Early Access Program With MaaT013 in Steroid-refractory Acute Gastrointestinal Graft Versus Host Disease
- Conditions
- Acute Graft Versus Host Disease in IntestineSteroid Refractory GVHD
- Registration Number
- NCT04768907
- Lead Sponsor
- MaaT Pharma
- 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
Recruitment & Eligibility
- Status
- AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
- 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
- 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
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (46)
Chu Rouen
🇫🇷Rouen, France
Institut de Cancerologie de Strabsourg
🇫🇷Strasbourg, France
Chu Angers
🇫🇷Angers, France
Chu Amiens Picardie Site Sud
🇫🇷Amiens, France
Chu Besancon
🇫🇷Besançon, France
Chu Morvan
🇫🇷Brest, France
Chu de Caen
🇫🇷Caen, France
Chu Grenoble
🇫🇷Grenoble, France
Chu de Lille
🇫🇷Lille, France
Chu Limoges
🇫🇷Limoges, France
MaaT Pharma
🇫🇷Lyon, France
Institut Paoli Calmettes
🇫🇷Marseille, France
Chu Montpellier - Hopital Saint Eloi
🇫🇷Montpellier, France
Chru Nancy
🇫🇷Nancy, France
Chu Nantes
🇫🇷Nantes, France
Chu de Nice - L'Archet 1
🇫🇷Nice, France
Aphp - Hopital Sant Antoine
🇫🇷Paris, France
Hopital Haut Leveque
🇫🇷Pessac, France
Aphp - Hopital Necker
🇫🇷Paris, France
Chu Bordeaux - Hopital Haut-Leveque
🇫🇷Pessac, France
Chu Lyon Sud
🇫🇷Pierre-Bénite, France
Chu La Miletrie
🇫🇷Poitiers, France
Chu de Rennes - Hopital Pontchaillou
🇫🇷Rennes, France
Crlcc Henri Becquerel
🇫🇷Rouen, France
Chu St Etienne
🇫🇷Saint-Priest-en-Jarez, France
Chu Strasbourg - Hopital de Hautepierre
🇫🇷Strasbourg, France
Iuct - Oncopole
🇫🇷Toulouse, France
Berlin University Medical Center - Charite Hospital
🇩🇪Berlin, Germany
Saarlan University Medical Center
🇩🇪Homburg, Germany
Az Sint-Jan Brugge Av
🇧🇪Brugge, Belgium
Ordensklinikum Linz Elisabethinen
🇦🇹Linz, Austria
Hub - Institut Jules Bordet
🇧🇪Brussel, Belgium
Foothills Medical Centre
🇨🇦Calgary, Canada
University Clinic - State Hospital of Innsbruck
🇦🇹Innsbruck, Austria
Institut Gustave Roussy
🇫🇷Villejuif, France
University Hospital Essen
🇩🇪Essen, Germany
University Hospital Mannheim
🇩🇪Mannheim, Germany
Ulm University Medical Center
🇩🇪Ulm, Germany
Aou Ospedali Riuniti
🇮🇹Ancona, Italy
Fondazione I.R.C.C.S. Policlinico San Matteo
🇮🇹Pavia, Italy
Gemelli University Hospital
🇮🇹Roma, Italy
Aou Citta Della Salute E Della Scienza
🇮🇹Torino, Italy
Hospital General Universitario Morales Meseguer
🇪🇸Murcia, Spain
Santa Maria Della Misericordia Hospital
🇮🇹Udine, Italy
La Fe University Hospital
🇪🇸Valencia, Spain
University Hospital of Geneva
🇨🇭Geneva, Switzerland