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

Early Phase 1Enrolling By InvitationNCT07803978

Chinese PLA General Hospital60 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Acute Gastrointestinal Graft-versus-host Disease

Interventions

Amimaitosai injectionDRUG

Amimaitosai injection (1×10⁶ cells/kg per infusion, twice weekly for 4 weeks; may extend to 8 weeks for partial responders).

MethylprednisoloneDRUG

Experimental Arm：Methylprednisolone at a daily dose of 1 mg/kg. Active Comparator Arm：Methylprednisolone at a daily dose of 2 mg/kg.

Eligibility

Sex: ALLMin age: 14 Years

Medical Language ↔ Plain English

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)

Chinese PLA General Hospital

Beijing, Beijing Municipality, China

Outcomes

Primary Outcomes

28-day overall response rate (ORR) of acute gastrointestinal GVHD

28-day gastrointestinal aGVHD ORR: CR+PR rate at Day 28 per MAGIC criteria.

Time frame: 28-day

Secondary Outcomes

GVHD-free, relapse-free survival (GRFS)

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.

Time frame: 1-year post-randomization

Cumulative incidence of relapse（CIR）

Competing-risk endpoint for hematological relapse from randomization, with non-relapse death as competing risk.

Time frame: 1-year post-randomization

Data from ClinicalTrials.gov

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