Clinical trial NCT04768907: Early Access Program With MaaT013 in Steroid-refractory Acute Gastrointestinal Graft Versus Host Disease
Study type: EXPANDED_ACCESS
Overall status: AVAILABLE
Conditions: Acute Graft Versus Host Disease in Intestine
Steroid Refractory GVHD
Lead sponsor: MaaT Pharma
Sponsor class: INDUSTRY
First posted: 2021-02-24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT

Brief summary: MaaT013 is still in clinical development phase and is not approved yet for marketing in any region. During the development program, MaaT Pharma has undertaken initial development with closely related product candidates, leading to the Phase II HERACLES study in which MaaT013 preliminary safety and efficacy were assessed in the context of steroid-resistant, gastrointestinal aGraft versus Host Disease (SR-GI-aGvHD). In addition, a pivotal Phase III study (ARES trial) is planned.

In the absence of medical options in patients with gastrointestinal acute GvHD refractory to multiple lines of treatment, this early access program has been implemented.

Detailed description: Acute Graft-versus-Host Disease (aGvHD) is a serious and life-threatening disease that arises as a complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT). At the onset of aGvHD, skin is the most frequently affected region (80% of patients) while GI tract or liver are involved in about 50% of patients (Martin 1990). aGvHD symptoms for the lower GI tract include watery diarrhea (≥500 mL), severe abdominal pain or bloody diarrhea (Ferrara 2009). While the incidence of severe GI-aGvHD has slightly decreased during the past decade, treatment remains unsuccessful in most cases (Gooley 2010, Castilla Llorente 2014), with a 2-months overall survival (OS) rate of 22% in steroid non-responsive or steroid refractory (SR) patients, non-responders to ruxolitinib (Jagasia 2020).

MaaT013 (pooled allogeneic fecal microbiota) is a live biotherapeutic product being developed by MaaT Pharma (Lyon, France) for the treatment of steroid-resistant, gastrointestinal aGvHD (SR-GI-aGvHD) adult patients with refractory, not eligible or who have failed second-line systemic therapy.

MaaT013 is still in clinical development phase and is not approved yet for marketing in any region. During the development program, MaaT Pharma has undertaken initial development with closely related product candidates, leading to the Phase II HERACLES study in which MaaT013 preliminary safety and efficacy were assessed in the context of SR-GI-aGvHD. In addition, a pivotal Phase III study (ARES trial) is planned

Eligibility criteria: Inclusion Criteria:

* Age \> or = 18
* Grade III-IV gastro intestinal acute graft versus host disease with or without involvement of other organs:
* patients resistant to corticosteroid (CS) and resistant to one or multiple lines of treatments
* patients who cannot tolerate CS tapering, i.e., begin of CS at 2.0 mg/kg/d, demonstrate response, but show disease progress before a 50% decrease from the initial starting dose of CS is achieved.
* acute GVHD with overlap syndrome

Exclusion Criteria:

* Active uncontrolled infection
* Relapsed/persistent malignancy requiring rapid immune suppression withdrawal
* Current or past veno-occlusive disease or other uncontrolled complication
* Absolute neutrophil count \<500/µL for 3 consecutive days. Use of growth factor supplementation is allowed
* Absolute platelet count \< 10 000/µL. Use of platelet infusion is allowed
* Current or past evidence of toxic megacolon, bowel obstruction or gastrointestinal perforation
* Known allergy or intolerance to trehalose or maltodextrin
* Pregnancy
* Breastfeeding

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Innsbruck
country: Austria
facility: University Clinic - State Hospital of Innsbruck
city: Linz
country: Austria
facility: Ordensklinikum Linz Elisabethinen
city: Brugge
country: Belgium
facility: Az Sint-Jan Brugge Av
city: Brussel
country: Belgium
facility: Hub - Institut Jules Bordet
city: Calgary
country: Canada
facility: Foothills Medical Centre
city: Amiens
country: France
facility: Chu Amiens Picardie Site Sud
city: Angers
country: France
facility: Chu Angers
city: Besançon
country: France
facility: Chu Besancon
city: Brest
country: France
facility: Chu Morvan
city: Caen
country: France
facility: Chu de Caen
city: Grenoble
country: France
facility: Chu Grenoble
city: Lille
country: France
facility: Chu de Lille
city: Limoges
country: France
facility: Chu Limoges
city: Lyon
country: France
facility: MaaT Pharma
city: Marseille
country: France
facility: Institut Paoli Calmettes
city: Montpellier
country: France
facility: Chu Montpellier - Hopital Saint Eloi
city: Nancy
country: France
facility: Chru Nancy
city: Nantes
country: France
facility: Chu Nantes
city: Nice
country: France
facility: Chu de Nice - L'Archet 1
city: Paris
country: France
facility: Aphp - Hopital Sant Antoine
city: Paris
country: France
facility: Aphp - Hopital Necker
city: Pessac
country: France
facility: Chu Bordeaux - Hopital Haut-Leveque
city: Pessac
country: France
facility: Hopital Haut Leveque
city: Pierre-Bénite
country: France
facility: Chu Lyon Sud
city: Poitiers
country: France
facility: Chu La Miletrie
city: Rennes
country: France
facility: Chu de Rennes - Hopital Pontchaillou
city: Rouen
country: France
facility: Chu Rouen
city: Rouen
country: France
facility: Crlcc Henri Becquerel
city: Saint-Priest-en-Jarez
country: France
facility: Chu St Etienne
city: Strasbourg
country: France
facility: Chu Strasbourg - Hopital de Hautepierre
city: Strasbourg
country: France
facility: Institut de Cancerologie de Strabsourg
city: Toulouse
country: France
facility: Iuct - Oncopole
city: Villejuif
country: France
facility: Institut Gustave Roussy
city: Berlin
country: Germany
facility: Berlin University Medical Center - Charite Hospital
city: Essen
country: Germany
facility: University Hospital Essen
city: Homburg
country: Germany
facility: Saarlan University Medical Center
city: Mannheim
country: Germany
facility: University Hospital Mannheim
city: Ulm
country: Germany
facility: Ulm University Medical Center
city: Ancona
country: Italy
facility: Aou Ospedali Riuniti
city: Pavia
country: Italy
facility: Fondazione I.R.C.C.S. Policlinico San Matteo
city: Roma
country: Italy
facility: Gemelli University Hospital
city: Torino
country: Italy
facility: Aou Citta Della Salute E Della Scienza
city: Udine
country: Italy
facility: Santa Maria Della Misericordia Hospital
city: Murcia
country: Spain
facility: Hospital General Universitario Morales Meseguer
city: Valencia
country: Spain
facility: La Fe University Hospital
city: Geneva
country: Switzerland
facility: University Hospital of Geneva
Study officials:
References: