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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05643638
Registration number
NCT05643638
Ethics application status
Date submitted
29/11/2022
Date registered
9/12/2022
Titles & IDs
Public title
A Study of CYP-001 in Combination With Corticosteroids in Adults With High-risk aGvHD
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Scientific title
A Multicenter, Randomized, Double-blind, Placebo-Controlled Phase II Study to Investigate the Efficacy and Safety of CYP-001 in Combination With Corticosteroids vs Corticosteroids Alone for the Treatment of High-Risk Acute Graft Versus Host Disease
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Secondary ID [1]
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CYP-GvHD-P2-01
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Graft Versus Host Disease, Acute
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Condition category
Condition code
Inflammatory and Immune System
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0
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Other inflammatory or immune system disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Other - CYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs)
Treatment: Other - Placebo
Treatment: Drugs - Corticosteroids
Experimental: CYP-001 plus corticosteroids -
Placebo comparator: Placebo plus corticosteroids -
Treatment: Other: CYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs)
Cymerus MSCs are derived from iPSCs using the proprietary Cymerus platform technology.
Treatment: Other: Placebo
The placebo product is identical to CYP-001, except that it contains no active agent
Treatment: Drugs: Corticosteroids
All enrolled subjects in this trial must receive corticosteroids at a minimum dose of oral prednisone 2 mg/kg/day (or methylprednisolone 1.6 mg/kg/day IV) as therapy for aGvHD for at least for 72 hours post enrollment.
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Intervention code [1]
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Treatment: Other
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Intervention code [2]
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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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Overall response rate (ORR)
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Assessment method [1]
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ORR is defined as the proportion of subjects demonstrating a complete response (CR) or partial response (PR) without requirement for additional systemic therapies for an earlier progression, a mixed response or a nonresponse.
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Timepoint [1]
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28 days
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Secondary outcome [1]
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Durable Overall response rate (ORR)
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Assessment method [1]
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Durable ORR is defined as the proportion of subjects demonstrating OR at Day 28 and maintaining OR at Day 60 and Day 100
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Timepoint [1]
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100 days
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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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ORR is defined as the proportion of subjects demonstrating a CR or PR without requirement for additional systemic therapies for an earlier progression, a mixed response or a nonresponse.
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Timepoint [2]
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0
100 days
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Secondary outcome [3]
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Complete response rate (CRR)
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Assessment method [3]
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ORR is defined as the proportion of subjects demonstrating a CR without requirement for additional systemic therapies for an earlier progression, a mixed response or a nonresponse.
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Timepoint [3]
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100 days
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Secondary outcome [4]
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Overall survival
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Assessment method [4]
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The Kaplan Meier curve will be used to estimate the distribution of overall survival and the probability of surviving to relevant timepoints.
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Timepoint [4]
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2 years
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Secondary outcome [5]
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Event-free survival
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Assessment method [5]
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Event-Free survival is defined as the time from the date of randomization to the date of hematologic disease relapse/progression, graft failure, or death due to any cause.
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Timepoint [5]
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2 years
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Secondary outcome [6]
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Time to non-relapse mortality
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Assessment method [6]
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Time to non-relapse mortality is defined as the time from the date of randomization to the date of death not preceded by hematologic disease relapse/progression.
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Timepoint [6]
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2 years
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Secondary outcome [7]
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Failure-free survival
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Assessment method [7]
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Failure-free survival is defined as the time from the date of randomization to date of hematologic disease relapse/progression, non-relapse mortality, or addition of new systemic aGvHD treatment
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Timepoint [7]
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2 years
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Secondary outcome [8]
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Time to malignancy relapse/progression
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Assessment method [8]
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Time to malignancy relapse/progression is defined as the time from the date of randomization to the date to hematologic malignancy relapse/progression.
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Timepoint [8]
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2 years
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Secondary outcome [9]
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Incidence of chronic GvHD
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Assessment method [9]
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Chronic GvHD is defined as the diagnosis of mild, moderate, or severe chronic GvHD.
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Timepoint [9]
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2 years
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Secondary outcome [10]
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Weekly cumulative steroid dose
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Assessment method [10]
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The total corticosteroid dose administered each week
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Timepoint [10]
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100 days
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Secondary outcome [11]
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Patient reported outcomes: Functional Assessment of Cancer Therapy - Bone Marrow Transplantation (FACT-BMT) instrument
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Assessment method [11]
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The FACT-BMT form was designed to measure the quality of life in patients undergoing bone marrow transplantation.
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Timepoint [11]
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2 years
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Secondary outcome [12]
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Patient reported outcomes: EuroQol 5-Dimension (EQ-5D) health-related quality of life instrument
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Assessment method [12]
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EQ-5D is a standardized measure of health-related quality of life
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Timepoint [12]
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2 years
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Secondary outcome [13]
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Incidence, severity, duration of treatment-emergent adverse events
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Assessment method [13]
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Assessment of safety
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Timepoint [13]
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2 years
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Eligibility
Key inclusion criteria
* Undergone allogeneic hematopoietic stem cell transplant (HSCT)
* Clinically diagnosed with acute GvHD requiring systemic therapy with corticosteroids.
* HR-aGvHD must meet one of the following clinical features within 72 hours prior to randomization: (a) high-risk as per Refined Minnesota Criteria; OR (b) One of the following: (i) isolated stage 2 involvement of the lower GI tract; (ii) Stage 1 lower GI tract disease with skin involvement
* Evidence of myeloid engraftment post allogeneic HSCT
* Life expectancy of at least one month
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Minimum age
18
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
* Received any systemic treatment for aGvHD other than corticosteroids +/- calcineurin inhibitors
* Chronic GvHD or overlap syndrome with both acute and chronic features of GvHD
* Relapsed primary malignancy since
* received more than one allogeneic HSCT
* Clinically significant respiratory, renal or cardiac disease
* Cholestatic disorders or sinusoidal obstructive syndrome/veno-occlusive disease of the liver
* Any active uncontrolled infection requiring treatment and likely to impact on the ability of the subject to participate in the trial.
* Known infection with CMV, EBV, HHV-6, HBV, HCV, HIV or Tuberculosis. If the treatment for CMV, EBV, HHV-6, HBV, HCV has commenced the subject is eligible.
* Known sensitivity to dimethylsulfoxide (DMSO) or any other component of CYP-001.
* Received any investigational treatment agent within 30 days or within 5 half-lives of Screening, whichever is greater.
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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 2
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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
4/03/2024
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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
31/12/2026
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Actual
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Sample size
Target
60
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,QLD
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Recruitment hospital [1]
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Royal Prince Alfred Hospital - Sydney
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Recruitment hospital [2]
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Westmead Hospital - Westmead
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Recruitment hospital [3]
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Royal Brisbane and Women's Hospital - Herston
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Recruitment postcode(s) [1]
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2050 - Sydney
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Recruitment postcode(s) [2]
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2145 - Westmead
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Recruitment postcode(s) [3]
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4029 - Herston
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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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Arkansas
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Country [2]
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United States of America
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State/province [2]
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Florida
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Country [3]
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0
United States of America
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State/province [3]
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Georgia
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Country [4]
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0
United States of America
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State/province [4]
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Illinois
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Country [5]
0
0
United States of America
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State/province [5]
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Michigan
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Country [6]
0
0
United States of America
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State/province [6]
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Nebraska
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Country [7]
0
0
United States of America
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State/province [7]
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New York
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Country [8]
0
0
United States of America
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State/province [8]
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Utah
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Country [9]
0
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France
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State/province [9]
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Lille
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Country [10]
0
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Italy
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State/province [10]
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Ancona
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Country [11]
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Italy
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State/province [11]
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Roma
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Country [12]
0
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Italy
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State/province [12]
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San Giovanni Rotondo
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Country [13]
0
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Italy
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State/province [13]
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Venezia
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Country [14]
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Lithuania
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State/province [14]
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Vilnius
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Country [15]
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Spain
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State/province [15]
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Madrid
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Country [16]
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Spain
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State/province [16]
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Murcia
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Country [17]
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Spain
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State/province [17]
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Pamplona
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Country [18]
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Turkey
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State/province [18]
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Eskisehir
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Country [19]
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Turkey
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State/province [19]
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Istanbul
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Country [20]
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Turkey
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State/province [20]
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Izmir
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Country [21]
0
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Turkey
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State/province [21]
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Malatya
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Country [22]
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Turkey
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State/province [22]
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Yenimahalle
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Cynata Therapeutics Limited
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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 study is a prospective randomized placebo-controlled phase 2 study to compare CYP-001 plus corticosteroids (CS) to placebo plus CS in allogeneic hematologic stem cell transplant recipients with HR-aGvHD. Severity of GvHD will be assessed at screening and throughout the study using Mount Sinai Acute GvHD International Consortium (MAGIC) guidelines. Eligible subjects will be randomized to receive either CYP-001 IV infusion on Days 0 and 4 or placebo on the same days. All subjects will receive ongoing CS therapy as appropriate per institutional guidelines. Subjects will have study visits up to Day 100 during the Primary Evaluation Period. During the Follow-Up Period, subjects will have study visits up to 24 months.
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Trial website
https://clinicaltrials.gov/study/NCT05643638
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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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Jolanta Airey, MD
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Address
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Cynata Therapeutics Limited
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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
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Cynata Project Manager
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Address
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Country
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Phone
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+61 3 7067 6940
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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
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
The Sponsor will consider requests to share IPD from this study for further research of a non-commercial nature. Requests should be submitted to
[email protected]
. Sharing of IPD will be subjects to the execution of an IPD sharing agreement, and applicable laws, regulations and guidance in force at that time.
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When will data be available (start and end dates)?
IPD sharing requests will be considered from 12 months after publication of results of this study.
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Available to whom?
Requests for sharing of IPD for non-commercial research purposes will be considered in good faith by the Sponsor. Requests must be accompanied by a detailed research plan, with a justification for the proposed research.
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Available for what types of analyses?
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How or where can data be obtained?
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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/NCT05643638