MTX and Steroid for III-IV aGVHD Treatment

Phase 2

• Conditions: Acute Graft Versus Host Disease
• Interventions: Drug: Methotrexate

• Registration Number: NCT04958538
• Lead Sponsor: Peking University People's Hospital

Brief Summary

The aim of the study is to identify the efficacy and safety of methotrexate (MTX) combined corticosteroid treatment for grade III-IV acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Detailed Description

Allo-HSCT is an effective treatment of malignant hematopoietic diseases. However, aGVHD remains a major complication after allo-HSCT and the destruction of recipient tissues by alloantigen-activated T cells is a key event in the development of aGVHD. Corticosteroid is the standard first-line therapy for aGVHD due to their roles in suppressing T cell responses. However, the response rate of corticosteroid was approximate 50%, and the clinical outcomes of patients with corticosteroid refractory GVHD were poor. Thus far, no combination therapy had been prove to be superior to corticosteroid alone as initial therapy for aGVHD. The study hypothesis: MTX combined corticosteroid treatment could help to further ameliorate the activity of T cells and control aGVHD.

Study Timeline

• Study Start: 2021-04-21
• Study First Submitted: 2021-06-23
• Study First Submitted QC: 2021-07-08
• Study First Posted: 2021-07-12
• Status Verified: 2022-04
• Last Update Submitted: 2022-04-19
• Last Update Posted: 2022-04-20
• Primary Completion: 2022-12-31
• Study Completion: 2023-01-30

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

1. Patients who are fully informed and sign informed consent by themselves or their guardians;
2. Patients receiving allogeneic hematopoietic stem cell transplantation;
3. Patients with acute graft-versus-host disease of grade III-IV were diagnosed after transplantation;
4. Patients with stable implantation of granulocytes and platelets.

Exclusion Criteria

1. Patients who have received more than one transplant;
2. Patients with overlap syndrome;
3. Patients within six months after the failure of the first transplantation;
4. Patients with uncontrollable active infection;
5. Patients with recurrence of primary malignant hematopathy;
6. Patients with donor lymphocyte infusion induced graft-versus-host disease after first intervention;
7. Patients with serious respiratory diseases;
8. Patients with severe renal insufficiency;
9. Patients with serious and uncontrolled heart disease;
10. Patients with severe hepatobiliary diseases unrelated to graft-versus host disease;
11. Within one week patients who need to use more than 1mg/kg/d methylprednisolone for reasons other than graft-versus-host disease;
12. patients who have participated in other clinical trials within 1 month;
13. The researcher judges that there are other factors that are not suitable for participating

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
MTX and corticosteroid	Methotrexate	Methylprednisolone 2 mg/kg/day MTX (5-10 mg/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was less than grade II

Primary Outcome Measures

Name	Time	Method
Overall response rate (ORR) for GVHD treatment after treatment	14 days	Overall response rate is defined as the proportion of patients demonstrating a complete response or partial response without requirement for additional systemic therapies for an earlier progression, mixed response or non-response

Secondary Outcome Measures

Name	Time	Method
Overall survival	1 year	Overall survival
Non-relapse mortality	1 year	Non-relapse mortality
Overall response rate (ORR) at 28 days after treatment	28 days	Overall response rate is defined as the proportion of patients demonstrating a complete response or partial response without requirement for additional systemic therapies for an earlier progression, mixed response or non-response
Failure free survival	1 year	Failure free survival
Disease free survival	1 year	Disease free survival
relapse rate	1 year	Relapse rate is defined as the proportion of patients demonstrating a morphological relapse of the original malignant hematological disease
Chronic GVHD	1 year	number of participants with chronic GVHD at one year

Trial Locations

Locations (1)

Peking University Institute of Hematology,; 🇨🇳 Beijing, Beijing, China