Clinical trial NCT05921305: Mini-dose MTX Plus Standard-dose Steroid for the Initial Treatment of Acute GVHD
Official title: Randomized Trial of Mini-dose Methotrexate Plus Standard-dose Steroid vs Steroids for the Initial Treatment of Acute Graft Versus Host Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Acute Graft Versus Host Disease
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2023-06-27
Enrollment: 218
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: This trial is a randomized (1:1) phase III open label study of frontline mini-MTX plus methylprednisolone 2mg/kg/day compared to methylprednisolone 2mg/kg in allogeneic stem cell transplant recipients with grade 2-4 aGVHD.

Detailed description: Allo-HSCT is an effective treatment of malignant hematopoietic diseases. However, aGVHD remains a major complication after allo-HSCT. Corticosteroid is the standard first-line therapy for aGVHD. 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 control aGVHD.

Eligibility criteria: Inclusion Criteria:

1. Patients who are fully informed and sign informed consent by themselves or their guardians;
2. Patients receiving first allogeneic hematopoietic stem cell transplantation;
3. Patients with acute graft-versus-host disease of grade II-IV were diagnosed after transplantation;
4. KPS\>60, Estimated survival \>3 months;
5. No serious organ damage:

   1. ANC in peripheral blood is greater than 0.5×109/l
   2. Creatinine \< 1.5mg/dl
   3. Cardiac ejection index \> 55%

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
8. Patients who received donor lymphocyte infusion

Primary outcomes:
measure: Overall response rate (ORR) for aGVHD treatment after treatment
timeFrame: 14 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 Institute of Hematology,
state: Beijing
city: Guangzhou
country: China
facility: Nanfang Hospital, Nanfang Medical University
state: Guangdong
Study officials:
References: