Clinical trial NCT05017688: Prospective Interventional Study Exploring the Microbiota Recolonization in SR-GvHD Patients Receiving MaaT013
Official title: PrOspective inteRventional Study Exploring the mIcrobiota recolONization in Steroid-Refractory Graft-versus-Host Disease Patients Receiving MaaT013
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Intestinal GVHD
Steroid Refractory GVHD
Keywords: gut microbiota
fecal microbiotherapy
intestinal GvHD
Lead sponsor: MaaT Pharma
Sponsor class: INDUSTRY
First posted: 2021-08-24
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Prospective interventional with low risks and constraints study (RIPH2)
Primary purpose: OTHER

Brief summary: The objective of the ORION study is to explore the changes of gut microbiota composition following MaaT013 administration and its impact on the immune system in GVHD patients.

Detailed description: The french regulatory authority ANSM approved in July 2019 the use of MaaT013, a pooled microbiome-based enema formulation, under a formalized named-patient use program in France called "Autorisation Temporaire d'Utilisation - ATU nominative protocolisée - ATUn" or Early Access.

The ATUn is indicated to provide a benefit in the treatment of patients with grade III-IV aGVHD:

* In case of initial resistance to CS alone or in association or in case of failure to other treaments
* In first-line therapy in association with CS in case of steroid-dependance (inability to taper CS dose \<0.5 mg/kg/d)
* In case of digestive aGvHD with overlap syndrome The objective of the ORION study is to explore the changes of gut microbiota composition following MaaT013 administration and its impact on the immune system in GVHD patients.

Blood and stool samples will be collected at each visit which explained why the study is thus categorized as research involving the human person with low risks and constraints clinical trial (RIPH2).

Eligibility criteria: Inclusion Criteria:

* Patient ≥18 years old with Grade III-IV aGVHD with gut involvement undergoing treatment with MaaT013 through named-patient use program ATUn / Early Access
* Signature of informed and written consent by the subject or by the subject's legally acceptable representative for patients under guardianship or trusteeship.
* Affiliated or recipient from a social security scheme

Exclusion Criteria:

* Pregnancy and breastfeeding
* Vulnerable patients such as: minors, persons deprived of liberty, persons in Intensive Care Unit unable to provide informed consent prior to the intervention.

Primary outcomes:
measure: Change of gut microbiota diversity
timeFrame: inclusion (day 0) to month 6
measure: Change of gut microbiota composition
timeFrame: inclusion (day 0) to month 6
measure: Change of gut microbiota composition (phylogenetic profiles)
timeFrame: inclusion (day 0) to month 6
Secondary outcomes:
measure: Correlation between gut microbiota and immune parameters
timeFrame: inclusion (day 0) to month 6
measure: Gut microbiota baseline characterization based on clinical response
timeFrame: day 0
measure: Change in immune parameters following MaaT013 administration
timeFrame: inclusion (day 0) to month 6
Other outcomes:
Locations:
city: Amiens
country: France
facility: Chu Amiens Picardie Site Sud
city: Caen
country: France
facility: Chu de Caen
city: Grenoble
country: France
facility: Chu Grenoble
city: Nice
country: France
facility: Chu de Nice - L'Archet 1
city: Paris
country: France
facility: Aphp - Hopital Saint Antoine
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: Toulouse
country: France
facility: Institut Universitaire Du Cancer de Toulouse - Oncopole
Study officials:
affiliation: IUCT ONCOPOLE
name: Sarah Guenounou, MD
role: PRINCIPAL_INVESTIGATOR
References: