Acute Graft Versus Host Disease in IntestineSteroid Refractory GVHD
Investigational drug(s) / intervention(s)
MaaT013
MaaT013: MaaT013 is made of allogeneic, full-ecosystem pooled biotherapeutic intestinal microbiota
Study summary
MaaT013 showed interesting results in steroids and ruxolitinib-resistant aGVHD patients with gut involvement (55% ORR at D28) and 47% and 39% OS at 6 and 12 months respectively (Malard 2020), therefore warrant being tested as salvage therapy in steroid and JAK inhibitors-resistant GI-aGvHD patients. Given the absence of an approved 3rd line strategy or 2nd line strategy in ruxolitinib intolerant patients and the extremely poor prognosis of these patients, who are mostly left with no viable therapeutic option, a single-arm open-label design was proposed.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Age ≥ 18 years old
* Allo-HSCT with any type of donor, stem cell source, GVHD prophylaxis or conditioning regimen.
* Acute GvHD episode with GI involvement per MAGIC guidelines (= grades II to IV), with or without involvement of other organs
* Patients resistant to steroids AND either resistant to OR with intolerance to ruxolitinib OR with contra-indication to ruxolitinib:
Exclusion Criteria:
* Patients with known hypersensitivity to vancomycin or to any of the excipients listed in the corresponding SmPC
* Patients with active CMV colitis
* Patients who had previously received other lines of systemic aGvHD treatment other than CS and ruxolitinib.
* Grade II-IV hyper-acute GvHD
* Overlap chronic GvHD
* Relapsed/persistent malignancy requiring rapid immune suppression withdrawal.
* Active uncontrolled infection according to the attending physician
* Severe organ dysfunction unrelated to underlying GvHD, including:
Cholestatic disorders or unresolved veno-occlusive disease of the liver (defined as persistent bilirubin abnormalities not attributable to GvHD and ongoing organ dysfunction).
Clinically significant or uncontrolled cardiac disease including unstable angina, acute myocardial infarction within 6 months before Day 1 of study drug administration, New York Heart Association Class III or IV congestive heart failure, circulatory collapse requiring vasopressor or inotropic support, or arrhythmia that requires therapy.
Clinically significant respiratory disease that requires mechanical ventilation support or 50% oxygen.
* Current or past veno-occlusive disease or other uncontrolled complication unless otherwise agreed in writing by the sponsor.
* 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.
* Patient with negative IgG EBV serology.
* Current or past evidence of toxic megacolon, bowel obstruction or gastrointestinal perforation.
* Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits; pose a significant risk to the subject; or interfere with interpretation of study data.
* Known allergy or intolerance to trehalose or maltodextrin.
* Vulnerable patients such as: minors, persons deprived of liberty, persons in Intensive Care Unit unable to provide informed consent prior to the intervention.
* Females of childbearing potential should have a negative urine or serum pregnancy test within 72 hours prior to receiving the first dose of study medication. Females of childbearing potential should be willing to use 2 methods of birth control or be surgically sterile or abstain from procreative sexual activity for the course of the study. Females of childbearing potential are those who have not been surgically sterilized or have not been free from menses for \>1 year. Males should agree to abstain from procreative sexual activity starting with the first dose of study therapy through the end of the study.
* Other ongoing interventional protocol that might interfere with the current study's primary endpoint.
Primary outcome measure(s)
ORR of gastro intestinal-aGvHD — Day 28 Overall Response Rate (Complete Response + Very Good Partial Response + Partial Response)
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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