Trial of Efficacy and Safety of MC0518 Versus Best Available Therapy in Participants With Steroid-Refractory Acute Graft Versus Host Disease

NCT ID: NCT06075706

Last Updated: 2026-01-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

48 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-11-13

Study Completion Date

2031-06-30

Brief Summary

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The purpose of this trial is the comparative evaluation of overall response rate (ORR) in paediatric participants with steroid-refractory acute graft-versus-host disease (SR-aGvHD) at Visit Day 28 after treatment with MC0518 or first used best available therapy (BAT).

Detailed Description

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Conditions

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Steroid-refractory Acute Graft-versus-host Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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MC0518

Participants will be treated with intravenous infusions of MC0518 at a dose of 1 to 2\*10\^6 cells per kilogram (cells/kg) (based on body weight at the Screening Visit). Infusions will be administered 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.

Group Type EXPERIMENTAL

MC0518

Intervention Type BIOLOGICAL

MC0518 will be intravenously infused immediately after thawing.

Best Available Therapy (BAT)

Participants will receive one of the following systemic BATs based on the Investigator's decision: extracorporeal photopheresis (ECP), anti-thymocyte globulin (ATG), etanercept, infliximab or ruxolitinib (RUX).

Group Type ACTIVE_COMPARATOR

BAT

Intervention Type BIOLOGICAL

BAT including ECP, ATG, etanercept, infliximab or RUX will be administered based on Investigator's decision.

Interventions

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MC0518

MC0518 will be intravenously infused immediately after thawing.

Intervention Type BIOLOGICAL

BAT

BAT including ECP, ATG, etanercept, infliximab or RUX will be administered based on Investigator's decision.

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.
Minimum Eligible Age

28 Days

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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medac GmbH

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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CHU de Bordeaux - Hopital des Enfants

Bordeaux, , France

Site Status

CHU Grenoble Alpes - Hopital Couple Enfant (HCE)

La Tronche, , France

Site Status

Centre Hospitalier Universitaire de Lille CHU Lille - Hopital Jeanne de Flandre HJF

Lille, , France

Site Status

Institut d'Hematologie et d'Oncologie Pediatrique (IHOPe)

Lyon, , France

Site Status

CHU de Marseille-Hopital de la Timone

Marseille, , France

Site Status

Centre Hospitalier Regional Universitaire (CHRU) Montpellier - hopital Arnaud de Villeneuve

Montpellier, , France

Site Status

CHU de Nantes - Hopital Mere Enfant

Nantes, , France

Site Status

Hopital Robert Debre

Paris, , France

Site Status

CHU de Rouen - Hopital Charles Nicolle

Rouen, , France

Site Status

CHRU de Strasbourg - Hopital de Hautepierre

Strasbourg, , France

Site Status

CHRU Nancy, Hopitaux de Brabois

Vandœuvre-lès-Nancy, , France

Site Status

Uniklinik RWTH Aachen, Klinik fur Kinder- und Jugendmedizin

Aachen, , Germany

Site Status

Universitaetsklinikum Essen

Essen, , Germany

Site Status

Klinikum der Johann Wolfgang Goethe

Frankfurt, , Germany

Site Status

Universitaetsklinikum Freiburg - Zentrum fuer Kinder- und Jugendmedizin (ZKJ)

Freiburg im Breisgau, , Germany

Site Status

Justus-Liebig-Universitaet Giessen

Giessen, , Germany

Site Status

Medizinische Hochschule Hannover MHH

Hanover, , Germany

Site Status

Department of Pediatrics, Jena University Hospital

Jena, , Germany

Site Status

Universitaetsklinikum Leipzig - Abteilung fuer Paediatrische Onkologie, Haematologie und Haemostaseologie

Leipzig, , Germany

Site Status

Universitaetsklinikum Muenster (UKM) - Klinik fuer Kinder- und Jugendmedizin - Paediatrische Haematologie und Onkologie

Münster, , Germany

Site Status

IRCCS Azienda Ospedaliero-Universitaria di Bologna Policlinico S.Orsola Malpighi

Bologna, , Italy

Site Status

Pediatric Clinic Onco Hematology San Gerardo Hospital

Monza, , Italy

Site Status

U.O.C. Oncoematologia Pediatrica, Fondazione IRCCS Policlinico San Matteo

Pavia, , Italy

Site Status

Hematology and Cellular Therapy Ospedale Bambino Gesu

Rome, , Italy

Site Status

A.O.U. Citta della Salute e della Scienza di Torino Ospedale Infantile Regina Margherita

Turin, , Italy

Site Status

Department of Pediatric Hematology, Oncology and BMT, Wroclaw Medical University

Wroclaw, Lower Silesian Voivodeship, Poland

Site Status

Dzieciecy Szpital Kliniczny im. A.Gebali w Lublinie

Lublin, , Poland

Site Status

Szpital Kliniczny im. Karola Jonschera UM

Poznan, , Poland

Site Status

Clinica Universitaria de Navarra

Pamplona, Navarre, Spain

Site Status

Hospital Universitario Vall dHebron

Barcelona, , Spain

Site Status

Hospital Sant Joan de Deu Barcelona (HSJDB)

Barcelona, , Spain

Site Status

Hospital Niño Jesus

Madrid, , Spain

Site Status

Hospital Infantil Universitario La Paz

Madrid, , Spain

Site Status

Instituto de Investigacion Biomedica de Malaga IBIMA - sede Hospital Regional Universitario de Malaga HRUM Hospital Carlos Haya

Málaga, , Spain

Site Status

Instituto Murciano de Investigacion Biosanitaria (IMIB) Virgen de la Arrixaca

Murcia, , Spain

Site Status

Hospital Universitari I politecnic La Fe Jose

Valencia, , Spain

Site Status

Countries

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France Germany Italy Poland Spain

Other Identifiers

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MC-MSC.2/aGvHD

Identifier Type: -

Identifier Source: org_study_id

2023-503952-28-00

Identifier Type: OTHER

Identifier Source: secondary_id

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