A medical research to evaluate if MaaT013 is safe and helps children and adolescent with GI-aGvHD after failed two prior therapies.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

MaaT PHARMA

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Decision date (initial)

2026-04-17

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety

Co- Primary: Safety and tolerability of MaaT013
Feasibility of MaaT013 administration in paediatric population

Secondary objectives 10

1. ORR for GI-aGvHD at D28, D56, Month 3 (M3), M6 and M12 assessed by IRC and investigator
2. ORR for all organs at D28, D56, M3, M6 and M12 assessed by IRC and investigator
3. Duration of response (DOR) assessed by IRC and investigator
4. Overall survival (OS)
5. Progression-free survival (PFS) assessed by investigator
6. Time to progression (TTP) assessed by investigator
7. Steroid-free rate (SFR).
8. Cumulative steroid dose
9. The frequency of participants that tapered off CS
10. Frequency of cGvHD

Conditions and MedDRA coding

Gastrointestinal Acute Graft-versus-Host Disease (GI-aGvHD)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-003435-PIP02-23

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

1. 1. Age ≥ 6 years and < 18 years old at the time point of informed consent.
2. 10. Negative urine or serum pregnancy test for females of childbearing potential within 72 hours prior to receiving the first dose of MaaT013 treatment.
3. 2. Karnofsky/Lansky performance status ≥ 40 at informed consent.
4. 3. Weight ≥ 15 kg.
5. 4. Allo-HSCT with any type of donor, stem cell source, GvHD prophylaxis or conditioning regimen.
6. 5. Acute GvHD episode with GI involvement per MAGIC guidelines (= grade II to IV), with or without involvement of other organs
7. 6. Adequate organ function, defined as: o Absolute neutrophil count (ANC) >500/µl, confirmed within 3 days prior to pre-treatment start. Use of growth factor supplementation to support the ANC count is allowed. o Platelet count > 10.000/µl, confirmed within 3 days prior to pre-treatment start. Use of platelet transfusion to support the platelet count is allowed.
8. 7. Resistance to steroids and ruxolitinib. Resistance to steroids is defined as participants administered high-dose systemic CS (methylprednisolone 2 mg/kg/day – or equivalent prednisone dose 2.5 mg/kg/day), given alone or combined with CNI or mechanistic Target of Rapamycin (mTOR) inhibitor and either: o Lack of improvement (i.e., no decrease in stage in at least 1 involved organ system) after ≥ 5 days of treatment with CS at 2 mg/kg/d methylprednisolone equivalent dose, OR o Progression (i.e., increase of GvHD in any organ system or any new organ involvement) after ≥ 3 days of treatment with CS at 2 mg/kg/d methylprednisolone equivalent dose, OR o Participants treated with 1 mg/kg/d of CS as deemed intolerant by investigator to a dose of 2 mg/kg/d, and who correspond to the definition of SR participants, OR o Participants 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, OR o Participants intolerant to CS tapering, i.e., begin of CS at 2.0 mg/kg/d, demonstrate response, but show disease progression before a 50% decrease from the initial starting dose of CS is achieved. Resistance to ruxolitinib is defined as any of the following (adapted from (Mohty et al. 2020)): o 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; or a lack of improvement for patients already at the highest stage or grade. OR o Lack of improvement in GvHD (PR or better) compared to baseline after at least 14 days of treatment with ruxolitinib, OR o 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, OR o Absence of CR or VGPR at D28 after ruxolitinib start. Intolerance to ruxolitinib is defined as: o GvHD manifestations that persist without improvement in participants who had grade 3 or higher ruxolitinib-emergent and ruxolitinib-attributed AE that did not resolve within 7 days of discontinuing ruxolitinib.
9. 8. Allo-HSCT participants with a non-malignant underlying disease may be enrolled only after their eligibility has been reviewed and approved by a multidisciplinary clinical board at clinical site.
10. 9. Signature of informed and written consent / assent by the participants legal representative.
11. 11. Use of an acceptable effective method of birth control for the course of the study for female of childbearing potential/sexually active male with partner of childbearing potential. This includes, but is not limited to: a) progestogen-only oral hormonal contraception b) male or female condom with or without spermicide c) cap, diaphragm or sponge with spermicide d) A combination of male condom with either cap, diaphragm or sponge with spermicide (double barrier methods) are also considered acceptable birth control methods

Exclusion criteria 18

1. 1. Presence of fungal, bacterial, viral, or other infection that is uncontrolled or requiring antimicrobials for management. Isolated urinary tract infection and uncomplicated bacterial pharyngitis are permitted if responding to active treatment.
2. 10. Clinically significant respiratory disease that requires mechanical ventilation support or oxygen support with a fraction of inspired oxygen at 50%.
3. 11. Current or past (<6 months) evidence of toxic megacolon, bowel obstruction or GI perforation
4. 12. 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 participant; or interfere with interpretation of study data.
5. 13. Known allergy or intolerance to trehalose or maltodextrin.
6. 14. Breastfeeding females.
7. 15. Participant having received any previous microbiome product (FMT). Note: Participants with medical history of probiotic intake are eligible.
8. 16. Other ongoing interventional protocol that might interfere with the current study’s primary and secondary endpoints.
9. 17. In the investigator’s judgement, the participant is unlikely to complete all protocol-requiring trial visits or procedures, including FU visits, or comply with the trial requirements for participation.
10. 2. Any other diseases, metabolic dysfunction, physical examination findings, or clinical laboratory abnormalities giving reasonable suspicion of a disease or condition that in the opinion of the investigator would contraindicate study treatment.
11. 3. Clinical presentation resembling de novo cGvHD or GvHD overlap syndrome with both acute and chronic GvHD features
12. 4. Known hypersensitivity to vancomycin or to any of the excipients listed in the corresponding SmPC.
13. 5. Active cytomegalovirus (CMV) colitis.
14. 6. Relapsed/persistent malignancy requiring rapid immune suppression withdrawal.
15. 7. Previous lines of systemic aGvHD treatment other than CS and ruxolitinib.
16. 8. Severe organ dysfunction or other uncontrolled complication, unrelated to underlying GvHD, including: cholestatic disorders or unresolved veno-occlusive disease (VOD) of the liver (defined as persistent bilirubin abnormalities not attributable to GvHD and ongoing organ dysfunction, with the exception of Gilbert syndrome).
17. 9. 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.
18. 18. For France only: Children who are, or who are expected to become, adults under legal protection (tutelle, curatelle, or other court‑ordered protection), children whose parents are not affiliated with a French social security scheme and who therefore cannot be enrolled as dependents (‘ayants droit’), or children who would not be eligible for health coverage under the Protection Universelle Maladie (PUMA) upon reaching 18 years of age.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 7

1. Co-primary: From inclusion to Month 6 (M6): Incidence of all AEs treatment-emergent AEs (TEAEs), serious AEs (SAEs), and assessment of all safety parameters (physical examinations, vital signs and laboratory clinically significant abnormalities).
2. Co-Primary: From M6 to M12, incidence of SAEs and AESIs only.
3. Co-Primary: A comprehensive and detailed analysis of the nature, severity and frequency of AEs and SAEs will be performed.
4. Co-Primary: 1) Retention time ▪ Proportion of participants able to retain MaaT013 for at least 30 min. ▪ Proportion of participants able to retain MaaT013 for at least 1h. ▪ Proportion of participants able to retain MaaT013 for at least 2h.
5. Co-Primary: 2) Stress/anxiety evaluated with the depression anxiety stress scale
6. Co-Primary: 3) Procedure-related AEs ▪ Solicited TEAEs related to administration procedure will be collected within 72h after each MaaT013 administration
7. Co-Primary: 4) Factors compromising the administration of the study drug ▪ TEAEs leading to treatment discontinuation, interruption and postponement will be collected.

Secondary endpoints 10

1. Proportion of participants achieving complete response (CR), very good partial response (VGPR) or partial response (PR) for GI at D28, D56, M3, M6 and M12 assessed by IRC and investigator, without requirement for additional systemic therapies prior to the assessment time point.
2. Proportion of participants achieving CR, VGPR or PR for all organs at D28, D56, M3, M6 and M12 assessed by IRC and investigator, without requirement for additional systemic therapies prior to the assessment time point.
3. DOR is assessed for responders only and is defined as the time from first response (at least PR) until aGvHD progression (loss of at least PR compared to baseline, confirmed with 2 evaluations), or the starting date of additional systemic therapies for aGvHD. Onset of cGvHD, or death without prior observation of aGvHD progression are considered as competing risks. DOR will be evaluated for both GI and overall aGvHD, until M12.
4. OS is defined as the time from the date of first MaaT013 administration to the date of death due to any cause.
5. PFS is defined as the time from first MaaT013 administration to the date of underlying malignancy relapse, progression or death (all causes). PFS will be evaluated up to M12.
6. TTP is defined as the time from first MaaT013 administration to the date of underlying malignancy relapse or progression. TTP will be evaluated up to M12.
7. SFR is defined as the number and percentage of participants who are steroid-free, defined by a daily dose of CS ≤ 0.25 mg/kg/day (methylprednisolone equivalent dose). Steroid-free rate at D28, D56, M3, M6, and M12 will be provided. SFR will be evaluated up to M12.
8. Cumulative total dose of CS in mg/kg from date of first MaaT013 administration to D28, D56, M3, M6, and M12 will be derived and summarized.
9. Number and percentage of participants who definitively tapered off CS at M12.
10. Proportion of participants with cGvHD, defined as the diagnosis of any cGvHD, including mild, moderate or severe, up to M12 (NIH criteria).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

MaaT PHARMA

Sponsor organisation

MaaT PHARMA

Address

70 Avenue Tony Garnier

City

Lyon

Postcode

69007

Country

France

Scientific contact point

Organisation

MaaT PHARMA

Contact name

Maat-Pharma Contact Point

Public contact point

Organisation

MaaT PHARMA

Contact name

Maat-Pharma Contact Point

Third parties 7

Locations

4 EU/EEA countries · 12 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 61 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications