RecruitingPhase 2NCT05643638

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

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


Sponsor

Cynata Therapeutics Limited

Enrollment

60 participants

Start Date

Mar 4, 2024

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 18 Years

Inclusion Criteria5

  • 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 Criteria10

  • 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.

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Interventions

BIOLOGICALCYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs)

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

BIOLOGICALPlacebo

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

DRUGCorticosteroids

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.


Locations(39)

Banner MD Anderson

Phoenix, Arizona, United States

Mayo Clinic Hospital

Phoenix, Arizona, United States

University of Arkansas Medical Center

Little Rock, Arkansas, United States

Mayo Clinic Hospital

Jacksonville, Florida, United States

Memorial healthcare System

Pembroke Pines, Florida, United States

BMT Group of Georgia

Atlanta, Georgia, United States

Northwestern University

Evanston, Illinois, United States

Karmanos Cancer Institute

Detroit, Michigan, United States

University Of Nebrasaka Medical Center

Omaha, Nebraska, United States

Weill Cornell Medicine - New York Presbyterian Hospital

New York, New York, United States

Cleveland Clinic

Cleveland, Ohio, United States

Penn State Health

Hershey, Pennsylvania, United States

University of Utah

Salt Lake City, Utah, United States

Royal Prince Alfred Hospital

Sydney, New South Wales, Australia

Westmead Hospital

Westmead, New South Wales, Australia

Royal Brisbane and Women's Hospital

Herston, Queensland, Australia

Hospital Claude Huriez

Lille, France

Hôpital Necker Enfants Malades

Paris, France

Hôpital Universitaire Pitié-Salpêtrière

Paris, France

Azienda Ospedaliero Universitaria delle Marche

Ancona, Italy

ASST Grande Ospedale Metropolitano Niguarda

Milan, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Italy

Istituto Clinico Humanitas

Rozzano, Italy

IRCCS Ospedale Casa Sollievo della Sofferenza

San Giovanni Rotondo, Italy

Ospedale dell'Angelo di Mestre

Venezia, Italy

Vilnius University Hospital Santaros Klinikos

Vilnius, Lithuania

ICO l'Hospitalet - Hospital Duran i Reynals

Barcelona, Spain

Hospital Universitario Fundacion Jimenez Diaz

Madrid, Spain

Hospital Universitario Ramon y Cajal

Madrid, Spain

Hospital Universitato De La Princesa

Madrid, Spain

Hospital Universitario Virgen de la Arrixaca

Murcia, Spain

Clínica Universidad de Navarra

Pamplona, Spain

Anadolu Medical Center

Eskişehir, Turkey (Türkiye)

Gayrettepe Florence Nightingale Hastanesi

Istanbul, Turkey (Türkiye)

Koc University

Istanbul, Turkey (Türkiye)

Memorial Bahcelievler Hospital

Istanbul, Turkey (Türkiye)

Izmir Medicalpark Hospital

Izmir, Turkey (Türkiye)

İnonu University

Malatya, Turkey (Türkiye)

Dr Abdurrahman Yurtaslan Ankara Onkoloji Egitim ve Arastirma Hastanesi

Yenimahalle, Turkey (Türkiye)

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NCT05643638


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