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Recruiting NCT04629833

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

Phase III Interventional Steroid-refractory Acute Graft-versus-host Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: MC0518, BAT.
Who it may be relevant to
Registry conditions: Steroid-refractory Acute Graft-versus-host Disease. Basic parameters: from 12 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
France, Germany, Poland, Spain, Sweden
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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)

Overview

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).

Interventions

  • Biological MC0518
    MC0518 will be intravenously infused immediately after thawing.
  • Biological BAT
    BAT including MMF, ECP, ATG, everolimus, and RUX will be administered based on Investigator's decision.

Primary outcome measures

  • Overall Response (OR) [Time frame: Day 28]
  • Overall Survival [Time frame: Up to Month 24]
Secondary outcome measures (12)
  • 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]

Eligibility criteria

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).

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

Germany · 22 centers
  • Klinikum rechts der Isar — Munich
  • Universitaetsklinikum Wuerzburg - Medizinische Klinik und Poliklinik II - Zentrum fuer All — Würzburg
  • Klinikum der Johann Wolfgang Goethe-Universitaet - Frankfurt am Main — Frankfurt am Main
  • Medizinische Hochschule Hannover — Hanover
  • Uniklinik Koeln — Cologne
  • Universitaetsklinikum Essen - Klinik fuer Knochenmarktransplantation (KMT) — Essen
  • Universitaetsklinikum Muenster — Münster
  • Universitaetsklinikum Carl Gustav Carus Dresden — Dresden
  • … and 14 more centers
France · 9 centers
  • Centre Hospitalier Universitaire (CHU) Amiens-Picardie - Hopital Sud — Amiens
  • CHU Jean Minjoz — Besançon
  • Hopital Michallon — Grenoble
  • CHRU Lille- Hopital Claude Huriez — Lille
  • CHU de Nantes - Hotel Dieu — Nantes
  • CHU de Nice Hopital Archet 1 — Nice
  • Centre Hospitalier Lyon Sud Pavillon Marcel Berard 1G — Pierre-Bénite
  • Centre Hospitalier Universitaire CHU de Toulouse — Toulouse
  • … and 1 more center
Spain · 8 centers
  • Hospital Germans Trias i Pujol — Badalona
  • Hospital Universitario Vall dHebron — Barcelona
  • Institut Catal dOncologia — Barcelona
  • Hospital Universitario Ramon y Cajal — Madrid
  • Hospital Puerta De Hierro — Madrid
  • Hospital Universitario Carlos Haya — Málaga
  • Hospital Clinico Universitario de Valencia — Valencia
  • Hospital Universitari i politecnic La Fe — Valencia
Poland · 2 centers
  • Department of Pediatric Hematology, Oncology and BMT, Wroclaw Medical University — Wroclaw
  • Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie, Paastwowy Instytut Badawczy — Gliwice
Sweden · 1 center
  • Center for Allogeneic Stem Cell Transplantation and Cell Therapy (CAST), Karolinska Univer — Huddinge

Identifiers

NCT: NCT04629833 · MC-MSC.1/aGvHD

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗