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)

Status: Recruiting
Location: See all (38) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 28 days
Maximum Age: 17
Healthy Volunteers: f
View:

• 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.

• 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.

• 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.

• 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.

• Participant has an estimated life expectancy of \>28 days.

• 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.

• 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.

• 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.

Locations
Other Locations
France
CHU de Bordeaux - Hopital des Enfants
RECRUITING
Bordeaux
CHU Grenoble Alpes - Hopital Couple Enfant (HCE)
RECRUITING
La Tronche
Centre Hospitalier Universitaire de Lille CHU Lille - Hopital Jeanne de Flandre HJF
RECRUITING
Lille
Institut d'Hematologie et d'Oncologie Pediatrique (IHOPe)
RECRUITING
Lyon
CHU de Marseille-Hopital de la Timone
RECRUITING
Marseille
Centre Hospitalier Regional Universitaire (CHRU) Montpellier - hopital Arnaud de Villeneuve
RECRUITING
Montpellier
CHU de Nantes - Hopital Mere Enfant
RECRUITING
Nantes
Hopital Robert Debre
RECRUITING
Paris
CHU Rennes - Hopital Pontchaillou
WITHDRAWN
Rennes
CHU de Rouen - Hopital Charles Nicolle
RECRUITING
Rouen
CHRU de Strasbourg - Hopital de Hautepierre
RECRUITING
Strasbourg
CHRU Nancy, Hopitaux de Brabois
RECRUITING
Vandœuvre-lès-nancy
Germany
Uniklinik RWTH Aachen, Klinik fur Kinder- und Jugendmedizin
RECRUITING
Aachen
Universitatsklinikum Dusseldorf
WITHDRAWN
Düsseldorf
Universitaetsklinikum Essen
RECRUITING
Essen
Klinikum der Johann Wolfgang Goethe
RECRUITING
Frankfurt
Universitaetsklinikum Freiburg - Zentrum fuer Kinder- und Jugendmedizin (ZKJ)
RECRUITING
Freiburg Im Breisgau
Justus-Liebig-Universitaet Giessen
RECRUITING
Giessen
Medizinische Hochschule Hannover MHH
RECRUITING
Hanover
Department of Pediatrics, Jena University Hospital
RECRUITING
Jena
Universitaetsklinikum Leipzig - Abteilung fuer Paediatrische Onkologie, Haematologie und Haemostaseologie
RECRUITING
Leipzig
Universitaetsklinikum Muenster (UKM) - Klinik fuer Kinder- und Jugendmedizin - Paediatrische Haematologie und Onkologie
RECRUITING
Münster
Italy
IRCCS Azienda Ospedaliero-Universitaria di Bologna Policlinico S.Orsola Malpighi
RECRUITING
Bologna
Pediatric Clinic Onco Hematology San Gerardo Hospital
RECRUITING
Monza
U.O.C. Oncoematologia Pediatrica, Fondazione IRCCS Policlinico San Matteo
RECRUITING
Pavia
Hematology and Cellular Therapy Ospedale Bambino Gesu
RECRUITING
Rome
A.O.U. Citta della Salute e della Scienza di Torino Ospedale Infantile Regina Margherita
RECRUITING
Turin
Poland
Dzieciecy Szpital Kliniczny im. A.Gebali w Lublinie
RECRUITING
Lublin
Szpital Kliniczny im. Karola Jonschera UM
RECRUITING
Poznan
Department of Pediatric Hematology, Oncology and BMT, Wroclaw Medical University
RECRUITING
Wroclaw
Spain
Hospital Sant Joan de Deu Barcelona (HSJDB)
RECRUITING
Barcelona
Hospital Universitario Vall dHebron
RECRUITING
Barcelona
Hospital Infantil Universitario La Paz
RECRUITING
Madrid
Hospital Niño Jesus
RECRUITING
Madrid
Instituto de Investigacion Biomedica de Malaga IBIMA - sede Hospital Regional Universitario de Malaga HRUM Hospital Carlos Haya
RECRUITING
Málaga
Instituto Murciano de Investigacion Biosanitaria (IMIB) Virgen de la Arrixaca
RECRUITING
Murcia
Clinica Universitaria de Navarra
RECRUITING
Pamplona
Hospital Universitari I politecnic La Fe Jose
RECRUITING
Valencia
Contact Information
Primary
Medac Clinical Trial Information
ClinicalTrialInformation@medac.de
+49 (0)4103 8006
Time Frame
Start Date: 2023-11-13
Estimated Completion Date: 2031-06
Participants
Target number of participants: 48
Treatments
Experimental: 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.
Active_comparator: 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).
Sponsors
Leads: medac GmbH

This content was sourced from clinicaltrials.gov

Similar Clinical Trials