Clinical trial NCT04960644: MTX and Steroid as First-line Therapy for aGVHD
Official title: A Multi-cohort, Phase Ⅲ Study of Methotrexate Combined Corticosteroid as First-line Therapy in Chinese Patients With Acute Graft vs. Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Acute GVH Disease
Keywords: acute GVHD
MTX
Corticosteroid
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2021-07-14
Enrollment: 157
Sex: ALL
Minimum age (years): 15
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Overall response rate (ORR) for aGVHD treatment after treatment
timeFrame: 10 days
Secondary outcomes:
measure: Overall response rate (ORR) for aGVHD treatment at 28 days after treatment
timeFrame: 28 days
measure: Overall response rate (ORR) for aGVHD treatment at 42 days after treatment
timeFrame: 42 days
measure: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: 42 days
measure: cGVHD
timeFrame: 1 years
measure: Infection and poor graft function
timeFrame: 1 year
measure: Relapse
timeFrame: 1 year
measure: Non-relapse mortality
timeFrame: 1 year
measure: Overall survival
timeFrame: 1 year
measure: Disease free survival
timeFrame: 1 year
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University People's Hospital
Study officials:
affiliation: Peking University People's Hospital
name: Xiao-Jun Huang, Dr.
role: PRINCIPAL_INVESTIGATOR
References: