Comparison of Different Dose of Post-transplantation Cyclophosphamide as Graft Versus Host Disease Prophylaxis

A Comparison Study of Reduced Dose of Post-transplantation Cyclophosphamide (PTCy 40mg/kg) Versus Standard (PTCY 50mg/kg): a Multi-center Study for Halo-identical Donor Transplantation

Post-transplantation cyclophosphamide (PTCY) is considered as major graft versus host disease (GVHD) prophylaxis. In our previous study, the investigators demonstrated that the standard dose PTCY of 50mg/kg with tacrolimus and post-engrafted low-dose anti-thymoglobulin (ATG) achieved low incidence of acute GVHD. More recently, it has been shown that reduced dose of PTCY of 40mg/kg is considered with similar efficacy as GVHD prophylaxis, In this study, a multi-center randomized comparison is planned to evaluate the clinical outcome of GVHD prophylaxis of PTCy 40 versus 50.

Study Overview

• Status: Not yet recruiting
• Conditions: GVHD
• Intervention / Treatment: Drug: PTCY

Detailed Description

Post-transplantation cyclophosphamide (PTCY) is considered as major graft versus host disease (GVHD) prophylaxis. In our previous study, the investigators demonstrated that the standard dose PTCY of 50mg/kg with tacrolimus and post-engrafted low-dose anti-thymoglobulin (ATG) achieved low incidence of acute GVHD. In the clinical setting, reduced dose of PTCY (40mg/kg) was used for patients over 60 or with high HCT-CI and the overall incidence of acute GVHD was similar to PTCY 50mg/kg but chronic GVHD was slightly increased. To confirmed our observation, in this study, a prospective multiple-center randomized comparison is planned to evaluate the clinical outcomes of reduced dose of PTCY (40mg/kg) versus 50mg/kg as GVHD prophylaxis.

• Study Type: Interventional
• Enrollment (Estimated): 138
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: chun Wang; Phone Number: 8613386259777; Email: wangchunsh@medmail.com.cn

Study Locations

• China
  • GaungDong
    • Shenzhen, GaungDong, China, 518000
      • ShenZhen People's Hospital
  • Shanghai
    • Shanghai, Shanghai, China, 200025
      • Rui Jin Hospital

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Patients with hematological malignancies
• Patients undergo allogeneic stem cell transplantation from haplo-identical donors
• Patents with informed consent provided

Exclusion Criteria:

• Patients with active infection ()bacteria, fungal or viral)
• Patients with liver, renal and cardiac dysfunction not suitable for undergoing allogeneic stem cell transplantation.

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: PTCy-40; Patients receive 40mg/kg PTCY (day+3 and +4) with tacrolimus from day+5 and low-dose ATG of 2,5mg/kg 72 hours after documentation neutrophil engraftment.	Drug: PTCY; PTCY as 40mg/kg or 50mg/kg at day +3 and +4 for GVHD prophylaxis
Active Comparator: PTCy-50; Patients receive 50mg/kg PTCY (day+3 and +4) with tacrolimus from day+5 and low-dose ATG of 2,5mg/kg 72 hours after documentation neutrophil engraftment.	Drug: PTCY; PTCY as 40mg/kg or 50mg/kg at day +3 and +4 for GVHD prophylaxis

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
acute GVHD (grade II-IV)	clinical documentation of grade II-IV aGVHD	day 100

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Non-relapse mortality	Death without documentation of disease relapse or progression (bone marrow >5% or with extra medullary diseases)	1 year
chronic GVHD	incidence of patients with clinical documentation of chronic GVHD	1 year
overall survival	Event defined as death of any causes	1 year
Disease-free survival	Event defined as death of any causes and disease relapse or progression	1 year
Survival without relapse and moderate to severe GVHD	Rate of patients remain alive without disease relapse or progression (bone marrow blast >5% or extra medullary diseases) and without documentation of grade III-IV acute GVHD and moderate to severe chronic GVHD	1 year

Collaborators and Investigators

• Sponsor: Shanghai Jiao Tong University School of Medicine
• Collaborators: Shanghai Clinical Research Center
• Investigators: Study Director: Chun Wang, Zhaxin Hospital, Go Broad Health Care

Study record dates

Study Major Dates

• Study Start (Estimated): October 1, 2023
• Primary Completion (Estimated): October 1, 2025
• Study Completion (Estimated): April 1, 2026

Study Registration Dates

• First Submitted: August 12, 2023
• First Submitted That Met QC Criteria: August 17, 2023
• First Posted (Actual): August 21, 2023

Study Record Updates

• Last Update Posted (Actual): August 21, 2023
• Last Update Submitted That Met QC Criteria: August 17, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Keywords: allogeneic stem cell transplantation; graft versus host disease (GVHD); post-transplantation cyclophosphamide (PTCy); haplo-identical donor
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: PTCY40-50

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No