Study Comparing Reduced Versus Standard Dose Post-transplantation Cyclophosphamide in Combination With Post-engraftment Anti-thymoglobin as Graft Versus Host Disease Prophylaxis in Alternative Donor Peripheral Stem Cell Transplantation

A Multi-center Randomized Clinical Study Comparing Reduced-dose (35mg/kg) Versus Standard Dose (50mg/kg) Post-transplantation Cyclophosphamide in Combination With Post-engraftment Anti-thymoglobin (ATG) and Tacrolimus and Post-engraftment Anti-thymoglobin as Graft Versus Host Disease (GVHD) Prophylaxis in Patients Undergo Alternative Donor Peripheral Hematopoietic Stem Cell Transplantation

This is a multi-center randomized study to compare the reduced-dose of post-transplantation cyclophosphamide (PTCY) at 35mg.kg to standard dose at 50mg/kg combined with tacrolimus and post-engraftment low dose anti-thymoglobin (ATG) as graft versus host disease (GVHD) prophylaxis in patients undergoing allogeneic stem cell transplantation from alternative donor.

Study Overview

• Status: Recruiting
• Conditions: Graft Versus Host Disease; Allogeneic Hematopoietic Cell Transplant
• Intervention / Treatment: Drug: reduced-dose PTCy; Drug: Standard dose PTCY

Detailed Description

This is a multi-center randomized study in patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) from alternative donors (9-10/10 matched unrelated donor or haplo-identical donors). The aim is to evaluate to the efficacy and feasibly of reduced-dose of PTCY at 35mg/kg as GVHD prophylaxis. Patients will be assigned to study group receiving PTCy at 35mg/kg on day +3 and +4 together with tacrolimus starting from day +5 and single dose anti-thymoglobin at 2.5mg/kg as GVHD prophylaxis or to the control group with standard dose of PTCY at 50mg/kg on day +3 and +4 with same dose of tacrolimus and ATG as the study group.

• Study Type: Interventional
• Enrollment (Estimated): 316
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Jiong Hu; Phone Number: 86-13764313546; Email: hj10709@rjh.com.cn
• Study Contact Backup: Name: Li-ning Wang; Phone Number: 86-21-64370045

Study Locations

• China
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China, 200025
      • Recruiting
      • Ruijin hsopital
      • Contact: JIong HU; Phone Number: 601878 86-21-64370045; Email: hj10709@rjh.com.cn
      • Principal Investigator: JIong HU
      • Contact: Li-ning Wang

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. patients undergo allo-HSCT with matched unrelated donor or haplo-identical donor;
2. normal organ function (creatinine clearance ≥ 50ml/min/1.73m2 or creatinine ≤2mg/d(or 177μmol/L); no hepatic abnormal (ALT or AST≤2.5xN; TBil≤1.5XN); Normal pulmonary function (FEV1、 FVC、DLCO≥80%); normal cardiac function (EFS ≥50%);
3. ECOG: 0-2;
4. Life expectation ≥3 months;
5. Informed consent provided.

Exclusion Criteria:

1. Pregnancies
2. active hepatitis (HBV-DNA≥1×103 copies/ml);
3. active infection require anti-biotics;
4. HIV infection

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
Experimental: Reduced dose of post-transplantation cyclophosphamide; study group	Drug: reduced-dose PTCy; Patients receiving reduced dose of PTCy at 35mg.kg on day +3 and +4 together with tacrolimus starting from day+5 and single dose of anti-thymoglobin (ATG) 2.5mg/kg at 72 hours after documentation of neutrophil engraftment.
Active Comparator: Standard-dose PTCy; Patients receiving standard dose of post-transplantation cyclophosphamide	Drug: Standard dose PTCY; Patients receiving standard dose PTCy at 50mg/kg on day +3 and +4 with tacrolimus starting from day +5 and single dose of anti-thymoglobin at 2.5mg/kg 72 hours after documentation of neutrophil engraftment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
100-day II-IV acute GVHD	Patients diagnosed with grade II-IV acute GVHD within 100 day after allo-HSCT	100 day

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
1-year NRM	Patients died of any causes without documentation of relapse or disease progression 1 year after allo-HSCT	1 year
100-day acute GVHD	Patients diagnosed with aGVHD of all grades within 100 days after allo-HSCT	100 day
100-day non-relapse mortality	Patients died of any causes without documentation of disease relapse or progression at day 100 after allo-HSCT	100 day
1-year chronic GVHD	Patients diagnosed with chronic GVHD of all grades at 1 year after allo-HSCT	1 year
1-year moderate to severe chronic GVHD	Patients diagnosed with moderate to severe chronic GVHD at 1 year after allo-HSCT	1 year
1-year cumulated incidence of relapse	Patients died of relapse or disease progression 1 year after allo-HSCT	1 year
1-year overall survival	Patients remained alive at 1 year after allo-HSCT	1 year
1-year graft host disease free and relapse free survival	Patients remained alive without relapse, grade III-IV aGVHD or moderate to severe chronic GVHD	1 year

Collaborators and Investigators

• Sponsor: Shanghai Jiao Tong University School of Medicine
• Collaborators: Shanghai Jiao Tong University Affiliated Sixth People's Hospital
• Investigators: Principal Investigator: Jiong HU, Ruijin Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): July 1, 2028

Study Registration Dates

• First Submitted: November 23, 2024
• First Submitted That Met QC Criteria: November 23, 2024
• First Posted (Actual): November 26, 2024

Study Record Updates

• Last Update Posted (Actual): March 17, 2026
• Last Update Submitted That Met QC Criteria: March 15, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: prophylaxis; graft versus host disease; reduced dose; post-transplantation cyclophosphamide
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: PTCY-REDSTAR-2024

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

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