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CYP-001 combined with corticosteroids for high-risk acute GvHD

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

Phase 2 Interventional Cynata Therapeutics Limited · NCT05643638

This study is testing if a new treatment called CYP-001, combined with corticosteroids, can help adults with high-risk acute graft versus host disease after a stem cell transplant feel better compared to a placebo with corticosteroids.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment60 (estimated)
Ages18 Years and up
SexAll
SponsorCynata Therapeutics Limited Industry-sponsored
Locations39 sites (Phoenix, Arizona and 38 other locations)
Trial IDNCT05643638 on ClinicalTrials.gov

What this trial studies

This phase 2 interventional study aims to evaluate the efficacy of CYP-001, a type of induced pluripotent stem cell-derived mesenchymal stem cell, in combination with corticosteroids compared to a placebo plus corticosteroids in adults who have undergone allogeneic hematologic stem cell transplants and are diagnosed with high-risk acute graft versus host disease (aGvHD). Participants will be randomized to receive either the treatment or placebo on specified days, with ongoing corticosteroid therapy as per institutional guidelines. The severity of aGvHD will be monitored using established MAGIC guidelines throughout the study period, which includes visits up to 100 days and follow-ups for up to 24 months.

Who should consider this trial

Good fit: Ideal candidates are adults who have undergone allogeneic hematopoietic stem cell transplants and are clinically diagnosed with high-risk acute GvHD requiring systemic corticosteroid therapy.

Not a fit: Patients with chronic GvHD, those who have received prior systemic treatments for aGvHD, or those with significant comorbidities may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve outcomes for patients suffering from high-risk acute graft versus host disease.

How similar studies have performed: While this approach is innovative, similar studies have shown promise in using stem cell therapies for treating aGvHD, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Phoenix, Arizona and 38 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Graft Versus Host Disease, Acute
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.