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)
3 other identifiers
interventional
260
23 countries
147
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Jan 2024
Longer than P75 for phase_3
147 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 16, 2023
CompletedFirst Posted
Study publicly available on registry
November 22, 2023
CompletedStudy Start
First participant enrolled
January 23, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 29, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 29, 2028
October 14, 2025
October 1, 2025
4.7 years
November 16, 2023
October 13, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
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 Outcomes (13)
Modified Lee Symptom Scale (mLSS)
Until the end of the study (up to 2.5 years since first patient in).
Durable overall response rate
Until the end of the study (up to 2.5 years since first patient in).
Rate of corticosteroid withdrawal
Until the end of the study (up to 2.5 years since first patient in).
Overall response rate (ORR)
Until the end of the study (up to 2.5 years since first patient in).
ORR by 24 weeks
Until the end of the study (up to 2.5 years since first patient in).
- +8 more secondary outcomes
Study Arms (2)
Belumosudil
EXPERIMENTALParticipants 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)
Placebo
PLACEBO COMPARATORParticipants 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
Interventions
Pharmaceutical form:Tablet-Route of administration:oral
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
- +10 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Sanofilead
Study Sites (151)
University of Arkansas for Medical Sciences-Site Number : 8400019
Little Rock, Arkansas, 72205, United States
City of Hope National Medical Center- Site Number : 8400001
Duarte, California, 91010, United States
University of California San Francisco - Parnassus Heights- Site Number : 8400035
San Francisco, California, 94143, United States
AdventHealth Orlando- Site Number : 8400023
Orlando, Florida, 32803, United States
The Hope Clinic of Emory University- Site Number : 8400020
Decatur, Georgia, 30030, United States
Northwestern University- Site Number : 8400017
Chicago, Illinois, 60611, United States
Indiana University Health University Hospital- Site Number : 8400006
Indianapolis, Indiana, 46202, United States
University of Kentucky Chandler Medical Center- Site Number : 8400024
Lexington, Kentucky, 40536, United States
Johns Hopkins Hospital- Site Number : 8400033
Baltimore, Maryland, 21287, United States
Dana Farber Cancer Institute Site Number : 8400005
Boston, Massachusetts, 02115, United States
Karmanos Cancer Institute - Detroit- Site Number : 8400013
Detroit, Michigan, 48201, United States
UNC Children's Hospital- Site Number : 8400025
Chapel Hill, North Carolina, 27514, United States
Atrium Health Wake Forest Baptist Medical Center - Winston-Salem - Medical Center Boulevard- Site Number : 8400007
Winston-Salem, North Carolina, 27157, United States
Oncology Hematology Care - Kenwood- Site Number : 8400030
Cincinnati, Ohio, 45236, United States
The Ohio State University Comprehensive Cancer Center (OSUCCC) - The James Cancer Hospital and Solove Research Institute- Site Number : 8400026
Columbus, Ohio, 43210, United States
Oregon Health and Science University- Site Number : 8400027
Portland, Oregon, 97239, United States
UPMC Hillman Cancer Center- Site Number : 8400008
Pittsburgh, Pennsylvania, 15232, United States
Sarah Cannon Research Institute Site Number : 8400003
Nashville, Tennessee, 37203, United States
St. David's South Austin Medical Center- Site Number : 8400002
Austin, Texas, 78704, United States
Texas Oncology - Dallas - Worth Street- Site Number : 8400010
Dallas, Texas, 75246, United States
Texas Transplant Institute - Methodist Hospital- Site Number : 8400037
San Antonio, Texas, 78229, United States
University of Virginia Comprehensive Cancer Center-Site Number : 8400031
Charlottesville, Virginia, 22903, United States
Fred Hutchinson Cancer Research Center- Site Number : 8400004
Seattle, Washington, 98109, United States
University of Wisconsin Carbone Cancer Center-Site Number : 8400029
Madison, Wisconsin, 53792, United States
Investigational Site Number : 0320006
Pilar, Buenos Aires, 1629, Argentina
Investigational Site Number : 0320005
Buenos Aires, 1227, Argentina
Investigational Site Number : 0320003
Buenos Aires, 1280, Argentina
Investigational Site Number : 0320001
Buenos Aires, 1426, Argentina
Investigational Site Number : 0320002
Buenos Aires, 1431, Argentina
Investigational Site Number : 0320004
Córdoba, 5000, Argentina
Investigational Site Number : 0360005
Westmead, New South Wales, 2145, Australia
Investigational Site Number : 0360003
Brisbane, Queensland, 4029, Australia
Investigational Site Number : 0360001
Melbourne, Victoria, 3000, Australia
Investigational Site Number : 0360004
Murdoch, Western Australia, 6150, Australia
Investigational Site Number : 0400003
Graz, 8036, Austria
Investigational Site Number : 0400001
Linz, 4020, Austria
Investigational Site Number : 0400004
Vienna, 1090, Austria
Cliniques Universitaires St. Luc-Investigational Site Number : 0560005
Woluwe, Brussels Capital, 1200, Belgium
UZ Leuven-Investigational Site Number : 0560001
Leuven, Vlaams-Brabant, 3000, Belgium
Investigational Site Number : 0560006
Bruges, 8000, Belgium
UZ Gent-Investigational Site Number : 0560003
Ghent, 9000, Belgium
CHU de Liège-Investigational Site Number : 0560004
Liège, 4000, Belgium
AZ Delta-Investigational Site Number : 0560002
Roeselare, 8800, Belgium
Hospital de Clinicas da Universidade Federal do Parana- Site Number : 0760008
Curitiba, Paraná, 80060-900, Brazil
Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760006
São José do Rio Preto, São Paulo, 15090-000, Brazil
INCA - Hospital do Câncer - Rio de Janeiro - Praça Da Cruz Vermelha- Site Number : 0760007
Rio de Janeiro, 20230-130, Brazil
Hospital das Clinicas FMUSP- Site Number : 0760004
São Paulo, 05403-000, Brazil
Hospital Israelita Albert Einstein- Site Number : 0760003
São Paulo, 05651-901, Brazil
Investigational Site Number : 1240007
Calgary, Alberta, T2N 5G2, Canada
Investigational Site Number : 1240005
Toronto, Ontario, M5G 2M9, Canada
Hôpital Maisonneuve Rosemont-Investigational Site Number : 1240001
Montreal, Quebec, H1T 2M4, Canada
CHU Sainte-Justine-Site Number : 1240003
Montreal, Quebec, H3T1C5, Canada
Investigational Site Number : 1240002
Montreal, Quebec, H4A 3J1, Canada
Investigational Site Number : 1240008
Saskatoon, Saskatchewan, S7N 4H4, Canada
Investigational Site Number : 1560019
Beijing, 100050, China
Investigational Site Number : 1560012
Changchun, 130021, China
Investigational Site Number : 1560010
Changsha, 410008, China
Investigational Site Number : 1560011
Fuzhou, 350001, China
Investigational Site Number : 1560005
Guangzhou, 510000, China
Investigational Site Number : 1560002
Hangzhou, 310003, China
Investigational Site Number : 1560014
Hefei, 230001, China
Investigational Site Number : 1560008
Jinan, 250001, China
Investigational Site Number : 1560007
Jinan, 250014, China
Investigational Site Number : 1560001
Suzhou, 215006, China
Investigational Site Number : 1560006
Suzhou, 215025, China
Investigational Site Number : 1560003
Tianjin, 300020, China
Investigational Site Number : 1560004
Tianjin, 300020, China
Investigational Site Number : 1560013
Wuhan, 430022, China
Investigational Site Number : 1560015
Wuhan, 430030, China
Investigational Site Number : 1560009
Zhengzhou, 450008, China
Interni hematologicka a onkologicka klinika Fakultni nemocnice Brno-Site Number : 2030003
Brno, 62500, Czechia
IV. Interni hematologicka klinika, Fakultni nemocnice Hradec Kralove-Site Number : 2030002
Hradec Králové, 50005, Czechia
Klinika hematoonkologie, Fakultni nemocnice Ostrava-Site Number : 2030001
Ostrava, 70852, Czechia
Ustav hematologie a krevni transfuze-Site Number : 2030004
Prague, 128 00, Czechia
Rigshospitalet Copenhagen University Hospital-Investigational Site Number : 2080002
Copenhagen, Capital, 2100, Denmark
Odense University Hospital-Investigational Site Number : 2080003
Odense, Funen, 5000, Denmark
Department of hematology-Investigational Site Number : 2080001
Aarhus, Jutland, 8200, Denmark
Institut Paoli Calmettes-Investigational Site Number : 2500004
Marseille, 13273, France
CHU Hotel Dieu-Investigational Site Number : 2500005
Nantes, 44093, France
Investigational Site Number : 2500003
Paris, 75010, France
Investigational Site Number : 2500001
Paris, 75571, France
CHU Bordeaux-Hôpital Haut-Lévêque-Investigational Site Number : 2500002
Pessac, 33600, France
Hôpital Lyon Sud-Investigational Site Number : 2500007
Pierre-Bénite, 69310, France
Hôpital Pontchaillou - CHU de Rennes-Investigational Site Number : 2500008
Rennes, 35033, France
Investigational Site Number : 2500006
Vandœuvre-lès-Nancy, 54511, France
Investigational Site Number : 2760007
Berlin, 13353, Germany
Investigational Site Number : 2760003
Cologne, 50937, Germany
Investigational Site Number : 2760001
Dresden, 01307, Germany
Investigational Site Number : 2760005
Freiburg im Breisgau, 79106, Germany
Investigational Site Number : 2760002
Hamburg, 20246, Germany
Investigational Site Number : 2760010
Kiel, 24105, Germany
Investigational Site Number : 2760009
Münster, 48149, Germany
Investigational Site Number : 2760006
Regensburg, 93053, Germany
Investigational Site Number : 2760008
Tübingen, 72076, Germany
Nosokomeio Paidon I Agia Sofia-Site Number : 3000002
Athens, 115 27, Greece
General Hospital of Thessaloniki "George Papanikolaou"-Site Number : 3000001
Thessaloniki, 570 10, Greece
Investigational Site Number : 3440001
Pok Fu Lam, 999077, Hong Kong
Investigational Site Number : 3760002
Haifa, 3109601, Israel
Investigational Site Number : 3760005
Jerusalem, 9112001, Israel
Investigational Site Number : 3760006
Petah Tikva, 4920235, Israel
Investigational Site Number : 3760003
Ramat Gan, 5262100, Israel
Investigational Site Number : 3760004
Ramat Gan, 5262100, Israel
Investigational Site Number : 3760001
Tel Aviv, 6423906, Israel
Investigational Site Number : 3800012
Torette, Ancona, 60020, Italy
Investigational Site Number : 3800005
Genoa, Genova, 16132, Italy
Investigational Site Number : 3800009
Milan, Milano, 20132, Italy
Investigational Site Number : 3800002
Rozzano, Milano, 20089, Italy
Investigational Site Number : 3800001
Rome, Roma, 00168, Italy
Investigational Site Number : 3800007
Turin, Torino, 10126, Italy
Investigational Site Number : 3800003
Bergamo, 24127, Italy
Investigational Site Number : 3800004
Bologna, 40138, Italy
Investigational Site Number : 3800011
Pavia, 27100, Italy
Investigational Site Number : 3800010
Perugia, 06156, Italy
Investigational Site Number : 3800006
Reggio Calabria, 89133, Italy
Investigational Site Number : 3800008
Udine, 33100, Italy
Amsterdam Universitair Medisch Centrum-Site Number : 5280005
Amsterdam, 1081 HV, Netherlands
Investigational Site Number : 5280003
Groningen, 9713 GR, Netherlands
Investigational Site Number : 5280002
Rotterdam, 3015 CE, Netherlands
Investigational Site Number : 5280001
Utrecht, 3584 CS, Netherlands
Investigational Site Number : 6160001
Gdansk, Pomeranian Voivodeship, 80-952, Poland
Investigational Site Number : 6160008
Szczecin, 71-252, Poland
Investigational Site Number : 6200003
Lisbon, 1099-023, Portugal
Investigational Site Number : 6200002
Lisbon, 1649-035, Portugal
Investigational Site Number : 4100002
Seoul, Seoul-teukbyeolsi, 03080, South Korea
Investigational Site Number : 4100003
Seoul, Seoul-teukbyeolsi, 03722, South Korea
Investigational Site Number : 4100004
Seoul, Seoul-teukbyeolsi, 06351, South Korea
Investigational Site Number : 4100001
Seoul, Seoul-teukbyeolsi, 06591, South Korea
Investigational Site Number : 7240011
Santander, Cantabria, 39008, Spain
Investigational Site Number : 7240005
Barcelona, Catalunya [Cataluña], 08036, Spain
ICO Institut Català d'Oncologia-Site Number : 7240006
Barcelona, Catalunya [Cataluña], 08908, Spain
Investigational Site Number : 7240004
Madrid, 28007, Spain
Hospital Niño Jesús-Site Number : 7240002
Madrid, 28009, Spain
Hospital Regional Universitarioa de Malaga-Site Number : 7240003
Málaga, 29010, Spain
Hospital Universitario de Salamanca-Site Number : 7240007
Salamanca, 37007, Spain
Hospital Universitario Virgen del Rocío.-Site Number : 7240008
Seville, 41013, Spain
Investigational Site Number : 7240012
Valencia, 46010, Spain
Hospital Universitari i Politècnic La Fe-Site Number : 7240001
Valencia, 46026, Spain
Investigational Site Number : 7520003
Gothenburg, 413 45, Sweden
Investigational Site Number : 7520001
Huddinge, 141 57, Sweden
Investigational Site Number : 7520002
Lund, 221 85, Sweden
Investigational Site Number : 7920006
Adana, 01120, Turkey (Türkiye)
Investigational Site Number : 7920002
Ankara, 06200, Turkey (Türkiye)
Investigational Site Number : 7920004
Ankara, 06560, Turkey (Türkiye)
Investigational Site Number : 7920001
Ankara, 06620, Turkey (Türkiye)
Investigational Site Number : 7920005
Istanbul, 34010, Turkey (Türkiye)
Investigational Site Number : 7920003
Izmir, 35100, Turkey (Türkiye)
Investigational Site Number : 8260006
Cardiff, Cardiff [Caerdydd Gb-crd], CF14 4XW, United Kingdom
Investigational Site Number : 8260002
London, London, City of, SE5 9RL, United Kingdom
Investigational Site Number : 8260003
Leeds, LS9 7TF, United Kingdom
Investigational Site Number : 8260001
Manchester, M20 4BX, United Kingdom
Investigational Site Number : 8260004
Newcastle upon Tyne, NE7 7DN, United Kingdom
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 16, 2023
First Posted
November 22, 2023
Study Start
January 23, 2024
Primary Completion (Estimated)
September 29, 2028
Study Completion (Estimated)
September 29, 2028
Last Updated
October 14, 2025
Record last verified: 2025-10
Data Sharing
- IPD Sharing
- Will share
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