A Study to Test an Oral Medicine, Belumosudil, in Combination With Corticosteroids in Participants at Least 12 Years of Age With Newly Diagnosed Chronic Graft Versus Host Disease. (ROCKnrol-1)

A Randomized, Double-blind, Multicenter, Phase 3 Study to Evaluate Efficacy and Safety of Belumosudil in Combination With Corticosteroids Versus Placebo in Combination With Corticosteroids in Participants at Least 12 Years of Age With Newly Diagnosed Chronic Graft Versus Host Disease (cGVHD)

This is a parallel, Phase 3, two-arm study for the treatment of newly diagnosed moderate or severe chronic GVHD.

The study duration for a participant includes up to 4 weeks for screening; a treatment period until clinically meaningful cGVHD progression (defined as progression requiring addition of new systemic treatment for cGVHD), relapse/recurrence of the underlying disease, participant starts new systemic treatment for cGVHD or experiences an unacceptable toxicity, at the request of the participants or the investigators, or until the end of study is reached, whichever comes first; at least 30 days follow-up of adverse events (AEs) after the last dose until resolution or stabilization, if applicable; and long-term follow-up until death or study close-out, whichever comes first.

Study Overview

• Status: Active, not recruiting
• Conditions: Chronic Graft Versus Host Disease
• Intervention / Treatment: Drug: Belumosudil; Drug: Prednisone; Drug: Prednisolone; Drug: Placebo

Detailed Description

Up to 2.5 years

• Study Type: Interventional
• Enrollment (Estimated): 260
• Phase: Phase 3

Contacts and Locations

Study Locations

• Argentina
  • Buenos Aires, Argentina, 1426
    • Investigational Site Number : 0320001
  • Buenos Aires, Argentina, 1227
    • Investigational Site Number : 0320005
  • Buenos Aires, Argentina, 1280
    • Investigational Site Number : 0320003
  • Buenos Aires, Argentina, 1431
    • Investigational Site Number : 0320002
  • Córdoba, Argentina, 5000
    • Investigational Site Number : 0320004
  • Buenos Aires
    • Pilar, Buenos Aires, Argentina, 1629
      • Investigational Site Number : 0320006
• Australia
  • New South Wales
    • Westmead, New South Wales, Australia, 2145
      • Investigational Site Number : 0360005
  • Queensland
    • Brisbane, Queensland, Australia, 4029
      • Investigational Site Number : 0360003
  • Victoria
    • Melbourne, Victoria, Australia, 3000
      • Investigational Site Number : 0360001
  • Western Australia
    • Murdoch, Western Australia, Australia, 6150
      • Investigational Site Number : 0360004
• Austria
  • Graz, Austria, 8036
    • Investigational Site Number : 0400003
  • Linz, Austria, 4020
    • Investigational Site Number : 0400001
  • Vienna, Austria, 1090
    • Investigational Site Number : 0400004
• Belgium
  • Bruges, Belgium, 8000
    • Investigational Site Number : 0560006
  • Ghent, Belgium, 9000
    • UZ Gent-Investigational Site Number : 0560003
  • Liège, Belgium, 4000
    • CHU de Liège-Investigational Site Number : 0560004
  • Roeselare, Belgium, 8800
    • AZ Delta-Investigational Site Number : 0560002
  • Brussels Capital
    • Woluwe, Brussels Capital, Belgium, 1200
      • Cliniques Universitaires St. Luc-Investigational Site Number : 0560005
  • Vlaams-Brabant
    • Leuven, Vlaams-Brabant, Belgium, 3000
      • UZ Leuven-Investigational Site Number : 0560001
• Brazil
  • Rio de Janeiro, Brazil, 20230-130
    • INCA - Hospital do Câncer - Rio de Janeiro - Praça Da Cruz Vermelha- Site Number : 0760007
  • São Paulo, Brazil, 05403-000
    • Hospital das Clinicas FMUSP- Site Number : 0760004
  • São Paulo, Brazil, 05651-901
    • Hospital Israelita Albert Einstein- Site Number : 0760003
  • Paraná
    • Curitiba, Paraná, Brazil, 80060-900
      • Hospital de Clinicas da Universidade Federal do Parana- Site Number : 0760008
  • São Paulo
    • São José do Rio Preto, São Paulo, Brazil, 15090-000
      • Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760006
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N 5G2
      • Investigational Site Number : 1240007
  • Ontario
    • Toronto, Ontario, Canada, M5G 2M9
      • Investigational Site Number : 1240005
  • Quebec
    • Montreal, Quebec, Canada, H4a 3j1
      • Investigational Site Number : 1240002
    • Montreal, Quebec, Canada, H1T 2M4
      • Hôpital Maisonneuve Rosemont-Investigational Site Number : 1240001
    • Montreal, Quebec, Canada, H3T1C5
      • CHU Sainte-Justine-Site Number : 1240003
  • Saskatchewan
    • Saskatoon, Saskatchewan, Canada, S7N 4H4
      • Investigational Site Number : 1240008
• China
  • Beijing, China, 100050
    • Investigational Site Number : 1560019
  • Changchun, China, 130021
    • Investigational Site Number : 1560012
  • Changsha, China, 410008
    • Investigational Site Number : 1560010
  • Fuzhou, China, 350001
    • Investigational Site Number : 1560011
  • Guangzhou, China, 510000
    • Investigational Site Number : 1560005
  • Hangzhou, China, 310003
    • Investigational Site Number : 1560002
  • Hefei, China, 230001
    • Investigational Site Number : 1560014
  • Jinan, China, 250001
    • Investigational Site Number : 1560008
  • Jinan, China, 250014
    • Investigational Site Number : 1560007
  • Suzhou, China, 215006
    • Investigational Site Number : 1560001
  • Suzhou, China, 215025
    • Investigational Site Number : 1560006
  • Tianjin, China, 300020
    • Investigational Site Number : 1560003
  • Tianjin, China, 300020
    • Investigational Site Number : 1560004
  • Wuhan, China, 430022
    • Investigational Site Number : 1560013
  • Wuhan, China, 430030
    • Investigational Site Number : 1560015
  • Zhengzhou, China, 450008
    • Investigational Site Number : 1560009
• Czechia
  • Brno, Czechia, 62500
    • Interni hematologicka a onkologicka klinika Fakultni nemocnice Brno-Site Number : 2030003
  • Hradec Králové, Czechia, 50005
    • IV. Interni hematologicka klinika, Fakultni nemocnice Hradec Kralove-Site Number : 2030002
  • Ostrava, Czechia, 70852
    • Klinika hematoonkologie, Fakultni nemocnice Ostrava-Site Number : 2030001
  • Prague, Czechia, 128 00
    • Ustav hematologie a krevni transfuze-Site Number : 2030004
• Denmark
  • Capital
    • Copenhagen, Capital, Denmark, 2100
      • Rigshospitalet Copenhagen University Hospital-Investigational Site Number : 2080002
  • Funen
    • Odense, Funen, Denmark, 5000
      • Odense University Hospital-Investigational Site Number : 2080003
  • Jutland
    • Aarhus, Jutland, Denmark, 8200
      • Department of hematology-Investigational Site Number : 2080001
• France
  • Marseille, France, 13273
    • Institut Paoli Calmettes-Investigational Site Number : 2500004
  • Nantes, France, 44093
    • CHU Hotel Dieu-Investigational Site Number : 2500005
  • Paris, France, 75010
    • Investigational Site Number : 2500003
  • Paris, France, 75571
    • Investigational Site Number : 2500001
  • Pessac, France, 33600
    • CHU Bordeaux-Hôpital Haut-Lévêque-Investigational Site Number : 2500002
  • Pierre-Bénite, France, 69310
    • Hôpital Lyon Sud-Investigational Site Number : 2500007
  • Rennes, France, 35033
    • Hôpital Pontchaillou - CHU de Rennes-Investigational Site Number : 2500008
  • Vandœuvre-lès-Nancy, France, 54511
    • Investigational Site Number : 2500006
• Germany
  • Berlin, Germany, 13353
    • Investigational Site Number : 2760007
  • Cologne, Germany, 50937
    • Investigational Site Number : 2760003
  • Dresden, Germany, 01307
    • Investigational Site Number : 2760001
  • Freiburg im Breisgau, Germany, 79106
    • Investigational Site Number : 2760005
  • Hamburg, Germany, 20246
    • Investigational Site Number : 2760002
  • Kiel, Germany, 24105
    • Investigational Site Number : 2760010
  • Münster, Germany, 48149
    • Investigational Site Number : 2760009
  • Regensburg, Germany, 93053
    • Investigational Site Number : 2760006
  • Tübingen, Germany, 72076
    • Investigational Site Number : 2760008
• Greece
  • Athens, Greece, 115 27
    • Nosokomeio Paidon I Agia Sofia-Site Number : 3000002
  • Thessaloniki, Greece, 570 10
    • General Hospital of Thessaloniki "George Papanikolaou"-Site Number : 3000001
• Hong Kong
  • Pok Fu Lam, Hong Kong, 999077
    • Investigational Site Number : 3440001
• Israel
  • Haifa, Israel, 3109601
    • Investigational Site Number : 3760002
  • Jerusalem, Israel, 9112001
    • Investigational Site Number : 3760005
  • Petah Tikva, Israel, 4920235
    • Investigational Site Number : 3760006
  • Ramat Gan, Israel, 5262100
    • Investigational Site Number : 3760003
  • Ramat Gan, Israel, 5262100
    • Investigational Site Number : 3760004
  • Tel Aviv, Israel, 6423906
    • Investigational Site Number : 3760001
• Italy
  • Bergamo, Italy, 24127
    • Investigational Site Number : 3800003
  • Bologna, Italy, 40138
    • Investigational Site Number : 3800004
  • Pavia, Italy, 27100
    • Investigational Site Number : 3800011
  • Perugia, Italy, 06156
    • Investigational Site Number : 3800010
  • Reggio Calabria, Italy, 89133
    • Investigational Site Number : 3800006
  • Udine, Italy, 33100
    • Investigational Site Number : 3800008
  • Ancona
    • Torette, Ancona, Italy, 60020
      • Investigational Site Number : 3800012
  • Genova
    • Genoa, Genova, Italy, 16132
      • Investigational Site Number : 3800005
  • Milano
    • Milan, Milano, Italy, 20132
      • Investigational Site Number : 3800009
    • Rozzano, Milano, Italy, 20089
      • Investigational Site Number : 3800002
  • Roma
    • Rome, Roma, Italy, 00168
      • Investigational Site Number : 3800001
  • Torino
    • Turin, Torino, Italy, 10126
      • Investigational Site Number : 3800007
• Netherlands
  • Amsterdam, Netherlands, 1081 HV
    • Amsterdam Universitair Medisch Centrum-Site Number : 5280005
  • Groningen, Netherlands, 9713 GR
    • Investigational Site Number : 5280003
  • Rotterdam, Netherlands, 3015 CE
    • Investigational Site Number : 5280002
  • Utrecht, Netherlands, 3584 CS
    • Investigational Site Number : 5280001
• Poland
  • Szczecin, Poland, 71-252
    • Investigational Site Number : 6160008
  • Pomeranian Voivodeship
    • Gdansk, Pomeranian Voivodeship, Poland, 80-952
      • Investigational Site Number : 6160001
• Portugal
  • Lisbon, Portugal, 1099-023
    • Investigational Site Number : 6200003
  • Lisbon, Portugal, 1649-035
    • Investigational Site Number : 6200002
• South Korea
  • Seoul-teukbyeolsi
    • Seoul, Seoul-teukbyeolsi, South Korea, 03080
      • Investigational Site Number : 4100002
    • Seoul, Seoul-teukbyeolsi, South Korea, 03722
      • Investigational Site Number : 4100003
    • Seoul, Seoul-teukbyeolsi, South Korea, 06351
      • Investigational Site Number : 4100004
    • Seoul, Seoul-teukbyeolsi, South Korea, 06591
      • Investigational Site Number : 4100001
• Spain
  • Madrid, Spain, 28007
    • Investigational Site Number : 7240004
  • Madrid, Spain, 28009
    • Hospital Niño Jesús-Site Number : 7240002
  • Málaga, Spain, 29010
    • Hospital Regional Universitarioa de Malaga-Site Number : 7240003
  • Salamanca, Spain, 37007
    • Hospital Universitario de Salamanca-Site Number : 7240007
  • Seville, Spain, 41013
    • Hospital Universitario Virgen del Rocío.-Site Number : 7240008
  • Valencia, Spain, 46010
    • Investigational Site Number : 7240012
  • Valencia, Spain, 46026
    • Hospital Universitari i Politècnic La Fe-Site Number : 7240001
  • Cantabria
    • Santander, Cantabria, Spain, 39008
      • Investigational Site Number : 7240011
  • Catalunya [Cataluña]
    • Barcelona, Catalunya [Cataluña], Spain, 08036
      • Investigational Site Number : 7240005
    • Barcelona, Catalunya [Cataluña], Spain, 08908
      • ICO Institut Català d'Oncologia-Site Number : 7240006
• Sweden
  • Gothenburg, Sweden, 413 45
    • Investigational Site Number : 7520003
  • Huddinge, Sweden, 141 57
    • Investigational Site Number : 7520001
  • Lund, Sweden, 221 85
    • Investigational Site Number : 7520002
• Turkey (Türkiye)
  • Adana, Turkey (Türkiye), 01120
    • Investigational Site Number : 7920006
  • Ankara, Turkey (Türkiye), 06200
    • Investigational Site Number : 7920002
  • Ankara, Turkey (Türkiye), 06560
    • Investigational Site Number : 7920004
  • Ankara, Turkey (Türkiye), 06620
    • Investigational Site Number : 7920001
  • Istanbul, Turkey (Türkiye), 34010
    • Investigational Site Number : 7920005
  • Izmir, Turkey (Türkiye), 35100
    • Investigational Site Number : 7920003
• United Kingdom
  • Leeds, United Kingdom, LS9 7TF
    • Investigational Site Number : 8260003
  • Manchester, United Kingdom, M20 4BX
    • Investigational Site Number : 8260001
  • Newcastle upon Tyne, United Kingdom, NE7 7DN
    • Investigational Site Number : 8260004
  • Cardiff [Caerdydd Gb-crd]
    • Cardiff, Cardiff [Caerdydd Gb-crd], United Kingdom, CF14 4XW
      • Investigational Site Number : 8260006
  • London, City of
    • London, London, City of, United Kingdom, SE5 9RL
      • Investigational Site Number : 8260002
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • University of Arkansas for Medical Sciences-Site Number : 8400019
  • California
    • Duarte, California, United States, 91010
      • City of Hope National Medical Center- Site Number : 8400001
    • San Francisco, California, United States, 94143
      • University of California San Francisco - Parnassus Heights- Site Number : 8400035
  • Florida
    • Orlando, Florida, United States, 32803
      • AdventHealth Orlando- Site Number : 8400023
  • Georgia
    • Decatur, Georgia, United States, 30030
      • The Hope Clinic of Emory University- Site Number : 8400020
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University- Site Number : 8400017
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University Health University Hospital- Site Number : 8400006
  • Kentucky
    • Lexington, Kentucky, United States, 40536
      • University of Kentucky Chandler Medical Center- Site Number : 8400024
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins Hospital- Site Number : 8400033
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Dana Farber Cancer Institute Site Number : 8400005
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Karmanos Cancer Institute - Detroit- Site Number : 8400013
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • UNC Children's Hospital- Site Number : 8400025
    • Winston-Salem, North Carolina, United States, 27157
      • Atrium Health Wake Forest Baptist Medical Center - Winston-Salem - Medical Center Boulevard- Site Number : 8400007
  • Ohio
    • Cincinnati, Ohio, United States, 45236
      • Oncology Hematology Care - Kenwood- Site Number : 8400030
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Comprehensive Cancer Center (OSUCCC) - The James Cancer Hospital and Solove Research Institute- Site Number : 8400026
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science University- Site Number : 8400027
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15232
      • UPMC Hillman Cancer Center- Site Number : 8400008
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Sarah Cannon Research Institute Site Number : 8400003
  • Texas
    • Austin, Texas, United States, 78704
      • St. David's South Austin Medical Center- Site Number : 8400002
    • Dallas, Texas, United States, 75246
      • Texas Oncology - Dallas - Worth Street- Site Number : 8400010
    • San Antonio, Texas, United States, 78229
      • Texas Transplant Institute - Methodist Hospital- Site Number : 8400037
  • Virginia
    • Charlottesville, Virginia, United States, 22903
      • University of Virginia Comprehensive Cancer Center-Site Number : 8400031
  • Washington
    • Seattle, Washington, United States, 98109
      • Fred Hutchinson Cancer Research Center- Site Number : 8400004
  • Wisconsin
    • Madison, Wisconsin, United States, 53792
      • University of Wisconsin Carbone Cancer Center-Site Number : 8400029

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients must be at least 12 years of age inclusive, at the time of signing the informed consent
• Participants who have undergone allogenic HCT with newly diagnosed moderate to severe cGVHD according to NIH consensus diagnosis and staging criteria (2014)
• Participants who require systemic treatment with corticosteroids for cGVHD
• Participants who have not received any prior systemic treatment for cGVHD (including ECP)
• If participants are receiving other immunosuppressive agents for the prophylaxis or treatment of acute GVHD, the dose should be under the threshold pre-defined in protocol
• For adult participants, the body weight should be ≥40 kg. For adolescent participants, the body weight should be ≥30 kg
• Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• Participants or their legally authorized representative must be capable of giving signed informed consent

Exclusion Criteria:

Participants are excluded from the study if any of the following criteria apply:

Medical conditions

• Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of progressive or relapsed underlying disease after the most recent allogeneic HCT
• Post-transplant lymphoproliferative disease within 4 weeks prior to randomization
• Female participants who are pregnant or breastfeeding
• Unable to tolerate a prednisone equivalent dose of corticosteroids ≥ 1 mg/kg/day Prior/concomitant therapy
• Participant has had previous exposure to belumosudil.
• Received any previous systemic treatment for cGVHD with the following exception: Corticosteroids for cGVHD received within 7 days prior to the planned administration of IMP only if in the interest of participant.

Prior/concurrent clinical study experience

• Received any investigational agents, or any investigational device or procedure, or prohibited therapy for this study within 28 days or 5 elimination half-lives prior to randomization, whichever is longer Diagnostic assessments
• Karnofsky (if aged ≥16 years)/Lansky (if aged <16 years) Performance Score of < 60
• Platelets <25 x 109/L. Platelet transfusion is not allowed within 3 days before the screening hematological test
• Absolute neutrophil count (ANC) <0.5 x 109/L. The use of granulocyte-colony stimulating factor (G-CSF) is not allowed to reach this level during screening
• Estimated Glomerular Filtration Rate (eGFR) <30 mL/min/1.73 m2 using the MDRD-4 variable formula (if aged ≥18 years) or using the Bedside Schwartz formula (if aged <18 years)
• Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) >3 x ULN without liver cGVHD or>5 × ULN with liver) cGVHD
• Total bilirubin >1.5 × (ULN) (>3 × ULN if Gilbert syndrome)
• Participant has forced expiratory volume in 1 second (FEV1) of predicted ≤39% or has lung score of 3 according to NIH consensus diagnostic and staging criteria (2014)
• 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)
• Known history of human immunodeficiency virus (HIV)
• Active viral disease including hepatitis B virus (HBV) or hepatitis C virus (HCV)
• Active uncontrolled cytomegalovirus (CMV) and Epstein-Barr virus (EBV) infection. Infections are considered controlled if appropriate therapy has been instituted and, at the time of screening, no signs of infection worsening are present according to Investigator's judgement
• Diagnosed or treated for another malignancy other than the underlying disease allogeneic HCT was indicated for, within 3 years prior to randomization with the exception of complete resection of basal cell carcinoma or squamous cell carcinoma of the skin, an in-situ malignancy, or low risk prostate cancer after curative therapy
• Unable to swallow tablets
• Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures
• Any active, uncontrolled infections assessed to be clinically significant by the Investigator

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Participants will receive matching placebo tablets PO QD per 28-day cycles starting on Day 1 until discontinuation criteria are met or until end of study	Drug: Prednisone; Pharmaceutical form:Tablet-Route of administration:oral; Drug: Prednisolone; Pharmaceutical form:Tablet-Route of administration:oral; Drug: Placebo; Pharmaceutical form:Table-Route of administration:oral
Experimental: Belumosudil; Participants will receive belumosudil 200 mg tablets per os(PO) once daily (QD) per 28-day cycles starting on Day 1 until discontinuation criteria are met or until end of study Note: 200mg two times a day (BID) is used in some cases, when the subject is taking a proton pump inhibitor or a strong CYP3A4 inducer)	Drug: Belumosudil; Pharmaceutical form:Tablet-Route of administration:oral; Other Names: REZUROCK; SAR445761/ KD025; Drug: Prednisone; Pharmaceutical form:Tablet-Route of administration:oral; Drug: Prednisolone; Pharmaceutical form:Tablet-Route of administration:oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Event-Free Survival (EFS)	From the date of randomization to the date of any predefined event, whichever occurs first	Until the end of the study (up to 2.5 years since first patient in).

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Modified Lee Symptom Scale (mLSS)	Proportion of participants who achieve a clinically relevant reduction in mLSS of at least 6 points from baseline (Only in participants at least 18 years of age)	Until the end of the study (up to 2.5 years since first patient in).
Durable overall response rate	Proportion of participants who achieve an overall response (PR or CR) as per 2014 NIH consensus response criteria by 48 weeks and maintained the response for a duration of at least 6 months	Until the end of the study (up to 2.5 years since first patient in).
Rate of corticosteroid withdrawal	Proportion of participants who successfully discontinue all systemic corticosteroids for cGVHD for at least 30 days before the occurrence of cGVHD progression, or start of a new systemic treatment for cGVHD, relapse or recurrence of the underlying disease, or unacceptable toxicity	Until the end of the study (up to 2.5 years since first patient in).
Overall response rate (ORR)	Proportion of participants who achieve an overall response (CR or PR) as per 2014 NIH consensus response criteria at any time before the start of new systemic treatment for cGVHD	Until the end of the study (up to 2.5 years since first patient in).
ORR by 24 weeks	Proportion of participants who achieve an overall response (CR or PR) as per 2014 NIH consensus response criteria by 24 weeks (Cycle 7 Day 1) before the start of new systemic treatment for cGVHD	Until the end of the study (up to 2.5 years since first patient in).
Duration of response (DOR)	Time from the date of the first response to the date of cGVHD progression, start of new systemic treatment for cGVHD, or death, whichever occurs first. DOR is determined only for participants who achieved overall response (PR or CR) as per 2014 NIH consensus response criteria	Until the end of the study (up to 2.5 years since first patient in).
Dose reduction in corticosteroid	Proportion of participants with a reduction in daily corticosteroid dose	Until the end of the study (up to 2.5 years since first patient in).
Failure Free Survival (FFS)	Failure Free Survival (FFS) is defined as the time from the date of randomization to the date of start of a new systemic treatment for cGVHD, relapse or recurrence of the underlying disease, or death, whichever occurs first.	Until the end of the study (up to 2.5 years since first patient in).
Change in patient reported outcome (PRO)	Change from baseline in Patient-Reported Outcomes Measurement Information System Global Health (PROMIS-GH) (Only in participants at least 18 years of age) and the European Quality of Life Group Questionnaire with 5 Dimensions and 5 Levels (EQ5D5L)	Until the end of the study (up to 2.5 years since first patient in).
Number of participants with treatment-emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs)		Until the end of the study (up to 2.5 years since first patient in).
Overall survival	The time from the date of randomization to the date of death due to any cause	Until the end of the study (up to 2.5 years since first patient in).
Time to response (TTR)	Time to Response is defined as the time from randomization to the date the patient has first response (CR or PR).	Until the end of study (up to 2.5 years since first patient in)
Response by organ	Proportion of participants who achieve CR or PR as per NIH consensus response criteria (2014) at any time point in each involved organ and before the start of a new systemic therapy for cGVHD.	Until the end of study (up to 2.5 years since first patient in)

Collaborators and Investigators

• Sponsor: Sanofi

Publications and helpful links

Helpful Links

• EFC17757 Plain Language Results Summary

Study record dates

Study Major Dates

• Study Start (Actual): January 23, 2024
• Primary Completion (Estimated): September 29, 2028
• Study Completion (Estimated): September 29, 2028

Study Registration Dates

• First Submitted: November 16, 2023
• First Submitted That Met QC Criteria: November 16, 2023
• First Posted (Actual): November 22, 2023

Study Record Updates

• Last Update Posted (Estimated): October 14, 2025
• Last Update Submitted That Met QC Criteria: October 13, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Organizing Pneumonia; Immune System Diseases; Respiratory Tract Diseases; Lung Diseases; Bronchial Diseases; Lung Diseases, Obstructive; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Graft vs Host Disease; Bronchiolitis Obliterans Syndrome; Polycyclic Compounds; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Pregnadienetriols; Pregnadienediols; Prednisone; Prednisolone; belumosudil; KD025
• Other Study ID Numbers: EFC17757; 2023-505394-32 (Registry Identifier: CTIS); U1111-1280-4918 (Registry Identifier: ICTRP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No