Leflunomide for Chronic Graft Versus Host Disease

Recruiting in Palo Alto (17 mi)

Overseen ByAmandeep Salhotra

Age: 18+

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: City of Hope Medical Center

No Placebo Group

Approved in 4 jurisdictions

Trial Summary

What is the purpose of this trial?This phase I trial studies the side effects of leflunomide in treating patients with steroid dependent chronic graft versus host disease (cGVHD). cGVHD is a common complication of bone marrow transplant. GVHD occurs when immune cells transplanted from a donor (the graft) recognize the transplant recipient (the host) as foreign, and cause damage to the skin, gastrointestinal tract or other organs. Steroids are the first line of therapy and benefits are seen in about one-third of patients with cGVHD. Prolonged use of steroids is associated with multiple complications. Leflunomide may decrease the body's immune response and reduce inflammation associated with cGVHD.

Eligibility Criteria

This trial is for patients with chronic graft versus host disease (cGVHD) after a bone marrow transplant, who are dependent on steroids without full relief of symptoms. Participants must have stable organ function and blood counts, be able to swallow pills, and not be pregnant or breastfeeding. They should agree to use birth control and have no active infections or other serious health issues that could interfere with the study.

Inclusion Criteria

I can swallow pills.

I have chronic GVHD that hasn't improved with 2 months of prednisone treatment.

I have received a stem cell transplant from another person.

My white blood cell count is healthy without needing medication in the past week.

I am able to care for myself but may not be able to do active work.

Exclusion Criteria

I need a machine to help me breathe.

I am currently receiving dialysis.

I am currently experiencing diarrhea not caused by cGVHD.

I do not have any uncontrolled serious illnesses.

I do not have any uncontrolled illnesses or active infections.

My cancer has returned and is active.

I do not have any active cancer except for minor skin cancer.

I am experiencing acute graft-versus-host disease.

I do not have severe heart problems like uncontrolled chest pain or heart failure.

I am not pregnant and not nursing.

I have a history of HIV or hepatitis B/C.

Participant Groups

The trial is testing Leflunomide's effectiveness in treating cGVHD symptoms when steroids alone aren't enough. It aims to see if this drug can reduce the immune response causing inflammation without the complications linked to prolonged steroid use.

1Treatment groups

Experimental Treatment

Group I: Treatment (leflunomide)Experimental Treatment1 Intervention

Patients receive leflunomide PO QD for days 1-28. Treatment repeats every 28 days for 6 cycles in the absence of disease progression or unacceptable toxicity. Patients may optionally continue leflunomide for an additional 6 cycles as long as response or stable disease is maintained.

Leflunomide is already approved in European Union, United States, United Kingdom for the following indications:

🇪🇺 Approved in European Union as Arava for: