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NCT Number: NCT05643638

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Graft Versus Host Disease, Acute Graft vs Host Disease Immune System Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Royal Prince Alfred Hospital, Sydney, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Placebo

Biological

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

Corticosteroids

Drug

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.

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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

  8. 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.

  9. Incidence of chronic GvHD

    Time frame: 2 years

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

  10. Weekly cumulative steroid dose

    Time frame: 100 days

    The total corticosteroid dose administered each week

  11. 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.

  12. 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

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

    Time frame: 2 years

    Assessment of safety

Sponsors and collaborators

Lead sponsor

Cynata Therapeutics Limited

Industry

Registry information

Official study 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

Important dates

Study start
2024
Primary completion
2026
Study completion
2027
First posted
Dec 9, 2022
Registry last updated
May 26, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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