Comparing Ruxolitinib and Prednisone for treating chronic GVHD

Phase II Randomized Study of Ruxolitinib vs Prednisone as First-Line Therapy for Chronic Graft vs Host Disease Needing Systemic Therapy

This study is testing whether Ruxolitinib works better than Prednisone for adults with chronic graft-versus-host disease who need treatment.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 120 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: H. Lee Moffitt Cancer Center and Research Institute (other)
• Drugs / interventions: Ruxolitinib, prednisone
• Locations: 5 sites (Miami, Florida and 4 other locations)
• Trial ID: NCT06660355 on ClinicalTrials.gov

What this trial studies

This phase 2 clinical trial aims to evaluate the effectiveness of Ruxolitinib compared to Prednisone as a first-line treatment for patients with chronic graft-versus-host disease (cGVHD) requiring systemic therapy. The study will enroll adults diagnosed with cGVHD who have not received any new immunosuppressive therapy in the two weeks prior to enrollment. Participants will be randomly assigned to receive either Ruxolitinib or Prednisone, with the goal of determining which treatment offers better outcomes in managing this condition.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older diagnosed with chronic GVHD who require systemic therapy.

Not a fit: Patients who have recently started new immunosuppressive therapy or those with severe organ dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a more effective first-line treatment option for patients with chronic GVHD.

How similar studies have performed: Previous studies have shown Ruxolitinib to be effective in treating cGVHD after prior therapies, making this approach promising but still requiring further investigation in the first-line setting.

Eligibility criteria

Inclusion Criteria:

* Age ≥ 18 years.
* Karnofsky performance status ≥60%.
* Patients with a diagnosis of chronic GVHD per NIH diagnostic criteria5 who are in need for first systemic therapy as per treating physician's discretion, Overlap chronic GVHD will be allowed.
* No new immune suppressive therapy added within preceding 2 weeks prior to study enrolment.
* Able to take oral medications.
* Participants must have adequate organ and marrow function as defined below:

  1. absolute neutrophil count ≥1,000/mcL
  2. platelets ≥30,000/mcL
  3. Hemoglobin ≥ 7 g/dL
  4. Bilirubin ≤ 3 times institutional upper limit of normal (ULN) unless attributable to GVH

  d. AST(SGOT)/ALT(SGPT) ≤5 × institutional ULN unless attributable to GVH e. creatinine clearance ≥30 ml/min
* Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of study drug administration.
* Ability to understand and the willingness to sign a written informed consent document.

Exclusion Criteria:

* Previously treated with systemic immune suppressive therapy for chronic GVHD (where the indication for start of that systemic immune suppressive therapy was chronic GVHD).
* Patients with clinically significant or uncontrolled cardiovascular disease, including unstable angina, acute myocardial infarction, or stroke within 6 months, New York Heart Association class III or IV heart failure will be excluded.
* Relapse malignancy post- transplant.
* Active hepatitis B, hepatitis C and HIV will be excluded.
* Any uncontrolled infection at the time if enrollment will be excluded.
* History of allergic reactions attributed to compounds of similar chemical or biologic composition to Ruxolitinib.
* Participants with psychiatric illness/social situations that would limit compliance with study requirements.
* Pregnant women and lactating women are excluded from this study because of the potential for teratogenic or abortifacient effects and an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with Ruxolitinib, breastfeeding should be discontinued if the mother is treated with Ruxolitinib.
* Current or history of active Tuberculosis.

Where this trial is running

Miami, Florida and 4 other locations

• Univ of Miami - Sylvester Comprehensive Cancer Center — Miami, Florida, United States (RECRUITING)
• Moffitt Cancer Center — Tampa, Florida, United States (RECRUITING)
• University of Kansas Cancer Center — Kansas City, Kansas, United States (RECRUITING)
• University of Virginia Comprehensive Cancer Center — Charlottesville, Virginia, United States (RECRUITING)
• Virginia Commonwealth University — Richmond, Virginia, United States (RECRUITING)

Study contacts

• Principal investigator: Farhad Khimani, MD — Moffitt Cancer Center
• Study coordinator: Sarah Starr

Conditions: Chronic Graft-versus-host Disease