A Phase 2 interventional study of Belumosudil (KD025) in Chronic Graft-versus-host-disease, sponsored by Kadmon, a Sanofi Company. Terminated at 35 sites in United States. Open to participants aged 12 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2024-12-12.
Sponsored by Kadmon, a Sanofi Company (part of Sanofi) · Phase 2, Interventional, and Treatment
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Among 14 studies testing belumosudil in Bronchiolitis Obliterans Syndrome — 7 open to participants now, 8 under way, 2 of them phase 3. belumosudil · Bronchiolitis Obliterans Syndrome.
Among the 14 studies testing belumosudil in Bronchiolitis Obliterans Syndrome, this one is the 3rd largest by enrollment, is the only one that started in 2018, is one of 8 in phase 2, and was the 1st registered.
714 studies on the registry are indexed under Bronchiolitis Obliterans Syndrome; 128 are open to participants now.
Browse Bronchiolitis Obliterans Syndrome studies →7 of the 14 studies testing belumosudil in Bronchiolitis Obliterans Syndrome are open now, in 12 countries. Recruiting first, the newest first:
Sanofi · Sites in Netherlands, Spain
Ages 12+
M.D. Anderson Cancer Center · Sites in United States
Ages 18+
Sanofi · Sites in United States, Belgium, Canada and 10 more countries
Ages 1–18
Dana-Farber Cancer Institute · Sites in United States
Ages 18+
Fred Hutchinson Cancer Center · Sites in United States
Ages 18+
By country, each count listing its studies:
belumosudil: first FDA approval 2021 (REZUROCK), first EMA approval 2026. What it is approved for, and how studies give it, is on its page →
Of the 100 Bronchiolitis Obliterans Syndrome studies recruiting now, 45 (45%) set no upper age limit and 45 (45%) stop at 75 or younger; 25 take children under 18; 12 take healthy volunteers.
This study: 12 Years and older · all sexes · healthy volunteers too.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
14 studies; the count open now beside each. This study's row is marked.
First in 2018; this study’s year is marked. 1 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 159 is above the median of 50 across 565 interventional studies indexed under Bronchiolitis Obliterans Syndrome.
Median enrollment 34 across the 14 interventional studies. Of the 4 that completed or were terminated, 3 (75%) have results posted.
This tab as a report to print or save as PDF → With an account.
10 of 14 have an industry lead sponsor. The 7 most frequent:
This study is registered under Kadmon, a Sanofi Company, one of the names counted as Sanofi. Under that name alone:
Kadmon, a Sanofi Company is the lead sponsor of 14 studies on the registry; none are open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 6 (67%) have results posted.
11 studies in Bronchiolitis Obliterans Syndrome by Sanofi: 4 open now, 5 under way, 2 of them phase 3. Of 5 finished, 3 (60%) have results posted.
Among the 11 studies in Bronchiolitis Obliterans Syndrome by Sanofi, this one is the 4th largest by enrollment, is the only one that started in 2018, is one of 5 in phase 2, and was the 3rd registered.
Sanofi’s Bronchiolitis Obliterans Syndrome studies by the year they started, since 2006.
3 of the studies testing belumosudil in Bronchiolitis Obliterans Syndrome that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07726914SanofiStudy to Compare the Plasma Concentration of Belumosudil Given as an Oral Suspension and as a Tablet to Healthy Adult Male Participants | 2027-01-26 |
| NCT05922761Dana-Farber Cancer InstituteBElumosudil for Bronchiolitis Obliterans Prevention/Therapy (BEBOP) | 2027-06-30 |
| NCT07135973SanofiA Study to Investigate Safety of Belumosudil in Participants Aged 12 Years and Above, With Chronic Graft-versus-host Disease (cGVHD) | 2027-08-30 |
Add all 3 to your calendar (.ics) → With an account.
Among the 14 studies testing belumosudil in Bronchiolitis Obliterans Syndrome, from their registry updates:
Among Sanofi’s 11 in Bronchiolitis Obliterans Syndrome:
This tab as a report to print or save as PDF → With an account.
This is a Phase 2, randomized, multicenter study to evaluate the efficacy and safety of KD025 in subjects with Chronic Graft Versus Host Disease (cGVHD) after at least 2 prior lines of systemic therapy
Phase 2, open label, randomized, multicenter study in subjects with cGVHD who have previously been treated with at least 2 prior lines of systemic therapy. Approximately 166 subjects with active cGVHD will be randomized (1:1) to receive treatment with one of two belumosudil (formerly known as KD025) regimens:
With Amendment 2, the sample size was increased from approximately 126 subjects, with additional subjects to be enrolled as follows:
These additional subjects will also be randomized (1:1) to Arm A or Arm B.
Any adolescent taking a proton pump inhibitor (PPI) or a strong CYP3A4 inducer will begin Cycle 1 Day 1 at the escalated dose of belumosudil 200 mg BID.
Randomization will be stratified according to prior cGVHD treatment with ibrutinib (Yes / No) and severe cGVHD at baseline (Yes / No). Subjects may receive treatment in 28-day treatment cycles until clinically significant progression of cGVHD. Subjects who have not achieved a response after 12 cycles of belumosudil should be withdrawn if in the Investigator's judgment there is no evidence of clinical benefit. Subjects will undergo evaluations as outlined in the Study Assessments table (Appendix A). The primary endpoint is the overall response rate (ORR) with responses as defined by the 2014 National Institute of Health (NIH) Consensus Development Project on clinical trials in cGVHD.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Eligible subjects randomized to arm A will take belumosudil 200 mg once daily
Drug: Belumosudil (KD025)
Eligible subjects randomized to arm B will take belumosudil 200 mg twice daily
Drug: Belumosudil (KD025)
Eligible subjects randomized to arm A will take belumosudil 200 mg once daily
Drug: Belumosudil (KD025)
Eligible subjects randomized to arm B will take belumosudil 200 mg twice daily
Drug: Belumosudil (KD025)
Belumosudil is an orally available Rho-associated protein kinase-2 (ROCK2) selective inhibitor.
Also known as: REZUROCK
Overall Response Rate (ORR)
The ORR was defined as the percentage of participants with a best response meeting the overall response criteria assessment of complete response (CR) or partial response (PR) at any post-baseline response assessment. CR was defined as resolution of all manifestations of cGVHD in each organ or site. PR was defined as the improvement in at least one organ or site without progression in any other organ or site. Responses were assessed by the 2014 National Institutes of Health (NIH) Consensus Development Project on Clinical Trials in cGVHD.
Time frame: From the date of randomization to the date of first documentation of progression or death due to any cause or data cut-off, whichever occurred first (maximum duration: up to 40.5 months for adult arms and 27.6 months for adolescent arms)
Duration of Response (DOR)
DOR is defined as the time from first documentation of response to the time of first documentation of deterioration from best response (e.g., CR to PR, or PR to Lack of response \[LR\]). CR was defined as resolution of all manifestations of cGVHD in each organ or site. PR was defined as the improvement in at least one organ or site without progression in any other organ or site. LR included the response status of mixed, unchanged, or progression. Mixed LR was defined as complete or partial response in at least one organ accompanied by progression in another organ. Unchanged LR was defined as outcomes that did not meet the criteria for complete response, partial response, progression or mixed response. Progression LR-P was defined as progression in at least one organ or site without a response in any other organ or site. Confidence interval (CI) is calculated using Kaplan-Meier method.
Time frame: From the date of randomization to the date of first documentation of progression or death due to any cause or data cut-off, whichever occurred first (maximum duration: up to 40.5 months for adult arms and 27.6 months for adolescent arms)
Number of Participants With Improvement (>=7-Point Reduction [7-PtR] From Baseline) as Assessed by Lee Symptom Scale (LSS) Score
The questionnaire asked participants to indicate the degree of bother that they experienced due to symptoms in 7 domains potentially affected by chronic GVHD. It consists of 30 items of 7 domains: skin, eyes and mouth, breathing, eating and digestion, muscles and joints, energy, and mental and emotional. Each question is scored 0, 1, 2, 3 or 4. A domain score was calculated for each domain by taking the mean of all items completed if more than 50% were answered and normalizing to a 0 to 100 scale. A summary score was calculated as average of all non-missing domain scores if more than 50% of them were non-missing. A higher score indicated more bothersome symptoms. A 7-point difference on the summary score of cGVHD symptom scale was found to be clinically meaningful.
Time frame: Baseline (Day 1) up to 40.5 months for adult arms; Baseline (Day 1) up to 27.6 months for adolescent arms
Number of Participants With Best Response by Organ System
The best response (CR, PR) for individual organs (skin, eyes, mouth, esophagus, upper gastrointestinal \[GI\], lower GI, liver, lungs, joints and fascia) were summarized. CR was defined as resolution of all manifestations of cGVHD in each organ or site. PR was defined as the improvement in at least one organ or site without progression in any other organ or site.
Time frame: From date of randomization until disease progression or data cut-off, whichever occurred first (maximum duration: up to 40.5 months for adult arms and 27.6 months for adolescent arms)
Percentage of Participants With Best Response of PR and CR
PR was defined as the improvement in at least one organ or site without progression in any other organ or site. CR was defined as resolution of all manifestations of cGVHD in each organ or site. Responses were assessed by the 2014 NIH Consensus Development Project on Clinical Trials in cGVHD.
Time frame: From the date of randomization to the date of first documentation of progression or death due to any cause or data cut-off, whichever occurred first (maximum duration: up to 40.5 months for adult arms and 27.6 months for adolescent arms)
Percent Change From Baseline in Corticosteroid Dose to Greatest Reduction
Change in corticosteroid doses was analyzed by using prednisone dose equivalents. If participants were not using prednisone as the systemic corticosteroid, then the prednisone dose equivalent would be determined according to following conversion ratios: 1 mg prednisone is equivalent to: 4.0 mg Hydrocortisone; 0.8 mg Methylprednisolone; 0.15 mg Dexamethasone; 1.0 mg Prednisolone and 0.8 mg Triamcinolone.
Time frame: Baseline (Day 1) and 40.5 months for adult arms; Baseline (Day 1) and 27.6 months for adolescent arms
Percentage of Participants With Reduction and Discontinuation of Calcineurin Inhibitor Dose
Calcineurin inhibitors include systemic tacrolimus and cyclosporine. Percentage of participants with reduction and discontinuation of calcineurin inhibitor dose is presented.
Time frame: Baseline (Day 1) up to 40.5 months for adult arms; Baseline (Day 1) up to 27.6 months for adolescent arms
Failure-Free Survival (FFS)
FFS was defined as the absence of new cGVHD systemic therapy, non-relapse mortality and recurrent malignancy (i.e. underlying disease) and was censored by last response assessment or long term follow up assessment, whichever was the latest and available.
Time frame: From first dose of study drug to the time of first documentation of response or data cut-off, whichever occurred first (maximum duration: up to 40.5 months for adult arms and 27.6 months for adolescent arms)
Overall Survival (OS)
OS was defined as time from first dose of belumosudil to the date of death due to any cause.
Time frame: From first dose of study drug to date of death from any cause or data cut-off, whichever occurred first (maximum duration: up to 40.5 months for adult arms and 27.6 months for adolescent arms)
Number of Participants With Best Response of Global Severity Rating Score as Based on the Clinician-Reported Global cGVHD Activity Assessment
The Clinician-reported global cGVHD Activity Assessment is a 0-10 point numeric rating scale with a score of 0 indicating "cGVHD symptoms not at all severe" and a score of 10 being "most severe cGVHD symptoms possible". Higher scores indicated worse symptoms. Best response was defined as PR+CR. PR was defined as the improvement in at least one organ or site without progression in any other organ or site. CR was defined as resolution of all manifestations of cGVHD in each organ or site. Participants were categorized in 3 categories at baseline based on the global severity scores of \<6, =6-7 and \>7 and number of participants with best response for them is reported.
Time frame: Baseline (Day 1) up to 40.5 months for adult arms; Baseline (Day 1) up to 27.6 months for adolescent arms
Change From Baseline in Patient Self-Reported Symptom Activity Based on cGVHD Activity Assessment
The symptom activity item is a 0-10-point numeric rating scale with a score of 0 indicating "cGVHD symptoms not at all severe" and a score of 10 being "most severe cGVHD symptoms possible". The status reported by participants were categorized as none, mild, moderate, and severe. Higher scores indicated worse symptoms. Baseline was defined as the valid and last non-missing value obtained within 14 days prior to participants receiving the first study drug.
Time frame: Baseline (Day 1) and 40.5 months for adult arms; Baseline (Day 1) and 27.6 months for adolescent arms
Maximum Concentration Observed (Cmax) of Belumosudil
Blood samples were collected at the specified timepoints to evaluate Cmax of belumosudil. As pre-specified in protocol, pharmacokinetic (PK) parameters were only calculated for participants with full PK samples. Thus, PK parameters were not calculated for adolescent participants who only had sparse PK samples.
Time frame: Cycles 1 and 2: Pre-dose and 1, 2, 3, 4, 5, 6, 7, 8, and 12 hours post dose on Day 1 for adult arms; Cycles 2 and 4: Pre-dose and 3 and 5 hours post dose on Day 1 for adolescent arms
Observed Time to Reach Peak Plasma Concentration (Tmax) of Belumosudil
Blood samples were collected at the specified timepoints to evaluate Tmax of belumosudil. As pre-specified in protocol, PK parameters were only calculated for participants with full PK samples. Thus, PK parameters were not calculated for adolescent participants who only had sparse PK samples.
Time frame: Cycles 1 and 2: Pre-dose and 1, 2, 3, 4, 5, 6, 7, 8, and 12 hours post dose on Day 1 for adult arms; Cycles 2 and 4: Pre-dose and 3 and 5 hours post dose on Day 1 for adolescent arms
Area Under the Curve Over Time Interval From 0 to 6 Hours (AUC0-6) of Belumosudil
Blood samples were collected at the specified timepoints to evaluate AUC0-6 of belumosudil. As pre-specified in protocol, PK parameters were only calculated for participants with full PK samples. Thus, PK parameters were not calculated for adolescent participants who only had sparse PK samples.
Time frame: Cycles 1 and 2: Pre-dose and 1, 2, 3, 4, 5, 6, 7, 8, and 12 hours post dose on Day 1 for adult arms; Cycles 2 and 4: Pre-dose and 3 and 5 hours post dose on Day 1 for adolescent arms
Time to Response (TTR)
TTR was measured as the time from first treatment to the time of first documentation of response.
Time frame: From first dose of study drug to the time of first documentation of response or data cut-off, whichever occurred first(maximum duration: up to 40.5 months for adult arms and 27.6 months for adolescent arms)
Time to Next Treatment (TTNT)
The TTNT was measured as the time from first treatment to the time of new systemic cGVHD treatment, censored by last response assessment or long term follow up assessment, whichever was the latest and available.
Time frame: From first dose of study drug to the time of new treatment or censoring date, whichever occurred first (maximum duration: up to 40.5 months for adult arms and 27.6 months for adolescent arms)
The study was conducted at 33 centers in the United States. A total of 251 participants were screened between 11 October 2018 to 08 August 2023, of which 92 participants were screen failures in adult cohort due to not meeting eligibility criteria, there were no screen failures in adolescent cohort.
| Milestone | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Started | 78 | 78 | 2 | 1 |
| Completed | 0 | 0 | 0 | 0 |
| Not completed | 78 | 78 | 2 | 1 |
| Withdrew: Death | 14 | 14 | 0 | 0 |
| Withdrew: Lost to follow-up | 2 | 0 | 0 | 0 |
| Withdrew: Site terminated by sponsor | 1 | 3 | 0 | 0 |
| Withdrew: Study terminated by sponsor | 29 | 42 | 1 | 0 |
| Withdrew: Withdrawal by subject | 5 | 4 | 0 | 1 |
| Withdrew: Other | 26 | 12 | 1 | 0 |
| Withdrew: Randomized, but not treated | 1 | 3 | 0 | 0 |
The ORR was defined as the percentage of participants with a best response meeting the overall response criteria assessment of complete response (CR) or partial response (PR) at any post-baseline response assessment. CR was defined as resolution of all manifestations of cGVHD in each organ or site. PR was defined as the improvement in at least one organ or site without progression in any other organ or site. Responses were assessed by the 2014 National Institutes of Health (NIH) Consensus Development Project on Clinical Trials in cGVHD.
| Percentage of participants | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Overall Response Rate (ORR) | 74.0 | 76.0 | 50.0 | 100.0 |
DOR is defined as the time from first documentation of response to the time of first documentation of deterioration from best response (e.g., CR to PR, or PR to Lack of response \[LR\]). CR was defined as resolution of all manifestations of cGVHD in each organ or site. PR was defined as the improvement in at least one organ or site without progression in any other organ or site. LR included the response status of mixed, unchanged, or progression. Mixed LR was defined as complete or partial response in at least one organ accompanied by progression in another organ. Unchanged LR was defined as outcomes that did not meet the criteria for complete response, partial response, progression or mixed response. Progression LR-P was defined as progression in at least one organ or site without a response in any other organ or site. Confidence interval (CI) is calculated using Kaplan-Meier method.
| Weeks | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Duration of Response (DOR) | 23.9 (11.43 to 50.43) | 32.0 (20.86 to 53.14) | 16.1 (NA to NA) | 4.1 (NA to NA) |
The questionnaire asked participants to indicate the degree of bother that they experienced due to symptoms in 7 domains potentially affected by chronic GVHD. It consists of 30 items of 7 domains: skin, eyes and mouth, breathing, eating and digestion, muscles and joints, energy, and mental and emotional. Each question is scored 0, 1, 2, 3 or 4. A domain score was calculated for each domain by taking the mean of all items completed if more than 50% were answered and normalizing to a 0 to 100 scale. A summary score was calculated as average of all non-missing domain scores if more than 50% of them were non-missing. A higher score indicated more bothersome symptoms. A 7-point difference on the summary score of cGVHD symptom scale was found to be clinically meaningful.
| Participants | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Number of Participants With Improvement (>=7-Point Reduction [7-PtR] From Baseline) as Assessed by Lee Symptom Scale (LSS) Score | 45 | 48 | 0 | 0 |
The best response (CR, PR) for individual organs (skin, eyes, mouth, esophagus, upper gastrointestinal \[GI\], lower GI, liver, lungs, joints and fascia) were summarized. CR was defined as resolution of all manifestations of cGVHD in each organ or site. PR was defined as the improvement in at least one organ or site without progression in any other organ or site.
| Participants | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Skin | 20 | 28 | 1 | — |
| Eyes | 24 | 26 | — | 1 |
| Mouth | 23 | 31 | 1 | 1 |
| Esophagus | 13 | 7 | — | — |
| Upper GI | 9 | 4 | — | — |
| Lower GI | 5 | 6 | — | — |
| Liver | 2 | 1 | — | — |
| Lungs | 9 | 6 | 0 | — |
| Joints and Fascia | 42 | 39 | — | — |
PR was defined as the improvement in at least one organ or site without progression in any other organ or site. CR was defined as resolution of all manifestations of cGVHD in each organ or site. Responses were assessed by the 2014 NIH Consensus Development Project on Clinical Trials in cGVHD.
| Percentage of participants | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| PR | 68.8 | 73.3 | 50.0 | 100.0 |
| CR | 5.2 | 2.7 | 0 | 0 |
Change in corticosteroid doses was analyzed by using prednisone dose equivalents. If participants were not using prednisone as the systemic corticosteroid, then the prednisone dose equivalent would be determined according to following conversion ratios: 1 mg prednisone is equivalent to: 4.0 mg Hydrocortisone; 0.8 mg Methylprednisolone; 0.15 mg Dexamethasone; 1.0 mg Prednisolone and 0.8 mg Triamcinolone.
| Percent change | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Percent Change From Baseline in Corticosteroid Dose to Greatest Reduction | -50.00 (-100.0 to 0.0) | -66.67 (-100.0 to 900.0) | -50.00 (-100.0 to 0.0) | -60.00 (-60.0 to -60.0) |
Calcineurin inhibitors include systemic tacrolimus and cyclosporine. Percentage of participants with reduction and discontinuation of calcineurin inhibitor dose is presented.
| Percentage of participants | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Reduction of Calcineurin Inhibitor Dose | 46.9 | 57.7 | 100.0 | — |
| Discontinuation of Calcineurin Inhibitor Dose | 21.9 | 34.6 | 0 | — |
FFS was defined as the absence of new cGVHD systemic therapy, non-relapse mortality and recurrent malignancy (i.e. underlying disease) and was censored by last response assessment or long term follow up assessment, whichever was the latest and available.
| Months | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Failure-Free Survival (FFS) | 16.3 (10.15 to 26.48) | 17.2 (11.27 to NA) | NA (NA to NA) | 6.8 (NA to NA) |
OS was defined as time from first dose of belumosudil to the date of death due to any cause.
| Months | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Overall Survival (OS) | NA (NA to NA) | NA (NA to NA) | NA (NA to NA) | NA (NA to NA) |
The Clinician-reported global cGVHD Activity Assessment is a 0-10 point numeric rating scale with a score of 0 indicating "cGVHD symptoms not at all severe" and a score of 10 being "most severe cGVHD symptoms possible". Higher scores indicated worse symptoms. Best response was defined as PR+CR. PR was defined as the improvement in at least one organ or site without progression in any other organ or site. CR was defined as resolution of all manifestations of cGVHD in each organ or site. Participants were categorized in 3 categories at baseline based on the global severity scores of \<6, =6-7 and \>7 and number of participants with best response for them is reported.
| Participants | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Baseline global severity rating score < 6: Best response | 9 | 8 | 0 | 0 |
| Baseline global severity rating score= 6-7: Best response | 15 | 28 | 1 | — |
| Baseline global severity rating score >7: Best response | 11 | 9 | — | — |
The symptom activity item is a 0-10-point numeric rating scale with a score of 0 indicating "cGVHD symptoms not at all severe" and a score of 10 being "most severe cGVHD symptoms possible". The status reported by participants were categorized as none, mild, moderate, and severe. Higher scores indicated worse symptoms. Baseline was defined as the valid and last non-missing value obtained within 14 days prior to participants receiving the first study drug.
| Score on a scale | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Change From Baseline in Patient Self-Reported Symptom Activity Based on cGVHD Activity Assessment | -0.9 ± 2.4 | -1.5 ± 2.4 | 0.0 ± 0.0 | -1.0 ± NA |
Blood samples were collected at the specified timepoints to evaluate Cmax of belumosudil. As pre-specified in protocol, pharmacokinetic (PK) parameters were only calculated for participants with full PK samples. Thus, PK parameters were not calculated for adolescent participants who only had sparse PK samples.
| Nanograms per milliliter (ng/mL) | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Cycle 1 | 1350 ± 127 | 870 ± 177 | — | — |
| Cycle 2 | 1780 ± 154 | 2050 ± 99.9 | NA ± NA | NA ± NA |
| Cycle 4 | — | — | NA ± NA | NA ± NA |
Blood samples were collected at the specified timepoints to evaluate Tmax of belumosudil. As pre-specified in protocol, PK parameters were only calculated for participants with full PK samples. Thus, PK parameters were not calculated for adolescent participants who only had sparse PK samples.
| Hours | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Cycle 1 | 2.90 (1.10 to 5.95) | 4.00 (1.18 to 7.75) | — | — |
| Cycle 2 | 1.98 (1.88 to 2.07) | 1.26 (1.00 to 4.03) | NA (NA to NA) | NA (NA to NA) |
| Cycle 4 | — | — | NA (NA to NA) | NA (NA to NA) |
Blood samples were collected at the specified timepoints to evaluate AUC0-6 of belumosudil. As pre-specified in protocol, PK parameters were only calculated for participants with full PK samples. Thus, PK parameters were not calculated for adolescent participants who only had sparse PK samples.
| Hours*ng/mL | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Cycle 1 | 4430 ± 153 | 2380 ± 204 | — | — |
| Cycle 2 | 6860 ± 123 | 6520 ± 108 | NA ± NA | NA ± NA |
| Cycle 4 | — | — | NA ± NA | NA ± NA |
TTR was measured as the time from first treatment to the time of first documentation of response.
| Weeks | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Time to Response (TTR) | 4.43 (3.7 to 80.1) | 4.43 (3.7 to 40.1) | 7.57 (7.5 to 7.6) | 4.14 (4.1 to 4.2) |
The TTNT was measured as the time from first treatment to the time of new systemic cGVHD treatment, censored by last response assessment or long term follow up assessment, whichever was the latest and available.
| Months | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| Time to Next Treatment (TTNT) | 24.2 (13.37 to NA) | NA (14.55 to NA) | NA (NA to NA) | 6.8 (NA to NA) |
Collected over Treatment-emergent adverse events (TEAEs): From first dose of study drug to the date of first documentation of progression or death due to any cause or data cut-off, whichever occurred first (maximum duration: up to 40.5 months for adult arms and 27.6 months for adolescent arms). All-cause mortality (deaths) were collected from first dose of study drug (Day 1) up to end of study, approximately 62 months.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Adult Arm A: Belumosudil 200 mg QD | 14/77 (18.2%) | 36/77 (46.8%) | 73/77 (94.8%) |
| Adult Arm B: Belumosudil 200 mg BID | 14/75 (18.7%) | 30/75 (40%) | 73/75 (97.3%) |
| Adolescent Arm A: Belumosudil 200 mg QD | 0/2 (0%) | 1/2 (50%) | 2/2 (100%) |
| Adolescent Arm B: Belumosudil 200 mg BID | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Event | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| PneumoniaInfections and infestations | 7/77 | 7/75 | 1/2 | 0/1 |
| HypersensitivityImmune system disorders | 0/77 | 0/75 | 1/2 | 0/1 |
| Steroid DiabetesMetabolism and nutrition disorders | 0/77 | 0/75 | 1/2 | 0/1 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 0/77 | 1/75 | 1/2 | 0/1 |
| Rash Maculo-PapularSkin and subcutaneous tissue disorders | 0/77 | 0/75 | 1/2 | 0/1 |
| Face OedemaGeneral disorders | 1/77 | 0/75 | 1/2 | 0/1 |
| Coronavirus InfectionInfections and infestations | 0/77 | 3/75 | 0/2 | 0/1 |
| PyrexiaGeneral disorders | 3/77 | 1/75 | 0/2 | 0/1 |
| Urinary Tract InfectionInfections and infestations | 0/77 | 2/75 | 0/2 | 0/1 |
| AnaemiaBlood and lymphatic system disorders | 0/77 | 2/75 | 0/2 | 0/1 |
| Event | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID |
|---|---|---|---|---|
| ConjunctivitisInfections and infestations | 6/77 | 2/75 | 0/2 | 1/1 |
| Cytomegalovirus Infection ReactivationInfections and infestations | 0/77 | 1/75 | 0/2 | 1/1 |
| ParonychiaInfections and infestations | 0/77 | 0/75 | 1/2 | 1/1 |
| EosinophiliaBlood and lymphatic system disorders | 0/77 | 1/75 | 1/2 | 1/1 |
| HyperkalaemiaMetabolism and nutrition disorders | 3/77 | 7/75 | 0/2 | 1/1 |
| HyperuricaemiaMetabolism and nutrition disorders | 1/77 | 3/75 | 1/2 | 1/1 |
| HypophosphataemiaMetabolism and nutrition disorders | 6/77 | 9/75 | 0/2 | 1/1 |
| Vision BlurredEye disorders | 5/77 | 4/75 | 0/2 | 1/1 |
| Sinus TachycardiaCardiac disorders | 4/77 | 3/75 | 0/2 | 1/1 |
| DysphagiaGastrointestinal disorders | 14/77 | 4/75 | 0/2 | 1/1 |
Modified intent-to-treat (mITT) population included all randomized participants who received at least one dose of study drug.
| Age, Customized(Participants) | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID | Total |
|---|---|---|---|---|---|
| 12-17 Years | 0 | 0 | 2 | 1 | 3 |
| 18-77 Years | 77 | 75 | 0 | 0 | 152 |
| Sex: Female, Male(Participants) | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID | Total |
|---|---|---|---|---|---|
| Female | 28 | 38 | 0 | 1 | 67 |
| Male | 49 | 37 | 2 | 0 | 88 |
| Race (NIH/OMB)(Participants) | Adult Arm A: Belumosudil 200 mg QD | Adult Arm B: Belumosudil 200 mg BID | Adolescent Arm A: Belumosudil 200 mg QD | Adolescent Arm B: Belumosudil 200 mg BID | Total |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 2 | 0 | 0 | 2 |
| Asian | 2 | 2 | 0 | 0 | 4 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 7 | 1 | 1 | 0 | 9 |
| White | 65 | 67 | 1 | 1 | 134 |
| More than one race | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 3 | 3 | 0 | 0 | 6 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: 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.
Ran from Oct 2018 to primary completion in Dec 2023, 62 months.
Results were first posted in Dec 2024, 1 year after primary completion.
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Studies of the drug tested here: Belumosudil
Bronchiolitis Obliterans Syndrome →