Chronic Graft-versus-host Disease (cGVHD)Phase 2NCT06660355
Ruxolitinib vs Prednisone as First-line Therapy for cGVHD Needing Systemic Therapy
A Phase 2 study testing Ruxolitinib and Prednisone in people with Chronic Graft-versus-host Disease (cGVHD), run by H. Lee Moffitt Cancer Center and Research Institute at 6 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Graft-versus-host Disease (cGVHD)
- What is being tested?
- Ruxolitinib and Prednisone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 6 sitesUnited States
- Study dates
- 23 Dec 2024to Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
Show all 13Show fewer
- 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
- 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.
Who cannot join
- 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.
Show all 9Show fewer
- 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.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
University of Virginia Comprehensive Cancer Center
Charlottesville, Virginia, 22908
Indumathy Varadarajan, MD
- Recruiting
University of Kansas Cancer Center
Kansas City, Kansas, 66160
Muhammad Umair Mushtaq, MD
- Recruiting
Univ of Miami - Sylvester Comprehensive Cancer Center
Miami, Florida, 33136
Noa Holtzman, MD
- Recruiting
Fox Chase Cancer Center
Philadelphia, Pennsylvania, 19111
Alexander Vartanov, MD
- Recruiting
Virginia Commonwealth University
Richmond, Virginia, 23219
William Clark, MD
- Recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
Farhad Khimani, MD
Melissa Alsina, MD
About this study
Official title: Phase II Randomized Study of Ruxolitinib vs Prednisone as First-Line Therapy for Chronic Graft vs Host Disease Needing Systemic Therapy
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.
- Being tested
- Ruxolitinib, Prednisone
- Medical subject headings
- Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Ruxolitinib is a Janus kinase inhibitor.
PrednisoneDrug
Prednisone is a glucocorticoid.
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib Treatment Arm | Experimental | Ruxolitinib is administered as 10 mg orally twice daily in 28-day cycles. Receives: Drug: Ruxolitinib |
| Prednisone Treatment Arm | Active comparator | Prednisone will be started at 1mg/kg/day based on patient current body weight in kilograms. Receives: Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 months
Secondary outcomes (1)
Treatment Success Rate
The proportion of subjects who are alive with NIH CR/PR, no new systemic therapy for cGVHD, and < 0.25 mg/kg/day prednisone at C7D1 with 95% CI for each arm will be estimated.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 2024
- Study start23 Dec 2024
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Graft-versus-host Disease (cGVHD)Studies of the same condition, open and under way.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Incyte Corporation
- Responsible party
- Sponsor
- Investigators
- Farhad Khimani, MD, Principal investigator, Moffitt Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06660355
- Sponsor's protocol ID
- MCC-23051
- Status last verified
- Jul 2026
- First submitted
- 24 Oct 2024
- Last updated
- 5 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Randomized Study of Ruxolitinib vs Prednisone as First-Line Therapy for Chronic Graft vs Host Disease Needing Systemic Therapy
- ClinicalTrials.gov ID
- NCT06660355
- Acronym / sponsor ID
- MCC-23051
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- Incyte Corporation
- Study start
- 23 Dec 2024 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 28 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib Treatment Arm | Experimental | Drug: RuxolitinibRuxolitinib is administered as 10 mg orally twice daily in 28-day cycles. |
| Prednisone Treatment Arm | Active comparator | Drug: PrednisonePrednisone will be started at 1mg/kg/day based on patient current body weight in kilograms. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RuxolitinibRuxolitinib is a Janus kinase inhibitor. | Drug | Ruxolitinib Treatment Arm |
| PrednisonePrednisone is a glucocorticoid. | Drug | Prednisone Treatment Arm |
Primary outcome
Treatment Success
Time frame: 6 months
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.
1 secondary outcome
Treatment Success Rate
Time frame: 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2026.
Related trials for Chronic Graft-versus-host Disease (cGVHD)
RecruitingPhase 2
Tafasitamab With Acalabrutinib and Venetoclax for the Treatment of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Chronic Lymphocytic Leukemia, Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Not yet recruiting
Feasibility of a Robot-Guided Active Play Intervention for Children With Cerebral Palsy
Cerebral Palsy
Recruiting
Empowered Relief® for Cancer Survivors With Chronic Pain
Chronic Pain Due to Cancer, Pain Catastrophizing
Recruiting
PAW-Hydrogel for Chronic Wound Healing
Diabetic Foot Ulcer (DFU), Pressure Ulcer (PU)
Active, not recruitingPhase 2
Safety and Efficacy of Empagliflozin in Hemodialysis
End Stage Renal Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Farhad Khimani, MD principal investigator
Moffitt Cancer Center
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ruxolitinib and Prednisone in people with Chronic Graft-versus-host Disease (cGVHD), run by H. Lee Moffitt Cancer Center and Research Institute at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sarah Starr (813-745-2690, Sarah.Starr@moffitt.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment Success (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2026.