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Comparing MC0518 to standard treatments for steroid-refractory acute graft-versus-host disease in children

A Randomised, Open-label, Controlled, Multicentre, Phase 2 Trial of First-line Treatment with Mesenchymal Stromal Cells MC0518 Versus Best Available Therapy in Paediatric Participants with Steroid-refractory Acute Graft-versus-host Disease After Allogeneic Stem Cell Transplantation (BALDER Trial)

Phase 2 Interventional medac GmbH · NCT06075706

This study is testing a new treatment called MC0518 to see if it helps children with steroid-refractory acute graft-versus-host disease better than standard therapies.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment48 (estimated)
Ages28 Days to 17 Years
SexAll
Sponsormedac GmbH Industry-sponsored
Drugs / interventionsinfliximab, ruxolitinib, methotrexate, cyclophosphamide, prednisone
Locations38 sites (Bordeaux and 37 other locations)
Trial IDNCT06075706 on ClinicalTrials.gov

What this trial studies

This trial evaluates the efficacy and safety of MC0518 compared to the best available therapy in pediatric patients suffering from steroid-refractory acute graft-versus-host disease (SR-aGvHD). Participants will be assessed for their overall response rate (ORR) at 28 days post-treatment. The study aims to provide insights into the effectiveness of MC0518 as a potential alternative treatment for this challenging condition. Inclusion criteria focus on children who have undergone allogeneic hematopoietic stem cell transplantation and have not responded to first-line treatments.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients diagnosed with Grade II to IV acute graft-versus-host disease who have not responded to standard steroid treatments.

Not a fit: Patients who have not undergone allogeneic hematopoietic stem cell transplantation or those with Grade I aGvHD may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could offer a new therapeutic option for children with SR-aGvHD who have limited responses to existing therapies.

How similar studies have performed: While there have been studies on treatments for aGvHD, the specific use of MC0518 in this context is novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. 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 hematological malignant disease or neuroblastoma.
2. Participant has been clinically diagnosed with Grade II to IV aGvHD according to Harris et al. A biopsy of the involved organs with aGvHD is encouraged but not required.
3. Participant has experienced failure of previous first-line aGvHD treatment (that is, SR-aGvHD), defined as:

   * aGvHD progression within 3 to 5 days of therapy onset with \>=2 milligram per kilogram per day (mg/kg/day) of prednisone equivalent or
   * failure to improve within 5 to 7 days of treatment initiation with \>=2 mg/kg/day of prednisone equivalent or
   * incomplete response after greater than (\>) 28 days of immunosuppressive treatment including at least 5 days with \>=2 mg/kg/day of prednisone equivalent.
4. Male or female participant who is \>=28 days and \<18 years of age and has a minimum body weight of 3.2 kilograms (kg) at the Screening Visit.
5. Participant has an estimated life expectancy of \>28 days.
6. Participant, if female and of childbearing potential, agrees to use a highly effective contraceptive measure starting at the Screening Visit and continuing throughout the entire trial period.
7. Participant, if a fertile male, agrees to sexual abstinence or to use a condom during sexual activity with their female partner of childbearing potential or pregnant partner. Additionally, if their partner is a woman of childbearing potential (WOCBP), then their partner must use an additional highly effective contraceptive method during sexual activity starting at the Screening Visit and continuing throughout the entire trial period.
8. A written informed consent of the participant's parent(s) / legal guardian(s) (and participant's assent, when applicable) has been obtained according to national regulations.

Exclusion Criteria:

1. Participant has overt relapse or progression or persistence of the underlying disease.
2. Participant has received the last HSCT for a solid tumor disease other than neuroblastoma.
3. Participant has graft-versus-host disease overlap syndrome.
4. 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, mycophenolate mofetil, methotrexate, abatacept, or cyclophosphamide. Note: In vitro or in vivo graft manipulation to prevent graft-versus-host disease (example, T-cell depletion) during HSCT is permitted. Restart of initial prophylaxis with calcineurin inhibitors, mammalian target of rapamycin inhibitors, or mycophenolate mofetil after aGvHD onset is permitted.
5. Participant has received prior mesenchymal stromal cell (MSC) treatment, including MC0518/Obnitix®.
6. Participant has a known pregnancy (as confirmed by a positive pregnancy test result at the Screening Visit) and / or is breastfeeding.
7. Participant has a known hypersensitivity to MC0518 and / or its excipients (dimethyl sulfoxide, human serum albumin, isotonic sodium chloride solution).
8. Participant has a known hypersensitivity or any contraindication to the Investigator's choice BAT (extracorporeal photopheresis, anti thymocyte globulin, etanercept, infliximab, or ruxolitinib) and / or its excipients. For a list of excipients please refer to the respective Summary of Product Characteristics.
9. Participant has an underlying or current medical or psychiatric condition that, in the opinion of the Investigator, would interfere with the evaluation of the participant.
10. Participant has an uncontrolled infection (examples, sepsis or multi-organ failure) including significant bacterial, fungal, viral, or parasitic infection requiring treatment.
11. Participant has received treatment with any other investigational agent within 30 days or 5 half-lives (whichever is longer) before the Screening Visit.

Where this trial is running

Bordeaux and 37 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.