MTX and Steroid as First-line Therapy for aGVHD

Phase 3

Active, not recruiting

• Conditions: Acute GVH Disease
• Interventions: Drug: Corticosteroid; Drug: MTX

• Registration Number: NCT04960644
• 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 as first-line therapy for acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplantation (alloHSCT).

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 First Submitted: 2021-06-23
• Study Start: 2021-06-25
• Study First Submitted QC: 2021-07-02
• Study First Posted: 2021-07-14
• Primary Completion: 2022-07-31
• Status Verified: 2022-11
• Last Update Submitted: 2022-11-14
• Last Update Posted: 2022-11-16
• Study Completion: 2022-12-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 157

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 II-IV were diagnosed after transplantation;
4. Patients with stable implantation of granulocytes and platelets.
5. ECOG score ≤3

Exclusion Criteria

1. Patients with severe brain, heart, kidney or liver dysfunction unrelated to graft-versus host disease;
2. Patients with uncontrollable active infection;
3. Patients with recurrence of primary malignant hematopathy;
4. Expected survival is less than 3 months
5. Patients who have histories of severe allergic reactions
6. Pregnant or lactating women
7. The researcher judges that there are other factors that are not suitable for participating

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
corticosteroid	Corticosteroid	perimental: corticosteroid Methylprednisolone 1 mg/kg/day was given for 10 days and then gradually reduce the dose according to patient's response
MTX and corticosteroid	MTX	perimental: MTX and corticosteroid Methylprednisolone 1 mg/kg/day was given for 10 days and then gradually reduce the dose according to patient's response MTX (5-6mg/m\^2/day,Maximum dose 10mg/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR

Primary Outcome Measures

Name	Time	Method
Overall response rate (ORR) for aGVHD treatment after treatment	10 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 response rate (ORR) for aGVHD treatment 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
cGVHD	1 years	The cumulative incidence of chronic GVHD
Overall response rate (ORR) for aGVHD treatment at 42 days after treatment	42 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
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	42 days	Data collection including questionnaires at individual and group visits and physician interviews at individual visits will be used to assess participants for treatment-related adverse events.
Infection and poor graft function	1 year	The cumulative incidence of severe infection and poor graft function
Relapse	1 year	The cumulative incidence of relapse
Non-relapse mortality	1 year	The cumulative incidence of non-relapse mortality
Overall survival	1 year	The cumulative incidence of overall survival
Disease free survival	1 year	The cumulative incidence of disease free survival

Trial Locations

Locations (1)

Peking University People's Hospital; 🇨🇳 Beijing, China