NCT05643638

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 Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
60

participants targeted

Target at P50-P75 for phase_2

Timeline
20mo left

Started Mar 2024

Typical duration for phase_2

Geographic Reach
7 countries

39 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress58%
Mar 2024Dec 2027

First Submitted

Initial submission to the registry

November 29, 2022

Completed
10 days until next milestone

First Posted

Study publicly available on registry

December 9, 2022

Completed
1.2 years until next milestone

Study Start

First participant enrolled

March 4, 2024

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2026

Completed
1.8 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2027

Expected
Last Updated

December 5, 2025

Status Verified

December 1, 2025

Enrollment Period

2 years

First QC Date

November 29, 2022

Last Update Submit

December 1, 2025

Conditions

Graft Versus Host Disease, Acute

Outcome Measures

Primary Outcomes (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.

    28 days

Secondary Outcomes (13)

  • Durable Overall response rate (ORR)

    100 days

  • Overall response rate (ORR)

    100 days

  • Complete response rate (CRR)

    100 days

  • Overall survival

    2 years

  • Event-free survival

    2 years

  • +8 more secondary outcomes

Study Arms (2)

CYP-001 plus corticosteroids

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

Placebo plus corticosteroids

PLACEBO COMPARATOR
Biological: PlaceboDrug: Corticosteroids

Interventions

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.

CYP-001 plus corticosteroids
PlaceboBIOLOGICAL

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

Placebo plus corticosteroids
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.

CYP-001 plus corticosteroidsPlacebo plus corticosteroids

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (39)

Banner MD Anderson

Phoenix, Arizona, 85012, United States

COMPLETED

Mayo Clinic Hospital

Phoenix, Arizona, 85288, United States

RECRUITING

University of Arkansas Medical Center

Little Rock, Arkansas, 72205, United States

RECRUITING

Mayo Clinic Hospital

Jacksonville, Florida, 32224, United States

RECRUITING

Memorial healthcare System

Pembroke Pines, Florida, 33026, United States

RECRUITING

BMT Group of Georgia

Atlanta, Georgia, 30342, United States

RECRUITING

Northwestern University

Evanston, Illinois, 60208, United States

RECRUITING

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

RECRUITING

University Of Nebrasaka Medical Center

Omaha, Nebraska, 68198, United States

RECRUITING

Weill Cornell Medicine - New York Presbyterian Hospital

New York, New York, 10065, United States

COMPLETED

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

Penn State Health

Hershey, Pennsylvania, 17033, United States

RECRUITING

University of Utah

Salt Lake City, Utah, 84112, United States

RECRUITING

Royal Prince Alfred Hospital

Sydney, New South Wales, 2050, Australia

COMPLETED

Westmead Hospital

Westmead, New South Wales, 2145, Australia

COMPLETED

Royal Brisbane and Women's Hospital

Herston, Queensland, 4029, Australia

ACTIVE NOT RECRUITING

Hospital Claude Huriez

Lille, France

RECRUITING

Hôpital Necker Enfants Malades

Paris, France

RECRUITING

Hôpital Universitaire Pitié-Salpêtrière

Paris, France

RECRUITING

Azienda Ospedaliero Universitaria delle Marche

Ancona, Italy

RECRUITING

ASST Grande Ospedale Metropolitano Niguarda

Milan, Italy

RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Italy

RECRUITING

Istituto Clinico Humanitas

Rozzano, Italy

RECRUITING

IRCCS Ospedale Casa Sollievo della Sofferenza

San Giovanni Rotondo, Italy

RECRUITING

Ospedale dell'Angelo di Mestre

Venezia, Italy

RECRUITING

Vilnius University Hospital Santaros Klinikos

Vilnius, Lithuania

RECRUITING

ICO l'Hospitalet - Hospital Duran i Reynals

Barcelona, Spain

RECRUITING

Hospital Universitario Fundacion Jimenez Diaz

Madrid, Spain

RECRUITING

Hospital Universitario Ramon y Cajal

Madrid, Spain

RECRUITING

Hospital Universitato De La Princesa

Madrid, Spain

COMPLETED

Hospital Universitario Virgen de la Arrixaca

Murcia, Spain

RECRUITING

Clínica Universidad de Navarra

Pamplona, Spain

RECRUITING

Anadolu Medical Center

Eskişehir, Turkey (Türkiye)

RECRUITING

Gayrettepe Florence Nightingale Hastanesi

Istanbul, 34318, Turkey (Türkiye)

COMPLETED

Koc University

Istanbul, Turkey (Türkiye)

RECRUITING

Memorial Bahcelievler Hospital

Istanbul, Turkey (Türkiye)

COMPLETED

Izmir Medicalpark Hospital

Izmir, Turkey (Türkiye)

RECRUITING

İnonu University

Malatya, Turkey (Türkiye)

RECRUITING

Dr Abdurrahman Yurtaslan Ankara Onkoloji Egitim ve Arastirma Hastanesi

Yenimahalle, Turkey (Türkiye)

RECRUITING

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

Adrenal Cortex Hormones

Condition Hierarchy (Ancestors)

Immune System Diseases

Intervention Hierarchy (Ancestors)

HormonesHormones, Hormone Substitutes, and Hormone Antagonists

Study Officials

  • Jolanta Airey, MD

    Cynata Therapeutics Limited

    STUDY DIRECTOR

Central Study Contacts

Cynata Project Manager

CONTACT

+61 3 7067 6940clinical@cynata.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 29, 2022

First Posted

December 9, 2022

Study Start

March 4, 2024

Primary Completion

March 1, 2026

Study Completion (Estimated)

December 1, 2027

Last Updated

December 5, 2025

Record last verified: 2025-12

Data Sharing

IPD Sharing
Will share

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.

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

Locations

IT(6)FR(3)US(13)AU(3)LI(1)ES(6)TU(7)