Clinical trial NCT04838704: Ruxolitinib With Calcineurin Inhibitor and Methotrexate vs. Calcineurin Inhibitor Plus Methotrexate and Mycophenolate Mofetil as Graft Versus Host Disease Prophylaxis for HLA-haploidentical Hematopoietic Stem Cell Transplantation
Official title: Ruxolitinib With Calcineurin Inhibitor and Methotrexate vs. Calcineurin Inhibitor Plus Methotrexate and Mycophenolate Mofetil as Graft Versus Host Disease Prophylaxis for HLA-haploidentical Hematopoietic Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Graft Versus Host Disease
Keywords: Graft Versus Host Disease
ruxolitinib
HLA-haploidentical hematopoietic stem cell transplantation
Lead sponsor: Zhejiang University
Sponsor class: OTHER
Collaborators: name: The Children's Hospital of Zhejiang University School of Medicine
name: Yinzhou Hospital Affiliated to Medical School of Ningbo University
name: Ruijin Hospital
name: The First Affiliated Hospital of Zhengzhou University
First posted: 2021-04-09
Enrollment: 215
Sex: ALL
Minimum age (years): 12
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

Brief summary: Low Dose Ruxolitinib with Calcineurin Inhibitor and Methotrexate vs. Calcineurin Inhibitor plus Methotrexate and Mycophenolate mofetil as Graft Versus Host Disease prophylaxis for HLA-haploidentical hematopoietic stem cell transplantation in low-dose antithymocyte globulin (ATG) system.

Detailed description: The is a prospective, randomized two-arm, and multicenter study. To compare the efficacy and safety of low-dose ruxolitinib combined with calcineurin inhibitor and methotrexate vs. calcineurin inhibitor plus methotrexate and mycophenolate mofetil as graft versus host disease prophylaxis for HLA-haploidentical hematopoietic stem cell transplantation in low-dose antithymocyte globulin (ATG) system.

Eligibility criteria: Inclusion Criteria:

1. Patients must be diagnosed with malignant hematological disease.
2. aged 12-70 years.
3. Received HLA-haploidentical hematopoietic stem cell transplantation.
4. received myeloablative conditioning
5. Karnofsky score ≥70.
6. creatinine clearance ≥60 mL/min (according to the Cockcroft-Gault formula). (7) liver and kidney function: aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 × upper limit of the normal range (ULN), total bilirubin ≤ 2 × ULN; serum creatinine ≤ 1.5 × ULN.

8) left ventricular ejection fraction (LVEF) ≥ 50% on echocardiography (ECHO). 9) life expectancy \>12 weeks. 10) Voluntarily signed the consent form and could understand and comply with the requirements of the study.

Exclusion Criteria:

1. Active autoimmune disease, such as SLE, rheumatoid arthritis, etc.
2. Current clinically significant active cardiovascular disease such as uncontrolled arrhythmia, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease as determined by New York Heart Association (NYHA) functional class, or a history of myocardial infarction within 6 months prior to enrollment.
3. Other serious medical conditions that may limit the patient's participation in this trial (e.g., progressive infection, uncontrolled diabetes).
4. human immunodeficiency virus (HIV) infection.
5. cirrhosis of the liver, active hepatitis.
6. Pregnant or lactating women.
7. Patients who are concurrently enrolled in any clinical trials of similar drugs.

Primary outcomes:
measure: 100-day cumulative II-IV aGVHD incidence after HSCT
timeFrame: From the date of transplantation to the first occurrence of grade II-IV acute GVHD, assessed up to 100 days.
Secondary outcomes:
measure: Cumulative Incidence of Chronic GVHD
timeFrame: From the date of transplantation to the first diagnosis of chronic GVHD, assessed up to 3 years.
measure: Overall Survival
timeFrame: From the date of transplantation until death from any cause, assessed up to 3 years.
measure: Adverse Events
timeFrame: From the date of transplantation to day 180 post-transplantation.
measure: Cumulative Incidence of Relapse
timeFrame: From the date of transplantation to the first documented relapse, assessed up to 3 years.
measure: GVHD-Free, Relapse-Free Survival
timeFrame: From the date of transplantation to the first occurrence of grade III-IV acute GVHD, systemic therapy-requiring chronic GVHD, relapse, or death, assessed up to 3 years.
measure: Non-Relapse Mortality
timeFrame: From the date of transplantation to death without prior relapse or disease progression, assessed up to 3 years.
measure: Relapse-Free Survival
timeFrame: From the date of transplantation to the first documented relapse or death from any cause, assessed up to 3 years.
Other outcomes:
Locations:
city: Hangzhou
country: China
facility: The First Hospital of Zhejiang Medical Colleage Zhejiang University
state: Zhejiang
Study officials:
References: