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    Summary
    EudraCT Number:2021-001841-11
    Sponsor's Protocol Code Number:MPOH06
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-06-17
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2021-001841-11
    A.3Full title of the trial
    Evaluation of the efficacy of MaaT013 as salvage therapy in acute GVHD patients with gastrointestinal involvement, refractory to ruxolitinib; a multi-center open-label phase III trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of the efficacy of MaaT013 as salvage therapy in acute GVHD patients with gastrointestinal involvement, refractory to ruxolitinib; a multi-center open-label phase III trial
    A.4.1Sponsor's protocol code numberMPOH06
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04769895
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorMaaT Pharma
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportMaaT Pharma
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMaat Pharma
    B.5.2Functional name of contact pointMélanie Tilte
    B.5.3 Address:
    B.5.3.1Street Address70 avenue Tony Garnier
    B.5.3.2Town/ cityLyon
    B.5.3.3Post code69007
    B.5.3.4CountryFrance
    B.5.6E-mailclinical@maat-pharma.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2083
    D.3 Description of the IMP
    D.3.1Product nameMaaT013
    D.3.4Pharmaceutical form Rectal suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPRectal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeMaaT013
    D.3.9.3Other descriptive nameAllogeneic pooled inoculum
    D.3.9.4EV Substance CodeSUB184464
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Salvage therapy of acute GVHD patients with gastrointestinal involvement, refractory to ruxolitinib.
    E.1.1.1Medical condition in easily understood language
    Treatment of acute graft-versus host disease (aGvHD) to the gastrointestinal (GI) tract of patients who are resistant or intolerant to ruxolitinib.
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10066264
    E.1.2Term Acute graft versus host disease in intestine
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluation of the Overall Response Rate (ORR: Complete Response +Very Good Partial Response + Partial Response) of gastrointestinal- acute graft-versus-host disease (aGvHD) at Day 28.
    E.2.2Secondary objectives of the trial
    •To evaluate MaaT013 safety
    •To evaluate ORR for GI at D56
    •To evaluate ORR for all organs at D28 and D56
    •To evaluate the best ORR (for GI and all organs) achieved between D0 and D28
    •To assess duration of response
    •To assess overall survival (OS)
    •To assess progression-free survival (PFS)
    •To assess time to progression
    •To assess steroid-free survival
    •To evaluate the frequency of patients that tapered off CS
    •To measure the incidence of chronic GvHD
    •To evaluate changes in patient reported outcomes (PROs)
    •To evaluate MaaT013 activity on immune markers
    E.2.3Trial contains a sub-study No
    E.3Principal 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 (Harris, Young, et al. 2016).
    •Patients resistant to steroids AND either resistant to OR with intolerance to ruxolitinib (intolerant patients: who had grade 3 or higher treatment-emergent and ruxolitinib-attributed adverse event that did not resolve within 7 days of discontinuing ruxolitinib):
    Resistance to steroids is defined as any of the following:
    Lack of improvement (i.e., no decrease in stage in at least 1 involved organ system) after ≥ 5 days of treatment with CS at 2mg/kg/d methylprednisolone equivalent dose,
    Progression (i.e., increase in any organ system or any new organ involvement) after ≥ 3 days of treatment with CS at 2 mg/kg/d methylprednisolone equivalent dose,
    Patients treated with 1 mg/kg/d of CS because the physician deemed they would not tolerate 2 mg/kg/d and who correspond to the definition of SR patients,
    Patients who previously began CS therapy at a lower dose (at least 1 mg/kg/d methylprednisolone equivalent) for skin or upper GI GvHD but develop new GVHD in another organ system,
    Patients who cannot tolerate corticosteroid 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.

    Resistance to ruxolitinib is defined as any of the following (Mohty et al. 2020):
    Progression of GvHD compared to baseline after at least 5 days of treatment with ruxolitinib, based either on objective increase in stage/grade, or new organ involvement;
    Lack of improvement in GvHD (partial response or better) compared to baseline after at least 14 days of treatment with ruxolitinib;
    Loss of response, defined as objective worsening of GvHD determined by increase in stage, grade or new organ involvement at any time after initial improvement
    Absence of complete response or very good partial response at day 28 after ruxolitinib

    Intolerance to ruxolitinib is defined as:
    GvHD manifestations that persist without improvement in patients who had grade 3 or higher treatment-emergent and ruxolitinib-attributed adverse event that did not resolve within 7 days of discontinuing ruxolitinib
    •Signature of informed and written consent by the subject or by the subject’s legally acceptable representative for patients under guardianship or trusteeship.
    E.4Principal 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 as defined by the MD Anderson’s criteria (Saliba et al. 2007) (aGvHD onset within 14 days after allo-HSCT)
    •Overlap chronic GvHD as defined by the NIH Consensus Criteria (Jagasia et al. 2015)
    •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, with the exception of Gilbert syndrome).
    -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 that requires therapy.
    -Clinically significant respiratory disease that requires mechanical ventilation support or 50% oxygen.
    •Current or past (< 6 months) veno-occlusive disease or other uncontrolled complication unless otherwise agreed in writing by the sponsor.
    •Absolute neutrophil count <500/µL, confirmed within 3 days prior to pre-treatment start. Use of growth factor supplementation is allowed.
    •Absolute platelet count < 10 000/µL, confirmed within 3 days prior to pre-treatment start. Use of platelet infusion is allowed.
    •Patient with negative IgG EBV serology.
    •Current or past (< 6 months) 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.
    •Breastfeeding females. Females of childbearing potential should be willing to use an acceptable method of birth control such as hormonal contraception (progestogen-only may be sufficient), male or female condom with or without spermicide, cap, diaphragm or sponge with spermicide for the course of the study. A combination of male condom with either cap, diaphragm or sponge with spermicide (double barrier methods) are also considered acceptable. Females of childbearing potential are those who have not been surgically sterilized or have not been free from menses for >1 year.
    •Other ongoing interventional protocol that might interfere with the current study’s primary endpoint.
    E.5 End points
    E.5.1Primary end point(s)
    To evaluate the efficacy of MaaT013 assessed by the overall response rate (ORR) of gastrointestinal (GI) acute graft-versus-host disease (aGVHD) response at day 28 (D28)
    E.5.1.1Timepoint(s) of evaluation of this end point
    ORR at D28 after first dosing
    The patient fails to achieve at least GI-PR at D28
    The patient stops treatment sequence and receives salvage aGvHD therapy before D28
    The patient dies before D28
    E.5.2Secondary end point(s)
    To evaluate MaaT013 safety
    To evaluate ORR for GI at D56 and M3
    To evaluate ORR for all organs at D28, D56 and M3
    To evaluate the best ORR (for GI and all organs) achieved between D0 and D28
    To assess duration of response
    To assess overall survival (OS)
    To assess progression-free survival (PFS)
    To assess time to progression
    To assess steroid-free survival
    To evaluate the frequency of patients that tapered off CS
    To measure the incidence of chronic GvHD
    To evaluate changes in patient reported outcomes (PROs)


    E.5.2.1Timepoint(s) of evaluation of this end point
    From inclusion to Month 6
    From Month 6 to Month 12
    Proportion of patients achieving CR, VGPR and PR of GI symptoms at D56 and M3
    Proportion of patients achieving CR, VGPR and PR for all organs at D28, D56 and M3
    at any timepoint up to and including D28 and before the start of any salvage therapy for aGVHD
    the time from first response (at least PR) until aGvHD progression or the starting date of additional systemic therapies for aGvHD
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA50
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months8
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 25
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 75
    F.4.2.2In the whole clinical trial 75
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-07-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-09-15
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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