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

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.

Study Overview

• Status: Completed
• Conditions: Graft Versus Host Disease
• Intervention / Treatment: Drug: Ruxolitinib+Calcineurin inhibitor+Methotrexate; Drug: Calcineurin Inhibitor+Methotrexate+Mycophenolate mofetil

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.

• Study Type: Interventional
• Enrollment (Actual): 215
• Phase: Not Applicable

Contacts and Locations

Study Locations

• China
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310006
      • The First Hospital of Zhejiang Medical Colleage Zhejiang University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 70 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Control group	Drug: Calcineurin Inhibitor+Methotrexate+Mycophenolate mofetil; calcineurin inhibitor and short course of methotrexate and mycophenolate mofetil
Experimental: RUX group	Drug: Ruxolitinib+Calcineurin inhibitor+Methotrexate; low-dose ruxolitinib combine with calcineurin inhibitor and short course of methotrexate.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
100-day cumulative II-IV aGVHD incidence after HSCT	Cumulative incidence of grade II-IV acute GVHD occurring within the first 100 days following transplantation.	From the date of transplantation to the first occurrence of grade II-IV acute GVHD, assessed up to 100 days.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative Incidence of Chronic GVHD	The cumulative incidence of chronic GVHD following transplantation.n	From the date of transplantation to the first diagnosis of chronic GVHD, assessed up to 3 years.
Overall Survival	Overall survival following is defined as the time from transplantation to death from any cause.	From the date of transplantation until death from any cause, assessed up to 3 years.
Adverse Events	Incidence and type adverse events following transplantation.	From the date of transplantation to day 180 post-transplantation.
Cumulative Incidence of Relapse	Cumulative incidence of disease relapse following transplantation is defined as the time from transplantation to the first documented disease recurrence.	From the date of transplantation to the first documented relapse, assessed up to 3 years.
GVHD-Free, Relapse-Free Survival	GVHD-free, relapse-free survival is defined as the time from transplantation to the first occurrence of grade III-IV acute GVHD, chronic GVHD requiring systemic therapy, disease relapse, or death from any cause.	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.
Non-Relapse Mortality	Non-relapse mortality is defined as death without evidence of relapse or progression of the primary disease following transplantation.	From the date of transplantation to death without prior relapse or disease progression, assessed up to 3 years.
Relapse-Free Survival	Relapse-free survival is defined as the time from transplantation to the first documented relapse of the underlying disease or death from any cause.	From the date of transplantation to the first documented relapse or death from any cause, assessed up to 3 years.

Collaborators and Investigators

• Sponsor: Zhejiang University
• Collaborators: Ruijin Hospital; The First Affiliated Hospital of Zhengzhou University; The Children's Hospital of Zhejiang University School of Medicine; Yinzhou Hospital Affiliated to Medical School of Ningbo University

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2021
• Primary Completion (Actual): December 1, 2024
• Study Completion (Actual): December 1, 2024

Study Registration Dates

• First Submitted: April 5, 2021
• First Submitted That Met QC Criteria: April 8, 2021
• First Posted (Actual): April 9, 2021

Study Record Updates

• Last Update Posted (Actual): June 4, 2025
• Last Update Submitted That Met QC Criteria: May 29, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: Graft Versus Host Disease; ruxolitinib; HLA-haploidentical hematopoietic stem cell transplantation
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease; Anti-Bacterial Agents; Anti-Infective Agents; Antibiotics, Antineoplastic; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antirheumatic Agents; Abortifacient Agents, Nonsteroidal; Abortifacient Agents; Reproductive Control Agents; Antimetabolites, Antineoplastic; Antimetabolites; Dermatologic Agents; Folic Acid Antagonists; Nucleic Acid Synthesis Inhibitors; Antibiotics, Antitubercular; Antitubercular Agents; Methotrexate; Mycophenolic Acid; Calcineurin Inhibitors
• Other Study ID Numbers: RCMvsCM

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No