Early Access Program With MaaT013 in Steroid-refractory Acute Gastrointestinal Graft Versus Host Disease

Launched by MAAT PHARMA · Feb 22, 2021

Keywords

ClinConnect Summary

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 c...

Gender

ALL

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