirolimus for Castleman Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests sirolimus, a medication that controls the immune system, on patients with iMCD who haven't responded to other treatments. Sirolimus blocks a pathway to reduce immune response and abnormal cell growth. Sirolimus has shown potential in treating various conditions.
Will I have to stop taking my current medications?
The trial requires that you stop taking any systemic therapies intended to treat Castleman disease, except for corticosteroids, at least 28 days before enrolling. If you are on anti-IL-6 therapy, you need to stop it at least 14 days before enrolling, unless you cannot or are unwilling to do so.
Is Sirolimus generally safe for humans?
Sirolimus, also known as Rapamune, is generally considered safe for humans, but it can cause some side effects. It has been used since 1999 to prevent organ rejection in kidney transplants and is known to have a lower risk of kidney and nerve problems compared to other similar drugs. However, it can increase lipid levels (fats in the blood), which might lead to heart and blood vessel issues, and in children, it can cause mouth sores.12345
How is the drug sirolimus unique in treating Castleman disease?
Sirolimus is unique for treating Castleman disease because it targets the mTOR pathway, which is often overactive in patients who do not respond to standard IL-6 inhibitor treatments. This makes it a promising option for those with IL-6 inhibitor-resistant idiopathic multicentric Castleman disease.13678
What data supports the effectiveness of the drug Sirolimus for Castleman Disease?
Sirolimus has shown effectiveness in treating other conditions like Kaposiform hemangioendothelioma (a rare tumor) and certain skin disorders due to its strong ability to suppress the immune system and reduce cell growth. This suggests it might also be helpful for Castleman Disease, which involves similar immune and cell growth issues.1391011
Who Is on the Research Team?
David C Fajgenbaum, MD, MBA, MSc
Principal Investigator
University of Pennsylvania
Are You a Good Fit for This Trial?
This trial is for people aged 2-80 with idiopathic multicentric Castleman disease who haven't responded well to anti-IL-6 therapy or can't tolerate it. Participants must have active disease, be able to take oral medication, and not be pregnant or nursing. They shouldn't have had sirolimus before, any recent other systemic therapies except corticosteroids, severe infections mimicking iMCD, certain cancers within the last year, serious psychiatric disorders affecting consent ability, or very poor health as defined by specific medical criteria.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive daily oral sirolimus for 12 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Sirolimus
Sirolimus is already approved in United States, European Union, Canada, Japan for the following indications:
- Prevention of organ rejection in kidney transplant patients
- Treatment of lymphangioleiomyomatosis (LAM)
- Prevention of organ rejection in kidney transplant patients
- Treatment of lymphangioleiomyomatosis (LAM)
- Prevention of organ rejection in kidney transplant patients
- Treatment of lymphangioleiomyomatosis (LAM)
- Prevention of organ rejection in kidney transplant patients
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Pennsylvania
Lead Sponsor