MC0518: MC0518 will be intravenously infused immediately after thawing.
BAT: BAT including ECP, ATG, etanercept, infliximab or RUX will be administered based on Investigator's decision.
Study summary
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).
Eligibility
Sex
ALL
Min age
28 Days
Max age
17 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Number of Participants With Overall Response (OR) — At Day 28 OR is defined as complete response (CR) or partial response (PR) at Day 28 relative to acute graft-versus-host disease (aGvHD) status at baseline. CR is defined as resolution of aGvHD in all involved organs. PR is defined as improvement in 1 stage in at least 1 or more organs involved with aGvHD symptoms without progression in others.
Trial sites (36)
Facility
City
Region
Status
CHU de Bordeaux - Hopital des Enfants
Bordeaux
France
CHU Grenoble Alpes - Hopital Couple Enfant (HCE)
La Tronche
France
Centre Hospitalier Universitaire de Lille CHU Lille - Hopital Jeanne de Flandre HJF
Lille
France
Institut d'Hematologie et d'Oncologie Pediatrique (IHOPe)
Lyon
France
CHU de Marseille-Hopital de la Timone
Marseille
France
Centre Hospitalier Regional Universitaire (CHRU) Montpellier - hopital Arnaud de Villeneuve
Montpellier
France
CHU de Nantes - Hopital Mere Enfant
Nantes
France
Hopital Robert Debre
Paris
France
CHU de Rouen - Hopital Charles Nicolle
Rouen
France
CHRU de Strasbourg - Hopital de Hautepierre
Strasbourg
France
CHRU Nancy, Hopitaux de Brabois
Vandœuvre-lès-Nancy
France
Uniklinik RWTH Aachen, Klinik fur Kinder- und Jugendmedizin
Aachen
Germany
Universitaetsklinikum Essen
Essen
Germany
Klinikum der Johann Wolfgang Goethe
Frankfurt
Germany
Universitaetsklinikum Freiburg - Zentrum fuer Kinder- und Jugendmedizin (ZKJ)
Freiburg im Breisgau
Germany
Justus-Liebig-Universitaet Giessen
Giessen
Germany
Medizinische Hochschule Hannover MHH
Hanover
Germany
Department of Pediatrics, Jena University Hospital
Jena
Germany
Universitaetsklinikum Leipzig - Abteilung fuer Paediatrische Onkologie, Haematologie und Haemostaseologie
Leipzig
Germany
Universitaetsklinikum Muenster (UKM) - Klinik fuer Kinder- und Jugendmedizin - Paediatrische Haematologie und Onkologie
Münster
Germany
IRCCS Azienda Ospedaliero-Universitaria di Bologna Policlinico S.Orsola Malpighi
Bologna
Italy
Pediatric Clinic Onco Hematology San Gerardo Hospital
Monza
Italy
U.O.C. Oncoematologia Pediatrica, Fondazione IRCCS Policlinico San Matteo
Pavia
Italy
Hematology and Cellular Therapy Ospedale Bambino Gesu
Rome
Italy
A.O.U. Citta della Salute e della Scienza di Torino Ospedale Infantile Regina Margherita
Turin
Italy
Department of Pediatric Hematology, Oncology and BMT, Wroclaw Medical University
Wroclaw
Lower Silesian Voivodeship
Dzieciecy Szpital Kliniczny im. A.Gebali w Lublinie
Lublin
Poland
Szpital Kliniczny im. Karola Jonschera UM
Poznan
Poland
Hospital Niño Jesus
Madrid
Madrid
Clinica Universitaria de Navarra
Pamplona
Navarre
Hospital Universitario Vall dHebron
Barcelona
Spain
Hospital Sant Joan de Deu Barcelona (HSJDB)
Barcelona
Spain
Hospital Infantil Universitario La Paz
Madrid
Spain
Instituto de Investigacion Biomedica de Malaga IBIMA - sede Hospital Regional Universitario de Malaga HRUM Hospital Carlos Haya
Málaga
Spain
Instituto Murciano de Investigacion Biosanitaria (IMIB) Virgen de la Arrixaca
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time.