Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05643638
Registration number
NCT05643638
Ethics application status
Date submitted
29/11/2022
Date registered
9/12/2022
Date last updated
5/04/2024
Titles & IDs
Public title
A Study of CYP-001 in Combination With Corticosteroids in Adults With High-risk aGvHD
Query!
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
Query!
Secondary ID [1]
0
0
CYP-GvHD-P2-01
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Graft Versus Host Disease, Acute
0
0
Query!
Condition category
Condition code
Inflammatory and Immune System
0
0
0
0
Query!
Other inflammatory or immune system disorders
Query!
Other
0
0
0
0
Query!
Research that is not of generic health relevance and not applicable to specific health categories listed above
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Other interventions - CYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs)
Other interventions - Placebo
Treatment: Drugs - Corticosteroids
Experimental: CYP-001 plus corticosteroids -
Placebo Comparator: Placebo plus corticosteroids -
Other interventions: 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.
Other interventions: 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.
Query!
Intervention code [1]
0
0
Other interventions
Query!
Intervention code [2]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Overall response rate (ORR)
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
28 days
Query!
Secondary outcome [1]
0
0
Durable Overall response rate (ORR)
Query!
Assessment method [1]
0
0
Durable ORR is defined as the proportion of subjects demonstrating OR at Day 28 and maintaining OR at Day 60 and Day 100
Query!
Timepoint [1]
0
0
100 days
Query!
Secondary outcome [2]
0
0
Overall response rate (ORR)
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
100 days
Query!
Secondary outcome [3]
0
0
Complete response rate (CRR)
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
100 days
Query!
Secondary outcome [4]
0
0
Overall survival
Query!
Assessment method [4]
0
0
The Kaplan Meier curve will be used to estimate the distribution of overall survival and the probability of surviving to relevant timepoints.
Query!
Timepoint [4]
0
0
2 years
Query!
Secondary outcome [5]
0
0
Event-free survival
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
2 years
Query!
Secondary outcome [6]
0
0
Time to non-relapse mortality
Query!
Assessment method [6]
0
0
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.
Query!
Timepoint [6]
0
0
2 years
Query!
Secondary outcome [7]
0
0
Failure-free survival
Query!
Assessment method [7]
0
0
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
Query!
Timepoint [7]
0
0
2 years
Query!
Secondary outcome [8]
0
0
Time to malignancy relapse/progression
Query!
Assessment method [8]
0
0
Time to malignancy relapse/progression is defined as the time from the date of randomization to the date to hematologic malignancy relapse/progression.
Query!
Timepoint [8]
0
0
2 years
Query!
Secondary outcome [9]
0
0
Incidence of chronic GvHD
Query!
Assessment method [9]
0
0
Chronic GvHD is defined as the diagnosis of mild, moderate, or severe chronic GvHD.
Query!
Timepoint [9]
0
0
2 years
Query!
Secondary outcome [10]
0
0
Weekly cumulative steroid dose
Query!
Assessment method [10]
0
0
The total corticosteroid dose administered each week
Query!
Timepoint [10]
0
0
100 days
Query!
Secondary outcome [11]
0
0
Patient reported outcomes: Functional Assessment of Cancer Therapy - Bone Marrow Transplantation (FACT-BMT) instrument
Query!
Assessment method [11]
0
0
The FACT-BMT form was designed to measure the quality of life in patients undergoing bone marrow transplantation.
Query!
Timepoint [11]
0
0
2 years
Query!
Secondary outcome [12]
0
0
Patient reported outcomes: EuroQol 5-Dimension (EQ-5D) health-related quality of life instrument
Query!
Assessment method [12]
0
0
EQ-5D is a standardized measure of health-related quality of life
Query!
Timepoint [12]
0
0
2 years
Query!
Secondary outcome [13]
0
0
Incidence, severity, duration of treatment-emergent adverse events
Query!
Assessment method [13]
0
0
Assessment of safety
Query!
Timepoint [13]
0
0
2 years
Query!
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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
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
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
4/03/2024
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
31/12/2026
Query!
Actual
Query!
Sample size
Target
60
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
NSW,QLD
Query!
Recruitment hospital [1]
0
0
Royal Prince Alfred Hospital - Sydney
Query!
Recruitment hospital [2]
0
0
Westmead Hospital - Westmead
Query!
Recruitment hospital [3]
0
0
Royal Brisbane and Women's Hospital - Herston
Query!
Recruitment postcode(s) [1]
0
0
2050 - Sydney
Query!
Recruitment postcode(s) [2]
0
0
2145 - Westmead
Query!
Recruitment postcode(s) [3]
0
0
4029 - Herston
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Arkansas
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Florida
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Illinois
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Nebraska
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
New York
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Utah
Query!
Country [7]
0
0
Turkey
Query!
State/province [7]
0
0
Eskisehir
Query!
Country [8]
0
0
Turkey
Query!
State/province [8]
0
0
Istanbul
Query!
Country [9]
0
0
Turkey
Query!
State/province [9]
0
0
Izmir
Query!
Country [10]
0
0
Turkey
Query!
State/province [10]
0
0
Malatya
Query!
Country [11]
0
0
Turkey
Query!
State/province [11]
0
0
Yenimahalle
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Cynata Therapeutics Limited
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT05643638
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Jolanta Airey, MD
Query!
Address
0
0
Cynata Therapeutics Limited
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Cynata Project Manager
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
+61 3 7067 6940
Query!
Fax
0
0
Query!
Email
0
0
[email protected]
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05643638
Download to PDF