Etude : IDUNN / MC-MSC.1/aGvHD



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Acronyme / Nom
Situation thérapeutique
Traitement
Cadre réglementaire
MÀJ
Présentation de l'étude
Acronyme / Nom : IDUNN / MC-MSC.1/aGvHD

Situation thérapeutique : GvH

Traitement :

Cadre réglementaire : RIPH1

Dernière MÀJ : 29/11/2021
Titre
Spécialité(s)
CIM10 - Localisation(s)
Informations principales
Titre : Étude de phase III randomisée, multicentrique, en ouvert portant sur le traitement de première intention par cellules stromales mésenchymateuses MC0518 par rapport au meilleur traitement disponible chez des patients adultes ou adolescents présentant la forme aiguë de la maladie du greffon contre l'hôte réfractaire aux stéroïdes, suite à une greffe de cellules souches hématopoïétiques allogéniques (étude IDUNN)

Spécialité : Tissus lymphoïde, hématopoïétique et apparentés
Localisation : C96 - Tumeurs malignes des tissus lymphoïde, hématopoïétique et apparentés, autres et non précisées
Schéma
Phase
Stade
Ligne(s)
Informations complémentaires
Schéma : 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) in adult and adolescent participants with steroid-refractory acute graft-versus-host disease (SR-aGvHD) at Day 28.

STUDY ARMS:
- Experimental: 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.

- Active Comparator: 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).


MAIN OBJECTIVES:
The primary objective of this trial is to demonstrate the superiority of MC0518 compared to the first used Best Available Therapy (BAT) with respect to ORR in adult and adolescent subjects with SR aGvHD at Visit Day 28.

SECONDARY OBJECTIVES:
- Demonstrate the superiority of MC0518 compared with the first used BAT with respect to freedom from treatment failure (FFTF) as defined by the absence of death, malignancy relapse or progression, or addition or change to any further systemic aGvHD immunosuppressive therapy within 6 months of the intervention from the date of randomisation up to the date of the event and before the diagnosis of chronic graft versus host disease (cGvHD)
- Demonstrate the superiority of MC0518 compared with the first used BAT with respect to overall survival (OS) until Visit Month 24
- Compare the efficacy of MC0518 and BAT in further secondary long-term efficacy endpoints and response to treatment
- Investigate the safety of MC0518
- Compare health related quality of life (HRQoL) when treated with MC0518 compared with BAT

Phase : III

Stade : NA

Réfractaire
Informations libres de droit
Critères d'inclusion
Critères de non-inclusion
Informations libres de droit
Critères d'inclusion et de non-inclusion
Critères d'inclusion : - Subject had a previous allogeneic HSCT as indicated for malignant or non-malignant haematological disease, irrespective of human leukocyte antigen match.
- Subject has been clinically diagnosed with Grade II to IV aGvHD at the Screening Visit.
- Subject 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.
- Male or female subject who is ≥ 12 years of age at the Screening Visit.
- Subject has an estimated life expectancy > 28 days at the Screening Visit.

Critères de non-inclusion : - Subject has overt relapse or progression or persistence of the underlying disease at the Screening Visit.
- Subject has received the last HSCT for a solid tumour disease.
- Subject has GvHD overlap syndrome at the Screening Visit.
- Subject has received systemic first-line treatment for aGvHD other than steroids and a prophylaxis with other than calcineurin inhibitors, ATG, MMF, MTX, and or cyclophosphamide before the Screening Visit.
- Subject has a known pregnancy (as confirmed by a positive pregnancy test at the Screening Visit) and or is breastfeeding at the Screening Visit.
- Subject 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).
NCT
Promoteur
Coordonnateur
Informations relatives au promoteur
NCT :
NCT04629833
Promoteur :
Medac GmbH
Type de sponsor : Industriel
Wedel, Allemagne - Wedel, Allemagne
00000 HORS FRANCE

Coordonnateur :
Centre investigateur
Investigateur
TEC / ARC / IDE
État
MÀJ
Informations relatives aux investigateurs
Centre investigateur :
Centre Hospitalier Universitaire de Lille - 2 Avenue Oscar Lambret - 59000 LILLE

Investigateur :
Professeur Ibrahim YAKOUB-AGHA

TEC / ARC / IDE :
Secrétariat de recherche
fanny.miquel@
chru-lille.fr
03.20.44.57.13

Statut de l'essai : À VENIR

MAJ : 29/11/2021