Recruiting
Phase 2

Ruxolitinib vs. Prednisone

Sponsor:

H. Lee Moffitt Cancer Center and Research Institute

Code:

NCT06660355

Conditions

Chronic Graft-versus-host Disease (cGVHD)

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Ruxolitinib

Prednisone

Study Details

Brief summary:

Allogeneic transplant is potentially curative for hematological malignancies but its use is limited by the development of GVHD. Ruxolitinib now has FDA approval for treatment of chronic GVHD that has failed 1-2 prior lines of therapy based on a prior large, randomized phase III study. Given this evidence of safety and efficacy in the early refractory setting (after prednisone failure), Ruxolitinib represents an ideal agent to test in the primary therapy setting. Here investigators propose a phase 2 randomized study to compare Ruxolitinib to prednisone as a first-line therapy in the treatment of chronic GVHD.

Conditions

Chronic Graft-versus-host Disease (cGVHD)

Study ID

NCT06660355

Start date

Dec 23, 2024

Status verified date

Jul, 2026

Completion date

Dec, 2028

Anticipated

Primary completion date

Dec, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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. Molecular relapse or Mixed chimerism or treated relapse in remission may be allowed on case by case basis but needs discussion with the study Chair/PI
  • 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.

Study Design

Enrollment

120 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Ruxolitinib Treatment Arm

Ruxolitinib is administered as 10 mg orally twice daily in 28-day cycles.

active comparator: Prednisone Treatment Arm

Prednisone will be started at 1mg/kg/day based on patient current body weight in kilograms.

Interventions

Ruxolitinib

Ruxolitinib is a Janus kinase inhibitor.

Prednisone

Prednisone is a glucocorticoid.

Primary outcome measure

  • Treatment Success [ Time Frame: 6 months ]

Central Contacts and Locations

Central contacts

Sarah Starr

813-745-2690Sarah.Starr@moffitt.org

Locations

Univ of Miami - Sylvester Comprehensive Cancer Center

Recruiting

Miami, Florida, United States, 33136

Principal Investigator:

Noa Holtzman, MD

Moffitt Cancer Center

Recruiting

Tampa, Florida, United States, 33612

Principal Investigator:

Farhad Khimani, MD

University of Kansas Cancer Center

Recruiting

Kansas City, Kansas, United States, 66160

Principal Investigator:

Muhammad Umair Mushtaq, MD

Fox Chase Cancer Center

Recruiting

Philadelphia, Pennsylvania, United States, 19111

Principal Investigator:

Alexander Vartanov, MD

University of Virginia Comprehensive Cancer Center

Recruiting

Charlottesville, Virginia, United States, 22908

Principal Investigator:

Indumathy Varadarajan, MD

Virginia Commonwealth University

Recruiting

Richmond, Virginia, United States, 23219

Principal Investigator:

William Clark, MD

More Information

Sponsor

H. Lee Moffitt Cancer Center and Research Institute

Last update posted

Aug 5, 2026

Last verified

Jul, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by H. Lee Moffitt Cancer Center and Research Institute on 2026-08-05.