Steroid-refractory Acute Graft-versus-host DiseasePhase 3IDUNNNCT04629833
Treatment Of Steroid-Refractory Acute Graft-versus-host Disease With Mesenchymal Stromal Cells Versus Best Available Therapy
A Phase 3 study testing MC0518 and BAT in people with Steroid-refractory Acute Graft-versus-host Disease, run by medac GmbH at 42 sites in 5 countries.
Trial summary
The primary purpose of this trial is to demonstrate the superiority of MC0518 compared to the first used best available therapy (BAT) with respect to overall response rate (ORR) at Day 28 and/or overall survival (OS) until Visit Month 24 in adult and adolescent subjects with steroid-refractory acute graft-versus-host… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Steroid-refractory Acute Graft-versus-host Disease
- What is being tested?
- MC0518 and BAT
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 210 participants
- Where
- 42 sitesin 5 countries
- Study dates
- 16 Aug 2021to Aug 2030
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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).
Trial locations and contacts
42 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
42 sites in 5 countries
Germany22 sites, 17 recruiting
- Withdrawn
Charite Universitaetsmedizin
Berlin, 13353
- Recruiting
Helios Klinikum Berlin-Buch
Berlin, 287706
Judith Niederland, +49 0309401-12195, judith.niederland@helios-gesundheit.de
- Recruiting
University Hospital Bonn, Medizinische Klinik III
Bonn, 53127
Tobias Holderried, +49 228-287-17233, tobias.holderried@ukbonn.de
- Active, not recruiting
Uniklinik Koeln
Cologne, North Rhine-Westphalia, 50937
- Recruiting
Universitaetsklinikum Carl Gustav Carus Dresden
Dresden, Saxony, 01307
Desiree Kunadt, +49-351-45819523, desiree.kunadt@uniklinikum-dresden.de
- Recruiting
Universitaetsklinikum Essen - Klinik fuer Knochenmarktransplantation (KMT)
Essen, North Rhine-Westphalia, 45147
Thomas Schroeder, 201 72385533 ext. 0049, thomas.schroeder@uk-essen.de
- Terminated
Universitaetsklinikum Essen - Klinik fuer Paediatrische Haematologie und Onkologie
Essen, 45147
- Recruiting
Klinikum der Johann Wolfgang Goethe University - University - Klinik fuer Paediatrische Haematologie und Onkologie
Frankfurt, 60590
Peter Bader, +49 69-6301-7541, peter.bader@kgu.de
- Recruiting
Klinikum der Johann Wolfgang Goethe-Universitaet - Frankfurt am Main
Frankfurt am Main, Hesse, 60590
Gesine Bug, +49 69-6301, g.bug@em.uni-frankfurt.de
- Terminated
Universitaetsklinikum Freiburg - Zentrum fuer Kinder- und Jugendmedizin (ZKJ) - Klinik fuer Paediatrische Haematologie und Onkologie
Freiburg im Breisgau, 151595
- Recruiting
Universitaetsklinikum Freiburg
Freiburg im Breisgau, 79106
Robert Zeiser, +49 076-127034580, robert.zeiser@uniklinik-freiburg.de
- Recruiting
Medizinische Hochschule Hannover
Hanover, Lower Saxony, 30625
Gernot Beutel, +49 511-532-3020, beutel.gernot@mh-hannover.de
- Recruiting
Universitaetsklinikum Jena Klinik fuer Innere Medizin II, Haematologie und Onkologie
Jena, Thuringia, 07740
Inken Hilgendorf, +49 3641-9-324513, inken.hilgendorf@med.uni-jena.de
- Terminated
Universitaetsklinikum Jena - Klinik fuer Paediatrische Haematologie und Onkologie
Jena, Thuringia, 07747
- Recruiting
Universitatsklinikum
Kiel, 24105
Friedrich Stoelzel, 49-351-4584673, friedrich.stoelzel@uksh.de
- Recruiting
Universitaetsklinikum Leipzig, Selbststaendige Abteilung fur Haematologie und Internistische Onkologie
Leipzig, Saxony, 04103
Georg-Nikolaus Franke, +49 3419713842, georg-nikolaus.franke@medizin.uni-leipzig.de
- Recruiting
University Medical Center Mainz
Mainz, 55131
Eva Wagner-Drouet, +49 6131-172712, eva.wagner@unimedizin-mainz.de
- Recruiting
Universitaetsklinikum Mannheim
Mannheim, 68167
Stefan Klein, +49 621-3830, stefan.klein@umm.de
- Recruiting
Klinikum rechts der Isar
Munich, Bavaria, 81675
Mareike Verbeek, +49 89-41405336, mareike.verbeek@tum.de
- Recruiting
Universitaetsklinikum Muenster
Münster, North Rhine-Westphalia, 48149
Matthias Stelljes, +49 251-8352801, matthias.stelljes@ukmuenster.de
- Recruiting
University Hospital Tuebingen Medical Center
Tübingen, 72076
Wolfgang Bethge, +49 70712983176, wolfgang.bethge@med.uni-tuebingen.de
- Recruiting
Universitaetsklinikum Wuerzburg - Medizinische Klinik und Poliklinik II - Zentrum fuer Allogene Blutstammzelltransplantation
Würzburg, Bavaria, 97080
Sabrina Kraus, +49 93120140411, kraus_s3@klinik-uni-wuerzburg.de
France9 sites, 9 recruiting
- Recruiting
Centre Hospitalier Universitaire (CHU) Amiens-Picardie - Hopital Sud
Amiens, 80054
Amandine Charbonnier, +33 3-22-45-60-00, charbonnier.amandine@chu-amiens.fr
- Recruiting
CHU Jean Minjoz
Besançon, 25030
Ana Berceanu, +33 3 81 66 90 97, edaguindau@chu-besancon.fr
- Recruiting
Hopital Michallon
Grenoble, 38700
Dr. Martin Carre, + 33 04 76 76 56 63, MCarre1@chu-grenoble.fr
- Recruiting
CHRU Lille- Hopital Claude Huriez
Lille, 59000
Ibrahim Yakoub Agha, +33 3-20445713, ibrahim.yakoubagha@chru-lille.fr
- Recruiting
CHU de Nantes - Hotel Dieu
Nantes, 44093
Patrice Chevallier, +33 240-083269, patrice.chevallier@chu-nantes.fr
- Recruiting
CHU de Nice Hopital Archet 1
Nice, 06200
Michael Loschi, +33 4 92 03 5558, loschi.m@chu-nice.fr
- Recruiting
Centre Hospitalier Lyon Sud Pavillon Marcel Berard 1G
Pierre-Bénite, 69630
Helene Labussiere-Wallet, +33 478862210, helene.labussiere-wallet@chu-lyon.fr
- Recruiting
Centre Hospitalier Universitaire CHU de Toulouse
Toulouse, 31059
Anne Huynh, +33 5-31-15-61-89, huynh.anne@iuct-oncopole.fr
- Recruiting
Hopitaux De Brabois
Vandœuvre-lès-Nancy, 54511
Maud D'Aveni-Piney, +33 383-153-282, m.daveni-piney@chru-nancy.fr
Spain8 sites, 8 recruiting
- Recruiting
Hospital Germans Trias i Pujol
Badalona, 08916
Anna Torrent Catarineu, +34 93-497-89-87, atorrent@iconcologia.net
- Recruiting
Hospital Universitario Vall dHebron
Barcelona, 08035
Maria Sola Soto, +34 93 2746000, mariasola@vhio.net
- Recruiting
Institut Catal dOncologia
Barcelona, 08908
Alberto Mussetti, +34 667-069-283, amussetti@iconcologia.net
- Recruiting
Hospital Universitario Ramon y Cajal
Madrid, 28034
Francisco Javier Lopez Jimenez, +34 913368367, jljimenez@salud.madrid.org
- Recruiting
Hospital Puerta De Hierro
Madrid, 28222
Rafael Duarte Palomino, +34 911916303, rduarte.work@gmail.com
- Recruiting
Hospital Universitario Carlos Haya
Málaga, 29010
Maria Jesus Pascual, +34-951-29 00 00, mjcascon@gmail.com
- Recruiting
Hospital Clinico Universitario de Valencia
Valencia, 46010
Carlos Solano, +34 96-1973838, carlos.solano@uv.es
- Recruiting
Hospital Universitari i politecnic La Fe
Valencia, 46026
Jaime Sanz Cabeller, +34-96-12 44 929, sanz_jai@gva.es
Poland2 sites, 1 recruiting
- Recruiting
Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie, Paastwowy Instytut Badawczy
Gliwice, 44-102
Sebastian Giebel, 48-32-278 85 23, ktsbadaniakliniczne@io.gliwice.pl
- Terminated
Department of Pediatric Hematology, Oncology and BMT, Wroclaw Medical University
Wroclaw, Lower Silesian Voivodeship, 50-556
Sweden1 site
- Terminated
Center for Allogeneic Stem Cell Transplantation and Cell Therapy (CAST), Karolinska Universitetssjukhuset Huddinge
Huddinge, 126464
About this study
Official title: 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)
The primary purpose of this trial is to demonstrate the superiority of MC0518 compared to the first used best available therapy (BAT) with respect to overall response rate (ORR) at Day 28 and/or overall survival (OS) until Visit Month 24 in adult and adolescent subjects with steroid-refractory acute graft-versus-host disease (SR-aGvHD).
- Being tested
- MC0518, BAT
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MC0518Biological
MC0518 will be intravenously infused immediately after thawing.
BATBiological
BAT including MMF, ECP, ATG, everolimus, and RUX will be administered based on Investigator's decision.
| Study group | Role | What they receive |
|---|---|---|
| MC0518 | Experimental | Participants will receive MC0518 1-2 million cells/ kilogram infusions (based on body weight at the Screening Visit) once a week for 4 weeks (Visit Day 1, 8, 15, and 22). Participants with partial response (PR) on Day 28 will have 2 additional MC0518 infusions administered on Day 29 and 36. Receives: Biological: MC0518 |
| Best Available Therapy (BAT) | Active comparator | Participants will receive any one of the following systemic BATs based on the Investigator's decision: mycophenolate mofetil (MMF), extracorporeal photopheresis (ECP), anti-thymocyte globulin (ATG), everolimus, and ruxolitinib (RUX). Receives: Biological: BAT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response (OR)
OR is defined as complete response (CR) or partial response (PR) at Day 28 relative to aGvHD status at baseline. CR is defined as resolution of aGvHD in all involved organs. PR is defined as improvement in 1 stage in 1 or more organs involved with aGvHD symptoms without progression in others. Number of participants with OR will be reported.
Measured: Day 28
Overall Survival
Overall survival is defined as the time from randomization to the date of death due to any cause.
Measured: Up to Month 24
Secondary outcomes (21)
Freedom from Treatment Failure (FFTF)
FFTF is defined as death, relapse or progression of the underlying disease, or addition or change to any further systemic immunosuppressive aGvHD therapy. Number of participants with FFTF will be reported.
Measured: Up to 6 months
Acute Graft-versus-host Disease (aGvHD) Response
Number of participants with aGvHD response will be reported. aGvHD response will be categorized as OR (CR + PR), CR, PR, and NR. NR is defined as the absence of CR or PR.
Measured: Days 28, 60, 100 and 180
Change from Baseline in aGvHD Grades
aGvHD grades: Grade 0- no organ involvement (ie, Stage 0 skin, Stage 0 liver, and Stage 0 GI); Grade I-Stage 1 - 2 skin without liver/GI involvement; Grade II- Stage 3 skin and / or Stage 1 liver and / or Stage 1 GI; Grade III- Stage 2 - 3 liver and / or Stage 2 - 3 GI; Grade IV- Stage 4 skin and / or Stage 4 liver and/or Stage 4 GI.
Measured: Baseline and Days 8, 15, 22, 28, 60, 100 and 180
Time to Response
Time to response is defined as the time from the date of the first treatment administration to the date of response.
Measured: Up to Month 24
Duration of Response
Duration is calculated from time from the first OR (CR or PR) until the time point of no aGvHD response in comparison to baseline.
Measured: Up to Month 24
Best Overall Response (OR)
Best OR is defined as the achievement of an OR at any time point up to and including Day 28. Number of participants with best OR will be reported.
Measured: Up to Day 28
Cumulative Dose of Steroids for SR-aGvHD per Kilogram (kg) of Body Weight
The cumulative dose of steroids given for SR-aGvHD per kg of body weight from baseline until Day 60 and until Visit Month 24 will be analyzed.
Measured: Up to Day 60 and Month 24
Number of Participants with Chronic Graft-versus-host Disease (cGvHD)
Number of participants with cGvHD will be reported.
Measured: Day 60 to Month 24
Time to Chronic Graft-versus-host Disease (cGvHD)
Time to cGvHD is defined as the time between the last day of haematopoietic stem cell transplantation (HSCT) to the first episode of cGvHD.
Measured: Day 60 to Month 24
Number of Participants with Graft Failure (GF)
Number of participants with GF will be reported.
Measured: Up to Month 24
Number of Participants with Relapse or Progression in Participants with Underlying Malignant Disease
Number of participants with relapse or progression in participants with underlying malignant disease will be reported.
Measured: Up to Month 24
Time to Relapse or Progression in Participants with Underlying Malignant Disease
Time to relapse or progression in participants with underlying malignant disease will be reported.
Measured: Up to Month 24
Event-free survival (EFS)
EFS is defined as the time from the date of randomization to the date of the event. An event is defined as GF, relapse or progression of the underlying disease, or death due to any cause.
Measured: Up to Month 24
Non-relapse Mortality (NRM)
NRM is defined as the time from the date of randomisation to the date of the event. An event is defined as death without previous relapse or progression of the underlying disease.
Measured: Up to Month 24
Number of Participants with Adverse Events (AEs) and Adverse Reactions (ARs)
Measured: Until Day 60 or until 30 days after last administration of trial treatment, whichever is later (Up to Month 24)
Number of Participants with Adverse Events (AEs) and Adverse Reactions (ARs) by Severity
Severity will be graded based on Common Terminology Criteria for Adverse Events (CTCAE) v5.0: Grade 1- Mild; Grade 2- Moderate; Grade 3- Severe; Grade 4- Life-threatening consequences; urgent intervention indicated; Grade 4- Death related to the AE.
Measured: Until Day 60 or until 30 days after last administration of trial treatment, whichever is later (Up to Month 24)
Change from Baseline in Performance score based on Karnofsky scale (recipient age >= 16 years)
The Karnofsky performance score (KPS), which is reported on an ordinal scale from 0 to 100, provides a rough measure of the participant's well-being, including their ability to conduct activities of daily living and functional capacity. Higher score indicates normal, no complaints and no evidence of disease.
Measured: Baseline, Days 8, 15, 22, 28, 60 and 100
Change from Baseline in Performance score based on Lansky Scale
A Lansky score (recipient age greater than or equal to [>=] 1 years and less than [<] 16 years) will be recorded pre-treatment and measured serially at regular intervals after treatment. The score is a standard performance score that measures overall function of the child with a scale range from 0 to 100. Higher score indicates full activeness.
Measured: Baseline, Days 8, 15, 22, 28, 60 and 100
Change from Baseline in EuroQol-5D-5L (EQ-5D-5L): Health Status Index (HSI)
EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L descriptive system and EQ VAS. EQ-5D-5L descriptive system comprises of 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each has 5 levels of perceived problems (1-no problem, 2-slight problems, 3-moderate problems, 4-severe problems, 5-extreme problems). Participant selects answer for each of 5 dimensions considering response that best matches his/her health "TODAY". Responses were used to generate a HSI. HSI ranges from 0 (dead) to 1.00 (full health).
Measured: Baseline, Days 28, 60, 100 and 180
Change from Baseline in EuroQol-5D-5L (EQ-5D-5L): Visual Analogue Scale (VAS)
EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L descriptive system and EQ VAS. The EQ VAS self-rating records the respondent's own assessment of his or her overall health status at the time of completion, on a scale of 0 (the worst health you can imagine) to 100 (the best health you can imagine).
Measured: Baseline, Days 28, 60, 100 and 180
Change from Baseline in Functional Assessment of Cancer Therapy-Bone Marrow Transplantation (FACT-BMT) Score
The FACT-BMT questionnaire was designed to measure the quality of life in subjects undergoing bone marrow (BM) transplantation. It consists of the following categories of assessment: physical well-being, social / family well-being, emotional well-being, functional well-being, and additional miscellaneous concerns that the subject may have concerning their healthcare, persons involved in their life, and other emotions and incapabilities. Score ranges from 0-164, with higher score indicating better quality of life.
Measured: Baseline, Days 28, 60, 100 and 180
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2020
- Study start16 Aug 2021
- Primary completionAug 2027 (estimated)
- Study completionAug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Steroid-refractory Acute Graft-versus-host DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 210 participants (estimated)
Sponsor and investigators
- Lead sponsor
- medac GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ann-Kristin Möller, Study director, medac GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04629833
- Sponsor's protocol ID
- MC-MSC.1/aGvHD
- Acronym
- IDUNN
- Status last verified
- Aug 2025
- First submitted
- 9 Nov 2020
- Last updated
- 13 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT04629833
- Acronym / sponsor ID
- IDUNN / MC-MSC.1/aGvHD
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 210 participants (estimated)
- Lead sponsor
- medac GmbHIndustry (pharma, biotech, devices)
- Study start
- 16 Aug 2021 (actual)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Aug 2030 (estimated)
- First posted
- 16 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary purpose of this trial is to demonstrate the superiority of MC0518 compared to the first used best available therapy (BAT) with respect to overall response rate (ORR) at Day 28 and/or overall survival (OS) until Visit Month 24 in adult and adolescent subjects with steroid-refractory acute graft-versus-host disease (SR-aGvHD).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MC0518 | Experimental | Biological: MC0518Participants will receive MC0518 1-2 million cells/ kilogram infusions (based on body weight at the Screening Visit) once a week for 4 weeks (Visit Day 1, 8, 15, and 22). Participants with partial response (PR) on Day 28 will have 2 additional MC0518 infusions administered on Day 29 and 36. |
| Best Available Therapy (BAT) | Active comparator | Biological: BATParticipants will receive any one of the following systemic BATs based on the Investigator's decision: mycophenolate mofetil (MMF), extracorporeal photopheresis (ECP), anti-thymocyte globulin (ATG), everolimus, and ruxolitinib (RUX). |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MC0518MC0518 will be intravenously infused immediately after thawing. | Biological | MC0518 |
| BATBAT including MMF, ECP, ATG, everolimus, and RUX will be administered based on Investigator's decision. | Biological | Best Available Therapy (BAT) |
Primary outcome
Overall Response (OR)
Time frame: Day 28
OR is defined as complete response (CR) or partial response (PR) at Day 28 relative to aGvHD status at baseline. CR is defined as resolution of aGvHD in all involved organs. PR is defined as improvement in 1 stage in 1 or more organs involved with aGvHD symptoms without progression in others. Number of participants with OR will be reported.
Overall Survival
Time frame: Up to Month 24
Overall survival is defined as the time from randomization to the date of death due to any cause.
21 secondary outcomes
Freedom from Treatment Failure (FFTF)
Time frame: Up to 6 months
Acute Graft-versus-host Disease (aGvHD) Response
Time frame: Days 28, 60, 100 and 180
Change from Baseline in aGvHD Grades
Time frame: Baseline and Days 8, 15, 22, 28, 60, 100 and 180
Time to Response
Time frame: Up to Month 24
Duration of Response
Time frame: Up to Month 24
Best Overall Response (OR)
Time frame: Up to Day 28
Cumulative Dose of Steroids for SR-aGvHD per Kilogram (kg) of Body Weight
Time frame: Up to Day 60 and Month 24
Number of Participants with Chronic Graft-versus-host Disease (cGvHD)
Time frame: Day 60 to Month 24
Time to Chronic Graft-versus-host Disease (cGvHD)
Time frame: Day 60 to Month 24
Number of Participants with Graft Failure (GF)
Time frame: Up to Month 24
Number of Participants with Relapse or Progression in Participants with Underlying Malignant Disease
Time frame: Up to Month 24
Time to Relapse or Progression in Participants with Underlying Malignant Disease
Time frame: Up to Month 24
Event-free survival (EFS)
Time frame: Up to Month 24
Non-relapse Mortality (NRM)
Time frame: Up to Month 24
Number of Participants with Adverse Events (AEs) and Adverse Reactions (ARs)
Time frame: Until Day 60 or until 30 days after last administration of trial treatment, whichever is later (Up to Month 24)
Number of Participants with Adverse Events (AEs) and Adverse Reactions (ARs) by Severity
Time frame: Until Day 60 or until 30 days after last administration of trial treatment, whichever is later (Up to Month 24)
Change from Baseline in Performance score based on Karnofsky scale (recipient age >= 16 years)
Time frame: Baseline, Days 8, 15, 22, 28, 60 and 100
Change from Baseline in Performance score based on Lansky Scale
Time frame: Baseline, Days 8, 15, 22, 28, 60 and 100
Change from Baseline in EuroQol-5D-5L (EQ-5D-5L): Health Status Index (HSI)
Time frame: Baseline, Days 28, 60, 100 and 180
Change from Baseline in EuroQol-5D-5L (EQ-5D-5L): Visual Analogue Scale (VAS)
Time frame: Baseline, Days 28, 60, 100 and 180
Change from Baseline in Functional Assessment of Cancer Therapy-Bone Marrow Transplantation (FACT-BMT) Score
Time frame: Baseline, Days 28, 60, 100 and 180
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ann-Kristin Möller study director
medac GmbH
42 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing MC0518 and BAT in people with Steroid-refractory Acute Graft-versus-host Disease, run by medac GmbH at 42 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 42 sites in Germany, France, Spain, Poland, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trial Information (+49 40103 8006-0, ClinicalTrialInformation@medac.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response (OR) (time frame: Day 28). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Aug 2025.