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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT06143891
Registration number
NCT06143891
Ethics application status
Date submitted
16/11/2023
Date registered
22/11/2023
Date last updated
30/05/2024
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.
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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)
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Secondary ID [1]
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2023-505394-32
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Secondary ID [2]
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EFC17757
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Universal Trial Number (UTN)
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Trial acronym
ROCKnrol-1
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Chronic Graft Versus Host Disease
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Condition category
Condition code
Respiratory
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Other respiratory disorders / diseases
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Inflammatory and Immune System
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Other inflammatory or immune system disorders
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Intervention/exposure
Study type
Interventional
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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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Event-Free Survival (EFS)
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Assessment method [1]
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from the date of randomization to the date of any predefined event, whichever occurs first
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Timepoint [1]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [1]
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modified Lee Symptom Scale (mLSS)
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Assessment method [1]
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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)
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Timepoint [1]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [2]
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overall response rate (ORR)
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Assessment method [2]
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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
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Timepoint [2]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [3]
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rate of corticosteroid withdrawal
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Assessment method [3]
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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
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Timepoint [3]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [4]
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Overall response rate (ORR)
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Assessment method [4]
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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
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Timepoint [4]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [5]
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ORR by 24 weeks
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Assessment method [5]
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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
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Timepoint [5]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [6]
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Duration of response (DOR)
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Assessment method [6]
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Time from the date of the first response to the date of cGVHD progression as defined by 2014 NIH consensus response criteria, 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
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Timepoint [6]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [7]
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Dose reduction in corticosteroid
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Assessment method [7]
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Proportion of participants with a reduction in daily corticosteroid dose
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Timepoint [7]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [8]
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Failure Free Survival (FFS)
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Assessment method [8]
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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.
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Timepoint [8]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [9]
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Change in patient reported outcome (PRO)
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Assessment method [9]
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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)
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Timepoint [9]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [10]
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Number of participants with treatment-emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs)
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Assessment method [10]
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Timepoint [10]
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Until the end of the study (up to 5 years since first patient in).
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Secondary outcome [11]
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Overall survival
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Assessment method [11]
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The time from the date of randomization to the date of death due to any cause
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Timepoint [11]
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Until the end of the study (up to 5 years since first patient in).
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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
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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 <50 x 109/L. Platelet transfusion is not allowed within 3 days before the
screening hematological test
- Absolute neutrophil count (ANC) <1.0 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 if due to cGVHD 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
23/01/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
29/09/2028
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Actual
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Sample size
Target
240
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
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Recruitment hospital [1]
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Investigational Site Number : 0360005 - Westmead
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Recruitment hospital [2]
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Investigational Site Number : 0360003 - Herston
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Recruitment hospital [3]
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Investigational Site Number : 0360001 - Melbourne
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Recruitment hospital [4]
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Investigational Site Number : 0360004 - Murdoch
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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4029 - Herston
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Recruitment postcode(s) [3]
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3000 - Melbourne
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Recruitment postcode(s) [4]
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6961 - Murdoch
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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United States of America
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Massachusetts
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United States of America
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Michigan
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United States of America
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Ohio
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Country [5]
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United States of America
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State/province [5]
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Tennessee
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Country [6]
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United States of America
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State/province [6]
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Texas
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Country [7]
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United States of America
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State/province [7]
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Virginia
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Country [8]
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United States of America
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State/province [8]
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Wisconsin
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Country [9]
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Argentina
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State/province [9]
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Buenos Aires
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Belgium
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Gent
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Belgium
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Leuven
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Belgium
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Roeselare
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Belgium
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Sint-Lambrechts-Woluwe
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Brazil
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São Paulo
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Canada
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Quebec
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Czechia
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Hradec Kralove
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Czechia
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Praha 2
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Denmark
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Aarhus N
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Denmark
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Copenhagen
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Denmark
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Odense C
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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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Münster
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Israel
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Haifa
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Israel
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Jerusalem
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Israel
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Petach 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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Israel
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Tel HaShomer
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Italy
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Lombardia
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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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Genova
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Italy
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Milano
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Italy
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Reggio Calabria
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Italy
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Roma
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Italy
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Torino
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Spain
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Barcelona [Barcelona]
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Spain
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State/province [39]
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Madrid, Comunidad De
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Spain
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State/province [40]
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Málaga
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Spain
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Salamanca
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Spain
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Sevilla
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Spain
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Valencia
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Sweden
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Göteborg
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Sweden
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Lund
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Sweden
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Stockholm
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Turkey
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Istanbul
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United Kingdom
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Leeds
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United Kingdom
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Manchester
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Country [50]
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United Kingdom
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Newcastle upon Tyne
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Sanofi
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT06143891
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
0
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Trial Transparency email recommended (Toll free for US & Canada)
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Address
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Country
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Phone
0
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800-633-1610
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT06143891
Download to PDF