Clinical trial NCT03701698: Ruxolitinib and Steroid As First Line Therapy for Acute GVHD
Official title: Ruxolitinib and Methylprednisolone As First Line Therapy for Acute Graft Versus Host Disease Following Allogeneic Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Acute GVHD
Lead sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Sponsor class: OTHER
First posted: 2018-10-10
Enrollment: 1
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: The purpose of this study is to evaluate the efficacy of ruxolitinib in combination with methylprednisolone as first line therapy in patients with Grades II to IV acute graft-versus-host disease (GVHD).

Detailed description: Treatment:

Once patients are diagnosed with grade II\~IV acute GVHD, the combination therapy should be initiated as soon as possible.

1. Methylprednisolone: 2mg/kg/d, iv or iv gtt, in two or three divided doses. Taper steroid every one or two weeks according to patient's response.
2. Ruxolitinib 5\~10mg bid po for at least 28 days. If patient's ANC\<0.5×10e9/L or PLT\< 20×10e9/L, cease ruxolitinib until recovery of ANC higher than 0.5×10e9/L or PLT higher than 20×10e9/L.

Indication for stopping Ruxolitinib treatment:

1. No response after ruxolitinib treatment for 28 days.
2. Develop life-threatening complication.
3. ANC\<0.5×10e9/L or PLT\< 20×10e9/L.

Indication for second line acute GVHD treatment:

1. deterioration of acute GVHD in 3 days
2. no response after 7 days
3. no complete remission after 2 weeks.

Suggestions of second line therapy:

Basiliximab 20mg, d1, d4, d8.

Eligibility criteria: Inclusion Criteria:

1. diagnosed with hematological diseases.
2. recipients of allogeneic peripheral blood stem cell transplantation.
3. new onset of grade II\~IV acute graft versus host disease within 100 days post-transplantation.

exclusion criteria:

1. recipients of second allogeneic stem cell transplant.
2. acute GVHD induced by donor lymphocyte infusion, interferon.
3. received treatment other than steroid before enrollment.
4. overlap GVHD syndrome.
5. pregnant or breast-feeding women.
6. absolute neutrophil count (ANC) \<0.5×10e9/L or platelet count (PLT) \< 20×10e9/L
7. non-GVHD related liver dysfunction: glutamic pyruvic transaminase\>= 4 times of upper normal limit, direct bilirubin \>= 4 times of upper normal limit
8. renal dysfunction: creatinine clearance \< 15 mL/min or glomerular filtration rate\< 15 mL/min
9. uncontrolled infection
10. human immunodeficiency virus infection
11. active hepatitis b virus, hepatitis C virus infection and need antivirus treatment.
12. relapse of primary malignant hematological diseases, or graft rejection.
13. allergic history to Janus kinase inhibitors.

Primary outcomes:
measure: overall response rate
timeFrame: 28 days after treatment
Secondary outcomes:
measure: time to response
timeFrame: 28 days after treatment
measure: reactivation rate of cytomegalovirus
timeFrame: 100 days within transplant
measure: 1 year incidence of chronic GVHD
timeFrame: 1 year within transplant
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Shanghai General Hospital
state: Shanghai
Study officials:
affiliation: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
name: Xianmin Song, M.D.
role: PRINCIPAL_INVESTIGATOR
References: