Treatment Of Steroid-Refractory Acute Graft-versus-host Disease With Mesenchymal Stromal Cells Versus Best Available Therapy

NCT ID: NCT04629833

Last Updated: 2025-08-13

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

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

210 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-08-16

Study Completion Date

2030-08-31

Brief Summary

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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).

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 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.

Group Type EXPERIMENTAL

MC0518

Intervention Type BIOLOGICAL

MC0518 will be intravenously infused immediately after thawing.

Best Available Therapy (BAT)

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).

Group Type ACTIVE_COMPARATOR

BAT

Intervention Type BIOLOGICAL

BAT including MMF, ECP, ATG, everolimus, and 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 MMF, ECP, ATG, everolimus, and RUX will be administered based on Investigator's decision.

Intervention Type BIOLOGICAL

Eligibility Criteria

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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).
Minimum Eligible Age

12 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

Principal Investigators

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Ann-Kristin Möller

Role: STUDY_DIRECTOR

medac GmbH

Locations

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Centre Hospitalier Universitaire (CHU) Amiens-Picardie - Hopital Sud

Amiens, , France

Site Status RECRUITING

CHU Jean Minjoz

Besançon, , France

Site Status RECRUITING

Hopital Michallon

Grenoble, , France

Site Status RECRUITING

CHRU Lille- Hopital Claude Huriez

Lille, , France

Site Status RECRUITING

CHU de Nantes - Hotel Dieu

Nantes, , France

Site Status RECRUITING

CHU de Nice Hopital Archet 1

Nice, , France

Site Status RECRUITING

Centre Hospitalier Lyon Sud Pavillon Marcel Berard 1G

Pierre-Bénite, , France

Site Status RECRUITING

Centre Hospitalier Universitaire CHU de Toulouse

Toulouse, , France

Site Status RECRUITING

Hopitaux De Brabois

Vandœuvre-lès-Nancy, , France

Site Status RECRUITING

Klinikum rechts der Isar

Munich, Bavaria, Germany

Site Status RECRUITING

Universitaetsklinikum Wuerzburg - Medizinische Klinik und Poliklinik II - Zentrum fuer Allogene Blutstammzelltransplantation

Würzburg, Bavaria, Germany

Site Status RECRUITING

Klinikum der Johann Wolfgang Goethe-Universitaet - Frankfurt am Main

Frankfurt am Main, Hesse, Germany

Site Status RECRUITING

Medizinische Hochschule Hannover

Hanover, Lower Saxony, Germany

Site Status RECRUITING

Uniklinik Koeln

Cologne, North Rhine-Westphalia, Germany

Site Status ACTIVE_NOT_RECRUITING

Universitaetsklinikum Essen - Klinik fuer Knochenmarktransplantation (KMT)

Essen, North Rhine-Westphalia, Germany

Site Status RECRUITING

Universitaetsklinikum Muenster

Münster, North Rhine-Westphalia, Germany

Site Status RECRUITING

Universitaetsklinikum Carl Gustav Carus Dresden

Dresden, Saxony, Germany

Site Status RECRUITING

Universitaetsklinikum Leipzig, Selbststaendige Abteilung fur Haematologie und Internistische Onkologie

Leipzig, Saxony, Germany

Site Status RECRUITING

Universitaetsklinikum Jena Klinik fuer Innere Medizin II, Haematologie und Onkologie

Jena, Thuringia, Germany

Site Status RECRUITING

Universitaetsklinikum Jena - Klinik fuer Paediatrische Haematologie und Onkologie

Jena, Thuringia, Germany

Site Status TERMINATED

Charite Universitaetsmedizin

Berlin, , Germany

Site Status WITHDRAWN

Helios Klinikum Berlin-Buch

Berlin, , Germany

Site Status RECRUITING

University Hospital Bonn, Medizinische Klinik III

Bonn, , Germany

Site Status RECRUITING

Universitaetsklinikum Essen - Klinik fuer Paediatrische Haematologie und Onkologie

Essen, , Germany

Site Status TERMINATED

Klinikum der Johann Wolfgang Goethe University - University - Klinik fuer Paediatrische Haematologie und Onkologie

Frankfurt, , Germany

Site Status RECRUITING

Universitaetsklinikum Freiburg - Zentrum fuer Kinder- und Jugendmedizin (ZKJ) - Klinik fuer Paediatrische Haematologie und Onkologie

Freiburg im Breisgau, , Germany

Site Status TERMINATED

Universitaetsklinikum Freiburg

Freiburg im Breisgau, , Germany

Site Status RECRUITING

Universitatsklinikum

Kiel, , Germany

Site Status RECRUITING

University Medical Center Mainz

Mainz, , Germany

Site Status RECRUITING

Universitaetsklinikum Mannheim

Mannheim, , Germany

Site Status RECRUITING

University Hospital Tuebingen Medical Center

Tübingen, , Germany

Site Status RECRUITING

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

Wroclaw, Lower Silesian Voivodeship, Poland

Site Status TERMINATED

Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie, Paastwowy Instytut Badawczy

Gliwice, , Poland

Site Status RECRUITING

Hospital Germans Trias i Pujol

Badalona, , Spain

Site Status RECRUITING

Hospital Universitario Vall dHebron

Barcelona, , Spain

Site Status RECRUITING

Institut Catal dOncologia

Barcelona, , Spain

Site Status RECRUITING

Hospital Universitario Ramon y Cajal

Madrid, , Spain

Site Status RECRUITING

Hospital Puerta De Hierro

Madrid, , Spain

Site Status RECRUITING

Hospital Universitario Carlos Haya

Málaga, , Spain

Site Status RECRUITING

Hospital Clinico Universitario de Valencia

Valencia, , Spain

Site Status RECRUITING

Hospital Universitari i politecnic La Fe

Valencia, , Spain

Site Status RECRUITING

Center for Allogeneic Stem Cell Transplantation and Cell Therapy (CAST), Karolinska Universitetssjukhuset Huddinge

Huddinge, , Sweden

Site Status TERMINATED

Countries

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France Germany Poland Spain Sweden

Central Contacts

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Clinical Trial Information

Role: CONTACT

+49 40103 8006-0

Facility Contacts

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Amandine Charbonnier

Role: primary

+33 3-22-45-60-00

Ana Berceanu

Role: primary

+33 3 81 66 90 97

Dr. Martin Carre

Role: primary

+ 33 04 76 76 56 63

Ibrahim Yakoub Agha

Role: primary

+33 3-20445713

Patrice Chevallier

Role: primary

+33 240-083269

Michael Loschi

Role: primary

+33 4 92 03 5558

Helene Labussiere-Wallet

Role: primary

+33 478862210

Anne Huynh

Role: primary

+33 5-31-15-61-89

Maud D'Aveni-Piney

Role: primary

+33 383-153-282

Mareike Verbeek

Role: primary

+49 89-41405336

Sabrina Kraus

Role: primary

+49 93120140411

Gesine Bug

Role: primary

+49 69-6301

Gernot Beutel

Role: primary

+49 511-532-3020

Thomas Schroeder

Role: primary

201 72385533 ext. 0049

Matthias Stelljes

Role: primary

+49 251-8352801

Desiree Kunadt

Role: primary

+49-351-45819523

Georg-Nikolaus Franke

Role: primary

+49 3419713842

Inken Hilgendorf

Role: primary

+49 3641-9-324513

Judith Niederland

Role: primary

+49 0309401-12195

Tobias Holderried

Role: primary

+49 228-287-17233

Peter Bader

Role: primary

+49 69-6301-7541

Robert Zeiser

Role: primary

+49 076-127034580

Friedrich Stoelzel

Role: primary

49-351-4584673

Eva Wagner-Drouet

Role: primary

+49 6131-172712

Stefan Klein

Role: primary

+49 621-3830

Wolfgang Bethge

Role: primary

+49 70712983176

Sebastian Giebel

Role: primary

48-32-278 85 23

Anna Torrent Catarineu

Role: primary

+34 93-497-89-87

Maria Sola Soto

Role: primary

+34 93 2746000

Alberto Mussetti

Role: primary

+34 667-069-283

Francisco Javier Lopez Jimenez

Role: primary

+34 913368367

Rafael Duarte Palomino

Role: primary

+34 911916303

Maria Jesus Pascual

Role: primary

+34-951-29 00 00

Carlos Solano

Role: primary

+34 96-1973838

Jaime Sanz Cabeller

Role: primary

+34-96-12 44 929

Other Identifiers

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MC-MSC.1/aGvHD

Identifier Type: -

Identifier Source: org_study_id

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