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Graft Versus Host Disease, AcutePhase 2NCT05643638

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

A Phase 2 study testing CYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs) and Corticosteroids in people with Graft Versus Host Disease, Acute, run by Cynata Therapeutics Limited at 39 sites in 7 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Graft Versus Host Disease, Acute
What is being tested?
CYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs) and Corticosteroids
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
65 participants
Where
39 sitesin 7 countries
Study dates
4 Mar 2024to 17 Jun 2026
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 10Show fewer
  • 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.

Trial locations and contacts

39 sites in 7 countries.

39 sites in 7 countries

United States13 sites

  • BMT Group of Georgia

    Atlanta, Georgia, 30342

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • Karmanos Cancer Institute

    Detroit, Michigan, 48201

  • Northwestern University

    Evanston, Illinois, 60208

  • Penn State Health

    Hershey, Pennsylvania, 17033

  • Mayo Clinic Hospital

    Jacksonville, Florida, 32224

  • University of Arkansas Medical Center

    Little Rock, Arkansas, 72205

  • Weill Cornell Medicine - New York Presbyterian Hospital

    New York, New York, 10065

  • University Of Nebrasaka Medical Center

    Omaha, Nebraska, 68198

  • Memorial healthcare System

    Pembroke Pines, Florida, 33026

  • Banner MD Anderson

    Phoenix, Arizona, 85012

  • Mayo Clinic Hospital

    Phoenix, Arizona, 85288

  • University of Utah

    Salt Lake City, Utah, 84112

Other trials in United States

Turkey (Türkiye)7 sites

  • Anadolu Medical Center

    Eskişehir

  • Gayrettepe Florence Nightingale Hastanesi

    Istanbul, 34318

  • Koc University

    Istanbul

  • Memorial Bahcelievler Hospital

    Istanbul

  • Izmir Medicalpark Hospital

    Izmir

  • İnonu University

    Malatya

  • Dr Abdurrahman Yurtaslan Ankara Onkoloji Egitim ve Arastirma Hastanesi

    Yenimahalle

Other trials in Turkey (Türkiye)

Italy6 sites

  • Azienda Ospedaliero Universitaria delle Marche

    Ancona

  • ASST Grande Ospedale Metropolitano Niguarda

    Milan

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma

  • Istituto Clinico Humanitas

    Rozzano

  • IRCCS Ospedale Casa Sollievo della Sofferenza

    San Giovanni Rotondo

  • Ospedale dell'Angelo di Mestre

    Venezia

Other trials in Italy

Spain6 sites

  • ICO l'Hospitalet - Hospital Duran i Reynals

    Barcelona

  • Hospital Universitario Fundacion Jimenez Diaz

    Madrid

  • Hospital Universitario Ramon y Cajal

    Madrid

  • Hospital Universitato De La Princesa

    Madrid

  • Hospital Universitario Virgen de la Arrixaca

    Murcia

  • Clínica Universidad de Navarra

    Pamplona

Other trials in Spain

Australia3 sites

  • Royal Brisbane and Women's Hospital

    Herston, Queensland, 4029

  • Royal Prince Alfred Hospital

    Sydney, New South Wales, 2050

  • Westmead Hospital

    Westmead, New South Wales, 2145

Other trials in Australia

France3 sites

  • Hospital Claude Huriez

    Lille

  • Hôpital Necker Enfants Malades

    Paris

  • Hôpital Universitaire Pitié-Salpêtrière

    Paris

Other trials in France

Lithuania1 site

  • Vilnius University Hospital Santaros Klinikos

    Vilnius

Other trials in Lithuania

About this study

Official 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

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.

Being tested
CYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs), Placebo, Corticosteroids
Medical subject headings
Graft vs Host Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs)Biological

    Cymerus MSCs are derived from iPSCs using the proprietary Cymerus platform technology.

  • PlaceboBiological

    The placebo product is identical to CYP-001, except that it contains no active agent

  • CorticosteroidsDrug

    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.

Study groups in NCT05643638
Study groupRoleWhat they receive
CYP-001 plus corticosteroidsExperimentalNot described
Receives: Biological: CYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs); Drug: Corticosteroids
Placebo plus corticosteroidsPlacebo comparatorNot described
Receives: Biological: Placebo; Drug: Corticosteroids

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall response rate (ORR)

    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.

    Measured: 28 days

Secondary outcomes (13)

  1. Durable Overall response rate (ORR)

    Durable ORR is defined as the proportion of subjects demonstrating OR at Day 28 and maintaining OR at Day 60 and Day 100

    Measured: 100 days

  2. Overall response rate (ORR)

    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.

    Measured: 100 days

  3. Complete response rate (CRR)

    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.

    Measured: 100 days

  4. Overall survival

    The Kaplan Meier curve will be used to estimate the distribution of overall survival and the probability of surviving to relevant timepoints.

    Measured: 2 years

  5. Event-free survival

    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.

    Measured: 2 years

  6. Time to non-relapse mortality

    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.

    Measured: 2 years

  7. Failure-free survival

    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

    Measured: 2 years

  8. Time to malignancy relapse/progression

    Time to malignancy relapse/progression is defined as the time from the date of randomization to the date to hematologic malignancy relapse/progression.

    Measured: 2 years

  9. Incidence of chronic GvHD

    Chronic GvHD is defined as the diagnosis of mild, moderate, or severe chronic GvHD.

    Measured: 2 years

  10. Weekly cumulative steroid dose

    The total corticosteroid dose administered each week

    Measured: 100 days

  11. Patient reported outcomes: Functional Assessment of Cancer Therapy - Bone Marrow Transplantation (FACT-BMT) instrument

    The FACT-BMT form was designed to measure the quality of life in patients undergoing bone marrow transplantation.

    Measured: 2 years

  12. Patient reported outcomes: EuroQol 5-Dimension (EQ-5D) health-related quality of life instrument

    EQ-5D is a standardized measure of health-related quality of life

    Measured: 2 years

  13. Incidence, severity, duration of treatment-emergent adverse events

    Assessment of safety

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Dec 2022
  2. Study start4 Mar 2024
  3. Primary completion17 Mar 2026
  4. Study completion17 Jun 2026

Why it stopped: Cynata Therapeutics has taken the decision to discontinue the study following the Primary Evaluation period analysis. The results did not demonstrate the anticipated clinical benefit of CYP-001 in combination with corticosteroids in HR-aGVHD.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
65 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05643638
Sponsor's protocol ID
CYP-GvHD-P2-01
Status last verified
Jul 2026
First submitted
29 Nov 2022
Last updated
22 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing CYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs) and Corticosteroids in people with Graft Versus Host Disease, Acute, run by Cynata Therapeutics Limited at 39 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 22 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 39 sites in United States, Turkey (Türkiye), Italy, Spain, Australia and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Cynata Therapeutics has taken the decision to discontinue the study following the Primary Evaluation period analysis. The results did not demonstrate the anticipated clinical benefit of CYP-001 in combination with corticosteroids in HR-aGVHD.). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall response rate (ORR) (time frame: 28 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05643638.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2026.

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