Prospective Randomized Controlled Study: Amimaitosai Injection Combined With Corticosteroids for Gastrointestinal aGVHD

NCT07803978 — Early Phase 1, Enrolling by invitation, 60 participants, Chinese PLA General Hospital

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Aprospective, Randomized, Controlled Study of Amimaitosai Injection Combined With Corticosteroids for the Treatment Ofgastrointestinal Acute Graft-versus-host Disease

Registry	NCT07803978
Status	Enrolling by invitation
Phase	Early Phase 1
Study type	Interventional; randomized, parallel, masking: none, treatment
Enrollment	60 (Estimated)
Sponsor	Chinese PLA General Hospital (Other)
Conditions	Acute Gastrointestinal Graft-versus-host Disease
Interventions	Amimaitosai injection, Methylprednisolone (Drug)
Dates	start 2026-05-01, primary completion 2029-05-01 (estimated), completion 2029-05-01 (estimated), first posted 2026-09-04, last updated 2026-09-04
Locations	1 site in China

-Summary

This study focuses on patients with gastrointestinal-dominant acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplantation. AmiMetosai Injection combined with corticosteroids is administered as first-line treatment. The objective is to evaluate the efficacy and safety of AmiMetosai Injection combined with corticosteroids for the treatment of gastrointestinal aGVHD.

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+Detailed description

This study focuses on patients with newly developed gastrointestinal-predominant aGVHD after allogeneic hematopoietic stem cell transplantation. The experimental group receives methylprednisolone 1 mg/kg/d combined with AmiMetosai Injection (1×10⁶ cells/kg, twice weekly for 4 weeks; extended to 8 weeks for partial responders). The control group receives methylprednisolone 2 mg/kg/d monotherapy. The primary endpoint is the Day 28 overall response rate. Secondary endpoints include GVHD-free relapse-free survival and cumulative incidence of relapse. Safety outcomes include adverse events, grade 3-4 toxicities, and abnormal laboratory parameters. Previous studies have reported that mesenchymal stromal cells are safe and effective for steroid-refractory gastrointestinal aGVHD. In this study, the combination is administered with the aim of improving treatment response and reducing corticosteroid-related toxicity.

+Arms and interventions

Arm	Type	Interventions	Description
Low-dose methylprednisolone combined with AmiMetosai	Experimental	Drug: Amimaitosai injection; Drug: Methylprednisolone	Patients in the experimental group receive low-dose methylprednisolone at a daily dose of 1 mg/kg combined with AmiMetosai Injection. AmiMetosai Injection is administered at a cell dose of 1×10⁶ cells/kg per infusion, twice weekly for an initial 4-week treatment course. Patients with partial response after 4 weeks may receive an additional 4-week extended treatment. Patients with complete response, no response or progressive disease will discontinue AmiMetosai Injection treatment ahead of schedule.
Methylprednisolone monotherapy	Active comparator	Drug: Methylprednisolone	Participants in the control group receive standard first-line treatment with methylprednisolone at a daily dose of 2 mg/kg. No UC-MSC intervention or other additional immunomodulatory agents will be administered during the trial treatment period.

Amimaitosai injection (Drug): Amimaitosai injection (1×10⁶ cells/kg per infusion, twice weekly for 4 weeks; may extend to 8 weeks for partial responders).
Methylprednisolone (Drug): Experimental Arm：Methylprednisolone at a daily dose of 1 mg/kg. Active Comparator Arm：Methylprednisolone at a daily dose of 2 mg/kg.

+Outcome measures

Primary outcomes

28-day overall response rate (ORR) of acute gastrointestinal GVHD [28-day]: 28-day gastrointestinal aGVHD ORR: CR+PR rate at Day 28 per MAGIC criteria.

Secondary outcomes

GVHD-free, relapse-free survival (GRFS) [1-year post-randomization]: GRFS is a composite endpoint measuring the time from treatment initiation to the first occurrence of grade 3-4 acute GVHD, chronic GVHD requiring systemic immunosuppression, hematological relapse, or all-cause death. Patients without any above events are censored at the last valid follow-up date.
Cumulative incidence of relapse（CIR） [1-year post-randomization]: Competing-risk endpoint for hematological relapse from randomization, with non-relapse death as competing risk.

+Eligibility

Sex: All, ages: 14 Years to any

Inclusion Criteria:

* Malignant hematologic diseases eligible for allogeneic hematopoietic stem cell transplantation, including MDS-RAEB, acute leukemia, and CML in chronic phase
* Availability of HLA-matched sibling donors, unrelated donors, or haploidentical donors
* Age 14 years and older
* Liver function: ALT and AST ≤ 2.5 times the upper limit of normal, bilirubin ≤ 2 times the upper limit of normal
* Kidney function: creatinine ≤ upper limit of normal
* No uncontrolled infections or severe psychiatric disorders
* Performance status 0-2 (ECOG)
* Signed informed consent
* Patients with acute gastrointestinal GVHD after transplantation (according to MAGIC criteria)

Exclusion Criteria:

* No suitable allogeneic donor
* Non-gastrointestinal GVHD as the main manifestation
* Either donor or recipient is pregnant
* Psychiatric illness or other conditions preventing protocol compliance

+Locations (1)

Facility	City	Country	Status
Chinese PLA General Hospital	Beijing, Beijing Municipality	China