A Study of CYP-001 in Combination With Corticosteroids in Adults With High-risk aGvHD

May 22, 2026 updated by: Cynata Therapeutics Limited

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
60

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2050
        • Royal Prince Alfred Hospital
      • Westmead, New South Wales, Australia, 2145
        • Westmead Hospital
    • Queensland
      • Herston, Queensland, Australia, 4029
        • Royal Brisbane and Women's Hospital
  • France
      • Lille, France
        • Hospital Claude Huriez
      • Paris, France
        • Hôpital Necker Enfants malades
      • Paris, France
        • Hôpital Universitaire Pitié-Salpêtrière
  • Italy
      • Ancona, Italy
        • Azienda Ospedaliero Universitaria Delle Marche
      • Milan, Italy
        • ASST Grande Ospedale Metropolitano Niguarda
      • Roma, Italy
        • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
      • Rozzano, Italy
        • Istituto Clinico Humanitas
      • San Giovanni Rotondo, Italy
        • IRCCS Ospedale Casa Sollievo della Sofferenza
      • Venezia, Italy
        • Ospedale dell'Angelo di Mestre
  • Lithuania
      • Vilnius, Lithuania
        • Vilnius University Hospital Santaros Klinikos
  • Spain
      • Barcelona, Spain
        • ICO L'Hospitalet - Hospital Duran i Reynals
      • Madrid, Spain
        • Hospital Universitario Ramon y Cajal
      • Madrid, Spain
        • Hospital Universitario Fundación Jiménez Díaz
      • Madrid, Spain
        • Hospital Universitato De La Princesa
      • Murcia, Spain
        • Hospital Universitario Virgen de la Arrixaca
      • Pamplona, Spain
        • Clinica Universidad de Navarra
  • Turkey (Türkiye)
      • Eskişehir, Turkey (Türkiye)
        • Anadolu Medical Center
      • Istanbul, Turkey (Türkiye)
        • Koc University
      • Istanbul, Turkey (Türkiye)
        • Memorial Bahçelievler Hospital
      • Istanbul, Turkey (Türkiye), 34318
        • Gayrettepe Florence Nightingale Hastanesi
      • Izmir, Turkey (Türkiye)
        • Izmir Medicalpark Hospital
      • Malatya, Turkey (Türkiye)
        • Inonu University
      • Yenimahalle, Turkey (Türkiye)
        • Dr Abdurrahman Yurtaslan Ankara Onkoloji Egitim ve Arastirma Hastanesi
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85012
        • Banner MD Anderson
      • Phoenix, Arizona, United States, 85288
        • Mayo Clinic Hospital
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • University of Arkansas Medical Center
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic Hospital
      • Pembroke Pines, Florida, United States, 33026
        • Memorial Healthcare System
    • Georgia
      • Atlanta, Georgia, United States, 30342
        • BMT Group of Georgia
    • Illinois
      • Evanston, Illinois, United States, 60208
        • Northwestern University
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Karmanos Cancer Institute
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • University Of Nebrasaka Medical Center
    • New York
      • New York, New York, United States, 10065
        • Weill Cornell Medicine - New York Presbyterian Hospital
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Health
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • University of Utah

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: CYP-001 plus corticosteroids
Drug: 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.
Biological: 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.
Placebo Comparator: Placebo plus corticosteroids
Drug: 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.
Biological: Placebo
The placebo product is identical to CYP-001, except that it contains no active agent

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Overall response rate (ORR)
Time Frame: 28 days
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.
28 days

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Durable Overall response rate (ORR)
Time Frame: 100 days
Durable ORR is defined as the proportion of subjects demonstrating OR at Day 28 and maintaining OR at Day 60 and Day 100
100 days
Overall response rate (ORR)
Time Frame: 100 days
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.
100 days
Complete response rate (CRR)
Time Frame: 100 days
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.
100 days
Overall survival
Time Frame: 2 years
The Kaplan Meier curve will be used to estimate the distribution of overall survival and the probability of surviving to relevant timepoints.
2 years
Event-free survival
Time Frame: 2 years
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.
2 years
Time to non-relapse mortality
Time Frame: 2 years
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.
2 years
Failure-free survival
Time Frame: 2 years
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
2 years
Time to malignancy relapse/progression
Time Frame: 2 years
Time to malignancy relapse/progression is defined as the time from the date of randomization to the date to hematologic malignancy relapse/progression.
2 years
Incidence of chronic GvHD
Time Frame: 2 years
Chronic GvHD is defined as the diagnosis of mild, moderate, or severe chronic GvHD.
2 years
Weekly cumulative steroid dose
Time Frame: 100 days
The total corticosteroid dose administered each week
100 days
Patient reported outcomes: Functional Assessment of Cancer Therapy - Bone Marrow Transplantation (FACT-BMT) instrument
Time Frame: 2 years
The FACT-BMT form was designed to measure the quality of life in patients undergoing bone marrow transplantation.
2 years
Patient reported outcomes: EuroQol 5-Dimension (EQ-5D) health-related quality of life instrument
Time Frame: 2 years
EQ-5D is a standardized measure of health-related quality of life
2 years
Incidence, severity, duration of treatment-emergent adverse events
Time Frame: 2 years
Assessment of safety
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Cynata Therapeutics Limited

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Jolanta Airey, MD, Cynata Therapeutics Limited

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 4, 2024

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 17, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 29, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 6, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 9, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 26, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 22, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CYP-GvHD-P2-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

The Sponsor will consider requests to share IPD from this study for further research of a non-commercial nature. Requests should be submitted to clinical@cynata.com. 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.

IPD Sharing Time Frame

IPD sharing requests will be considered from 12 months after publication of results of this study.

IPD Sharing Access Criteria

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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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