Ruxolitinib vs Prednisone as First-line Therapy for cGVHD Needing Systemic Therapy

NCT06660355 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: MCC-23051
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 5

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)

Outcome Measures

Primary Outcomes (1)

• Treatment Success

  Treatment Success is defined as C7D1 NIH CR/PR without death or new line of immune suppressive therapy and will be estimated for both arms.

  6 months

Secondary Outcomes (1)

• Treatment Success Rate

  6 months

Study Arms (2)

Ruxolitinib Treatment Arm

EXPERIMENTAL

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

Drug: Ruxolitinib

Prednisone Treatment Arm

ACTIVE COMPARATOR

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

Drug: Prednisone

Interventions

Prednisone DRUG

Prednisone is a glucocorticoid.

Prednisone Treatment Arm

Ruxolitinib DRUG

Ruxolitinib is a Janus kinase inhibitor.

Ruxolitinib Treatment Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• absolute neutrophil count ≥1,000/mcL
• platelets ≥30,000/mcL
• Hemoglobin ≥ 7 g/dL
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• H. Lee Moffitt Cancer Center and Research Institute: lead
• Incyte Corporation: collaborator

Study Sites (5)

Univ of Miami - Sylvester Comprehensive Cancer Center

Miami, Florida, 33136, United States

RECRUITING

Moffitt Cancer Center

Tampa, Florida, 33612, United States

RECRUITING

University of Kansas Cancer Center

Kansas City, Kansas, 66160, United States

RECRUITING

University of Virginia Comprehensive Cancer Center

Charlottesville, Virginia, 22908, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23219, United States

RECRUITING

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Interventions

ruxolitinib; Prednisone

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Farhad Khimani, MD

  Moffitt Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sarah Starr

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2024
• First Posted: October 28, 2024
• Study Start: December 23, 2024
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: April 8, 2026

Record last verified: 2026-04

Locations

US (5)