A Study of CYP-001 in Combination with Corticosteroids in Adults with High-risk AGvHD

Purpose

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.

Condition

  • Graft Versus Host Disease, Acute

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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 Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
CYP-001 plus corticosteroids
  • 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.
  • 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.
Placebo Comparator
Placebo plus corticosteroids
  • Biological: Placebo
    The placebo product is identical to CYP-001, except that it contains no active agent
  • 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.

Recruiting Locations

Banner MD Anderson
Phoenix 5308655, Arizona 5551752 85012

Mayo Clinic Hospital
Phoenix 5308655, Arizona 5551752 85288

University of Arkansas Medical Center
Little Rock 4119403, Arkansas 4099753 72205

Mayo Clinic Hospital
Jacksonville 4160021, Florida 4155751 32224

Memorial healthcare System
Pembroke Pines 4168139, Florida 4155751 33026

BMT Group of Georgia
Atlanta 4180439, Georgia 4197000 30342

Northwestern University
Evanston 4891382, Illinois 4896861 60208

Karmanos Cancer Institute
Detroit 4990729, Michigan 5001836 48201

University Of Nebrasaka Medical Center
Omaha 5074472, Nebraska 5073708 68198

Cleveland Clinic
Cleveland 5150529, Ohio 5165418 44195

Penn State Health
Hershey 5193342, Pennsylvania 6254927 17033

University of Utah
Salt Lake City 5780993, Utah 5549030 84112

More Details

NCT ID
NCT05643638
Status
Recruiting
Sponsor
Cynata Therapeutics Limited

Study Contact

Cynata Project Manager
+61 3 7067 6940
clinical@cynata.com