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Treatment for steroid-refractory acute graft-versus-host disease using mesenchymal stromal cells

A Randomised, Open-label, Multicentre, Phase 3 Trial of First-line Treatment With Mesenchymal Stromal Cells MC0518 Versus Best Available Therapy in Adult and Adolescent Subjects With Steroid-refractory Acute Graft-versus-host Disease After Allogeneic Haematopoietic Stem Cell Transplantation (IDUNN Trial)

Phase 3 Interventional medac GmbH · NCT04629833

This study is testing a new type of cell therapy to see if it can help adults and teens with a tough form of graft-versus-host disease that hasn't improved with standard treatments.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment210 (estimated)
Ages12 Years and up
SexAll
Sponsormedac GmbH Industry-sponsored
Drugs / interventionsmethotrexate, cyclophosphamide, prednisone
Locations42 sites (Amiens and 41 other locations)
Trial IDNCT04629833 on ClinicalTrials.gov

What this trial studies

This trial aims to compare the effectiveness of MC0518, a type of mesenchymal stromal cell therapy, against the best available therapy (BAT) for patients suffering from steroid-refractory acute graft-versus-host disease (SR-aGvHD). The primary goal is to assess the overall response rate at Day 28 and overall survival up to 24 months. Participants include adults and adolescents who have previously undergone allogeneic hematopoietic stem cell transplantation and have not responded to first-line treatments. The study is designed to provide insights into a potentially more effective treatment option for this challenging condition.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 12 years and older who have been diagnosed with Grade II to IV acute graft-versus-host disease and have not responded to previous steroid treatments.

Not a fit: Patients who have not undergone allogeneic hematopoietic stem cell transplantation or those with a life expectancy of less than 28 days may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve outcomes for patients with steroid-refractory acute graft-versus-host disease.

How similar studies have performed: Other studies have explored mesenchymal stromal cell therapies for similar conditions, showing promising results, but this specific approach is still being evaluated.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Participant had a previous allogeneic HSCT as indicated for non-malignant (including inborn errors of metabolism, primary immunodeficiencies, haemoglobinopathies, and bone marrow failure syndromes) or haematological malignant disease, irrespective of human leukocyte antigen match
* Participant has been clinically diagnosed with Grade II to IV aGvHD at the Screening Visit
* Participant has experienced failure of previous first-line aGvHD treatment (ie, SR-aGvHD), defined as: a) aGvHD progression within 3 to 5 days of therapy onset with \>= 2 mg/kg/day of prednisone equivalent or b) failure to improve within 5 to 7 days of treatment initiation with \>= 2 mg/kg/day of prednisone equivalent or c) incomplete response after \> 28 days of immunosuppressive treatment including at least 5 days with \>= 2 mg/kg/day of prednisone equivalent
* Participant has an estimated life expectancy \> 28 days at the Screening Visit
* Male or female participant who is \>= 12 years of age at the Screening Visit

Exclusion Criteria:

* Participant has overt relapse or progression or persistence of the underlying disease at the Screening Visit
* Participant has received the last HSCT for a solid tumour disease
* Participant has GvHD overlap syndrome at the Screening Visit
* Participant has received systemic first line treatment for aGvHD other than steroids and a prophylaxis with other than calcineurin inhibitors, mammalian target of rapamycin (mTOR) inhibitors, anti-thymocyte globulin (ATG), mycophenolate mofetil (MMF), methotrexate (MTX), and / or cyclophosphamide before the Screening Visit
* Participant has a known pregnancy (as confirmed by a positive pregnancy test at the Screening Visit) and or is breastfeeding at the Screening Visit
* Participant has received treatment with any other investigational agent within 30 days or 5 half-lives (whichever is longer) before the Screening Visit (compliance to be confirmed for the period between the Screening Visit and the Baseline Visit at the Baseline Visit).

Where this trial is running

Amiens and 41 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 Steroid-refractory Acute Graft-versus-host Disease
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.