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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06143891




Registration number
NCT06143891
Ethics application status
Date submitted
16/11/2023
Date registered
22/11/2023

Titles & IDs
Public title
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.
Scientific title
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)
Secondary ID [1] 0 0
2023-505394-32
Secondary ID [2] 0 0
EFC17757
Universal Trial Number (UTN)
Trial acronym
ROCKnrol-1
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Graft Versus Host Disease 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Belumosudil
Treatment: Drugs - Placebo
Treatment: Drugs - Prednisone
Treatment: Drugs - Prednisolone

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)

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


Treatment: Drugs: Belumosudil
Pharmaceutical form:Tablet-Route of administration:oral

Treatment: Drugs: Placebo
Pharmaceutical form:Table-Route of administration:oral

Treatment: Drugs: Prednisone
Pharmaceutical form:Tablet-Route of administration:oral

Treatment: Drugs: Prednisolone
Pharmaceutical form:Tablet-Route of administration:oral
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Event-Free Survival (EFS)
Timepoint [1] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [1] 0 0
Modified Lee Symptom Scale (mLSS)
Timepoint [1] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [2] 0 0
Durable overall response rate
Timepoint [2] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [3] 0 0
Rate of corticosteroid withdrawal
Timepoint [3] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [4] 0 0
Overall response rate (ORR)
Timepoint [4] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [5] 0 0
ORR by 24 weeks
Timepoint [5] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [6] 0 0
Duration of response (DOR)
Timepoint [6] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [7] 0 0
Dose reduction in corticosteroid
Timepoint [7] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [8] 0 0
Failure Free Survival (FFS)
Timepoint [8] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [9] 0 0
Change in patient reported outcome (PRO)
Timepoint [9] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [10] 0 0
Number of participants with treatment-emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs)
Timepoint [10] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [11] 0 0
Overall survival
Timepoint [11] 0 0
Until the end of the study (up to 5 years since first patient in).
Secondary outcome [12] 0 0
Time to response (TTR)
Timepoint [12] 0 0
Until the end of study (up to 5 years since first patient in)
Secondary outcome [13] 0 0
Response by organ
Timepoint [13] 0 0
Until the end of study (up to 5 years since first patient in)

Eligibility
Key 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
* Body weight = 40kg
* 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
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Participants are excluded from the study if any of the following criteria apply:

Medical conditions

* Histological relapse of the underlying disease after 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 design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
23/01/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
29/09/2028
Actual
Sample size
Target
260
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Investigational Site Number : 0360005 - Westmead
Recruitment hospital [2] 0 0
Investigational Site Number : 0360003 - Brisbane
Recruitment hospital [3] 0 0
Investigational Site Number : 0360001 - Melbourne
Recruitment hospital [4] 0 0
Investigational Site Number : 0360004 - Murdoch
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4029 - Brisbane
Recruitment postcode(s) [3] 0 0
3000 - Melbourne
Recruitment postcode(s) [4] 0 0
6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Kentucky
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Michigan
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
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Ohio
Country [11] 0 0
United States of America
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Oregon
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United States of America
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Pennsylvania
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Tennessee
Country [14] 0 0
United States of America
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Texas
Country [15] 0 0
United States of America
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Virginia
Country [16] 0 0
United States of America
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Washington
Country [17] 0 0
United States of America
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Wisconsin
Country [18] 0 0
Argentina
State/province [18] 0 0
Ciudad De Buenos Aires
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Argentina
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Buenos Aires
Country [20] 0 0
Austria
State/province [20] 0 0
Linz
Country [21] 0 0
Belgium
State/province [21] 0 0
Brussels
Country [22] 0 0
Belgium
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Vlaams-Brabant
Country [23] 0 0
Belgium
State/province [23] 0 0
Gent
Country [24] 0 0
Belgium
State/province [24] 0 0
Liège
Country [25] 0 0
Belgium
State/province [25] 0 0
Roeselare
Country [26] 0 0
Brazil
State/province [26] 0 0
Paraná
Country [27] 0 0
Brazil
State/province [27] 0 0
São Paulo
Country [28] 0 0
Brazil
State/province [28] 0 0
Rio de Janeiro
Country [29] 0 0
Canada
State/province [29] 0 0
Alberta
Country [30] 0 0
Canada
State/province [30] 0 0
Ontario
Country [31] 0 0
Canada
State/province [31] 0 0
Quebec
Country [32] 0 0
Canada
State/province [32] 0 0
Saskatchewan
Country [33] 0 0
China
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Changchun
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China
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Changsha
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China
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Fuzhou
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China
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Guangzhou
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China
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Hangzhou
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China
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Hefei
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China
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Jinan
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China
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Suzhou
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China
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Tianjin
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China
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Wuhan
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China
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Zhengzhou
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Czechia
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Brno
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Czechia
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Hradec Kralove
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Czechia
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Ostrava
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Czechia
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Praha
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Denmark
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Capital
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Denmark
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Funen
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Denmark
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Jutland
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France
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Marseille
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France
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Nantes
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France
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Paris
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France
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Pessac
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France
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Pierre Benite
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France
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Rennes
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France
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Vandœuvre-lès-nancy
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Germany
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Berlin
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Germany
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Cologne
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Germany
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Dresden
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Germany
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Hamburg
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Germany
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Kiel
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Germany
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Münster
Country [64] 0 0
Germany
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Tübingen
Country [65] 0 0
Greece
State/province [65] 0 0
Athens
Country [66] 0 0
Greece
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Thessaloniki
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Hong Kong
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Pok Fu Lam
Country [68] 0 0
Israel
State/province [68] 0 0
Haifa
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Israel
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Jerusalem
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Israel
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Petah Tikva
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Israel
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Ramat Gan
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Israel
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Tel Aviv
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Italy
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Genova
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Italy
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Lombardia
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Italy
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Milano
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Italy
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Roma
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Italy
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Torino
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Italy
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Bergamo
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Italy
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Bologna
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Italy
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Perugia
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Italy
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Reggio Calabria
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Italy
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Udine
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Netherlands
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Amsterdam
Country [84] 0 0
Netherlands
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Groningen
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Netherlands
State/province [85] 0 0
Rotterdam
Country [86] 0 0
Poland
State/province [86] 0 0
Pomorskie
Country [87] 0 0
Portugal
State/province [87] 0 0
Lisbon
Country [88] 0 0
Spain
State/province [88] 0 0
Andalucia
Country [89] 0 0
Spain
State/province [89] 0 0
Barcelona [Barcelona]
Country [90] 0 0
Spain
State/province [90] 0 0
Castilla Y Leon
Country [91] 0 0
Spain
State/province [91] 0 0
Cataluña
Country [92] 0 0
Spain
State/province [92] 0 0
Comunidad Valenciana
Country [93] 0 0
Spain
State/province [93] 0 0
Madrid, Comunidad De
Country [94] 0 0
Spain
State/province [94] 0 0
Madrid
Country [95] 0 0
Sweden
State/province [95] 0 0
Gothenburg
Country [96] 0 0
Sweden
State/province [96] 0 0
Huddinge
Country [97] 0 0
Sweden
State/province [97] 0 0
Lund
Country [98] 0 0
Turkey
State/province [98] 0 0
Adana
Country [99] 0 0
Turkey
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Ankara
Country [100] 0 0
Turkey
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Istanbul
Country [101] 0 0
Turkey
State/province [101] 0 0
Izmir
Country [102] 0 0
United Kingdom
State/province [102] 0 0
Vale Of Glamorgan, The
Country [103] 0 0
United Kingdom
State/province [103] 0 0
Leeds
Country [104] 0 0
United Kingdom
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Manchester
Country [105] 0 0
United Kingdom
State/province [105] 0 0
Newcastle Upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sanofi
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Transparency email recommended (Toll free for US & Canada)
Address 0 0
Country 0 0
Phone 0 0
800-633-1610
Fax 0 0
Email 0 0
contact-us@sanofi.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06143891