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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
Date last updated
20/08/2024

Titles & IDs
Public title
A Study of CYP-001 in Combination With Corticosteroids in Adults With High-risk aGvHD
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
Secondary ID [1] 0 0
CYP-GvHD-P2-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Graft Versus Host Disease, Acute 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Overall response rate (ORR)
Timepoint [1] 0 0
28 days
Secondary outcome [1] 0 0
Durable Overall response rate (ORR)
Timepoint [1] 0 0
100 days
Secondary outcome [2] 0 0
Overall response rate (ORR)
Timepoint [2] 0 0
100 days
Secondary outcome [3] 0 0
Complete response rate (CRR)
Timepoint [3] 0 0
100 days
Secondary outcome [4] 0 0
Overall survival
Timepoint [4] 0 0
2 years
Secondary outcome [5] 0 0
Event-free survival
Timepoint [5] 0 0
2 years
Secondary outcome [6] 0 0
Time to non-relapse mortality
Timepoint [6] 0 0
2 years
Secondary outcome [7] 0 0
Failure-free survival
Timepoint [7] 0 0
2 years
Secondary outcome [8] 0 0
Time to malignancy relapse/progression
Timepoint [8] 0 0
2 years
Secondary outcome [9] 0 0
Incidence of chronic GvHD
Timepoint [9] 0 0
2 years
Secondary outcome [10] 0 0
Weekly cumulative steroid dose
Timepoint [10] 0 0
100 days
Secondary outcome [11] 0 0
Patient reported outcomes: Functional Assessment of Cancer Therapy - Bone Marrow Transplantation (FACT-BMT) instrument
Timepoint [11] 0 0
2 years
Secondary outcome [12] 0 0
Patient reported outcomes: EuroQol 5-Dimension (EQ-5D) health-related quality of life instrument
Timepoint [12] 0 0
2 years
Secondary outcome [13] 0 0
Incidence, severity, duration of treatment-emergent adverse events
Timepoint [13] 0 0
2 years

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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

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 2
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
4/03/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2026
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Sydney
Recruitment hospital [2] 0 0
Westmead Hospital - Westmead
Recruitment hospital [3] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment postcode(s) [1] 0 0
2050 - Sydney
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4029 - Herston
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
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
Nebraska
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Utah
Country [9] 0 0
France
State/province [9] 0 0
Lille
Country [10] 0 0
Italy
State/province [10] 0 0
Ancona
Country [11] 0 0
Italy
State/province [11] 0 0
Roma
Country [12] 0 0
Italy
State/province [12] 0 0
San Giovanni Rotondo
Country [13] 0 0
Italy
State/province [13] 0 0
Venezia
Country [14] 0 0
Lithuania
State/province [14] 0 0
Vilnius
Country [15] 0 0
Spain
State/province [15] 0 0
Madrid
Country [16] 0 0
Spain
State/province [16] 0 0
Murcia
Country [17] 0 0
Spain
State/province [17] 0 0
Pamplona
Country [18] 0 0
Turkey
State/province [18] 0 0
Eskisehir
Country [19] 0 0
Turkey
State/province [19] 0 0
Istanbul
Country [20] 0 0
Turkey
State/province [20] 0 0
Izmir
Country [21] 0 0
Turkey
State/province [21] 0 0
Malatya
Country [22] 0 0
Turkey
State/province [22] 0 0
Yenimahalle

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Cynata Therapeutics Limited
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT05643638
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Jolanta Airey, MD
Address 0 0
Cynata Therapeutics Limited
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Cynata Project Manager
Address 0 0
Country 0 0
Phone 0 0
+61 3 7067 6940
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05643638