Chronic Graft Versus Host DiseasePhase 2NCT05567406
Safety and Efficacy of Oral Belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander Male and Female Participants Aged 12 Years and Above With Chronic Graft Versus Host Disease (cGVHD) After At Least 2 Prior Lines of Systemic Therapy
A Phase 2 study testing Belumosudil in people with Chronic Graft Versus Host Disease, run by Kadmon, a Sanofi Company.
Trial summary
The purpose of this study is to measure safety and efficacy of oral belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander male and female participants with cGVHD who have previously been treated with at least 2 prior lines of systemic therapy aged 12… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Chronic Graft Versus Host Disease
- What is being tested?
- Belumosudil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 16 Jun 2025to 31 Aug 2026
- Phase
- Phase 2
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants are included in the study if any of the following criteria apply:
- Participant is Black or African American, or American Indian or Alaska Native, or Native Hawaiian or Other Pacific Islander by self-identification.
- Previously received at least 2 and not more than 5 lines of systemic therapy for cGVHD.
- Receiving glucocorticoid therapy with a stable dose over the 2 weeks prior to screening.
- Have persistent cGVHD manifestations and systemic therapy is indicated.
- Karnofsky (if aged ≥ 16 years) / Lansky (if aged < 16 years) Performance Score of ≥ 60.
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- At least 12 years of age; weight ≥ 40 kilograms (kg).
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x upper limit of normal (ULN).
- Total bilirubin ≤ 1.5 x ULN.
- Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants
- Capable of giving signed informed consent.
Who cannot join
- Participants are excluded from the study if any of the following criteria apply:
- Participant has not been on a stable dose/regimen of systemic cGVHD treatment(s) for at least 2 weeks prior to screening. (Note: Concomitant corticosteroids, calcineurin inhibitors, sirolimus, MMF, methotrexate, rituximab, and ECP are acceptable. Systemic investigational GVHD treatments are not permitted).
- Histological relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening.
- Current treatment with ibrutinib or ruxolitinib. Prior treatment with ibrutinib or ruxolitinib is allowed with a washout of at least 28 days prior to enrollment.
- History or other evidence of severe illness or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study (such as malabsorption syndromes, poorly controlled psychiatric disease, or coronary artery disease).
- Corrected QT interval using Fridericia's formula (QTc[F]) > 480 ms.
- Forced expiratory volume (in the first second; FEV1) ≤ 39% The above information is not intended to contain all considerations relevant to the potential participation in a clinical trial.
About this study
Official title: A Phase 2, Open-label, Multicenter Study to Evaluate the Safety and Efficacy of Belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander Participants With Chronic Graft Versus Host Disease (cGVHD) After At Least 2 Prior Lines of Systemic Therapy
The purpose of this study is to measure safety and efficacy of oral belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander male and female participants with cGVHD who have previously been treated with at least 2 prior lines of systemic therapy aged 12 years and above.
The duration of participants participation will be up to 4 weeks for screening, treatment until clinically significant progression of disease, and 4 weeks of safety follow-up, and then long-term follow-up every 12 weeks.1 Cycle = 28 days.
Read the detailed description
Up to 4 weeks for screening, treatment until clinically significant progression of disease, 4 weeks of safety follow-up and then long-term follow-up every 12 weeks.
- Conditions
- Chronic Graft Versus Host Disease
- Being tested
- Belumosudil
- 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.
BelumosudilDrug
Also called KD025, SAR445761, Rezurock
Pharmaceutical form: Tablet; Route of administration: Oral
| Study group | Role | What they receive |
|---|---|---|
| Belumosudil | Experimental | Participants will receive belumosudil orally, once daily (QD) or twice daily (BID) if they are taking strong CYP3A4 inducers or proton pump inhibitors. Receives: Drug: Belumosudil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment emergent adverse events and serious adverse events
Safety will be assessed by monitoring adverse events, physical Examinations, clinical laboratory evaluations, vital sign measurements, and ECG parameters.
Measured: Up to approximately 48 months
Number of participants with clinically significant laboratory abnormalities
Measured: Up to approximately 12 months
Change from baseline in systolic and diastolic blood pressure
Measured: Baseline; up to approximately 12 months
Change from baseline in heart rate
Measured: Baseline; up to approximately 12 months
Change from baseline in corrected QT interval using Fridericia's formula (QTc[F])
Measured: Baseline; up to approximately 12 months
Overall Response Rate (ORR)
The ORR is defined as the proportion of participants meeting the overall response criteria assessment of Complete Response (CR) or Partial Response (PR) as defined by the 2014 NIH Consensus Development Project on Clinical Trials in cGVHD at any post-baseline response assessment.
Measured: Up to approximately 12 months
Secondary outcomes (15)
Duration of Response (DOR)
Assessments of DOR includes
- The time from first response to progression, death, or new systemic therapy for cGVHD
- Time from initial response to start of additional systemic cGVHD therapy or death
Measured: Up to approximately 12 months
Change from baseline in the Lee Symptom Scale Score: Number of participants with a ≥ 7-point reduction
Changes in symptom burden/bother will be assessed using the Lee Symptom Scale, a symptom scale designed for individuals with chronic Graft Versus Host Disease (cGVHD). The questionnaire asks participants to indicate the degree of bother that they experienced due to symptoms in seven domains potentially affected by cGVHD (skin, eyes, mouth, breathing, eating and digestion, energy, and emotional distress). The response will be determined based on clinician's assessment.
Measured: Baseline; up to approximately 12 months
Change from baseline in the Lee Symptom Scale Score: Number of participants with a ≥ 7-point reduction on 2 consecutive assessments
Changes in symptom burden/bother will be assessed using the Lee Symptom Scale, a symptom scale designed for individuals with chronic Graft Versus Host Disease (cGVHD). The questionnaire asks participants to indicate the degree of bother that they experienced due to symptoms in seven domains potentially affected by cGVHD (skin, eyes, mouth, breathing, eating and digestion, energy, and emotional distress). The response will be determined based on clinician's assessment.
Measured: Baseline; up to approximately 12 months
Change from baseline in the Lee Symptom Scale Score: Duration of a ≥ 7 point reduction
Changes in symptom burden/bother will be assessed using the Lee Symptom Scale, a symptom scale designed for individuals with chronic Graft Versus Host Disease (cGVHD). The questionnaire asks participants to indicate the degree of bother that they experienced due to symptoms in seven domains potentially affected by cGVHD (skin, eyes, mouth, breathing, eating and digestion, energy, and emotional distress). The response will be determined based on clinician's assessment.
Measured: Baseline; up to approximately 12 months
Response rate by organ system
The response rate for the nine individual organs (skin, eyes, mouth, esophagus, upper GI, lower GI, liver, lungs, and joints and fascia) will be assessed by the clinician.
Measured: Up to approximately 12 months
Time to Response (TTR)
TTR defined as the time from the first dose of belumosudil to the first documented cGVHD response.
Measured: Up to approximately 12 months
Time to Next Treatment (TTNT)
TTNT defined as the time from the first dose of belumosudil to the start of additional systemic cGVHD therapy.
Measured: Up to approximately 12 months
Number of participants who have a best response of PR and CR
CR is defined as resolution of all manifestations of cGVHD in each organ or site. PR is defined as Improvement in at least 1 organ or site without progression in any other organ or site.
Measured: Up to approximately 12 months
Change from baseline in corticosteroid dose
The prednisone equivalent dose of corticosteroids (mg/kg/day) during the study will be analyzed. The change in systemic corticosteroid dose over time will be determined.
Measured: Baseline; up to approximately 12 months
Change from baseline in calcineurin inhibitor dose
The change in calcineurin inhibitor dose over time will be determined.
Measured: Baseline; up to approximately 12 months
Failure-free survival (FFS)
FFS defined as the absence of new cGVHD systemic therapy, non-relapse mortality and recurrent malignancy. Median FFS (from first dose of belumosudil) will be analyzed.
Measured: Up to approximately 12 months
Overall survival (OS)
OS defined as time from first dose of belumosudil to the date of death due to any cause.
Measured: Up to approximately 12 months
Change from baseline in cGVHD global severity rating using the Clinician-Reported Global cGVHD Activity Assessment
Patient-reported outcome
Measured: Baseline; up to approximately 12 months
Change from baseline in symptom activity as based on cGVHD Activity Assessment Patient Self-Report
Patient-reported outcome
Measured: Baseline; up to approximately 12 months
Plasma belumosudil concentrations
Measured: Day 1 of Cycles 2, 3, 5, and 7 (1 Cycle = 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2022
- Study start16 Jun 2025 (estimated)
- Primary completion31 Aug 2026 (estimated)
- Study completion31 Aug 2026 (estimated)
Why it stopped: Sponsor decision.
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 DiseaseStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kadmon, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05567406
- Sponsor's protocol ID
- SFY17661
- Other IDs
- U1111-1277-6715, KD025-218
- Status last verified
- Feb 2026
- First submitted
- 3 Oct 2022
- Last updated
- 12 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org