Prospective Clinical Trial for Children With TCRαβ Depleted vs Traditional Haplo Identicle HSCT

TCRαβ+ Cell Deleted Versus Traditional Haploidentical Hematopoietic Stem Cell Transplantation ：A Single-center, Prospective, Non-randomized Controlled Clinical Study

This is a single-center prospective, non-randomized controlled clinical study in China using CliniMACS TCRα/β+ cell depleted stem cell haploidentical donors versus conventional Beijing protocol for haploidentical hematopoietic stem cell transplantation in children.

Study Overview

• Status: Recruiting
• Conditions: Child, Only; Hematopoietic Stem Cell Transplantation; Graft-Versus-Host Disease; Haploidentical Hematopoietic Stem Cell Transplantation
• Intervention / Treatment: Procedure: TCRαβ Depleted haploidentical HCT

Detailed Description

1:1 non-randomized controlled clinical study in single center using CliniMACS TCRα/β+ cell depleted stem cell versus conventional Beijing protocol for haploidentical hematopoietic stem cell transplant. The purpose of this study is to obtain 30% decrease grade II-IV aGVHD and better qulity of life for TCRα/β+ cell depleted haploidentical stem cell transplantaion.

• Study Type: Interventional
• Enrollment (Estimated): 110
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Jing Chen, PhD; Phone Number: 82073 38626161; Email: chenjing@scmc.com.cn

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China
      • Recruiting
      • Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University
      • Contact: J Chen, PhD; Phone Number: 82073 38626161; Email: chenjing@scmc.com.cn

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Age 8 weeks to 18 years
• Children who meet the indicators haploidentical hematopoietic cell transplantation
• No HLA ≥ 9/10 donor or not suitable for this type of donor due to illness
• Informed consent must be signed (by the patient or legal representative)

Exclusion Criteria:

• Liver function abnormalities with bilirubin >2 mg/dL and elevation of transaminases higher than 400 U/L
• Chronic active viral hepatitis
• Ejection fraction <50%
• Respiratory failure necessitating supplemental oxygen
• Patients with a history of psychiatric illness or a condition which could interfere with their ability to understand the requirements of the study
• Patients unwilling or unable to comply with the protocol or unable to give informed consent
• Concurrent severe or uncontrolled infection

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
No Intervention: Traditional Haplo identicle HCT; Beijing haploidenticla HCT is the routinely traditional way used for haploidentical HCT
Experimental: TCRαβ Depleted haploidentical HCT; TCRαβ Depleted haploidentical HCT is experimental used to decrease GVHD and obtain better qulity of life for children with haploidentical HCT.	Procedure: TCRαβ Depleted haploidentical HCT; Use the CliniMACS TCRα/β deplete from the mobilized peripheral blood stem cells of haploidentical donor in children

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of grade II-IV acute GVHD	Incidence of grade II-IV acute graft-versus-host disease (GVHD) until Day 100 post-transplantation.	day+100
incidence of treatment related mortality(TRM)	compare the incidence of TRM until Day 100 post-transplantation	day +100

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
post HSCT day 1y and 2y GRFS and OS	compare the incidence of GRFS and OS post HSCT 1y and 2y	day +1y and +2y
cGVHD post day 1y and 2y	compare the incidence of cGVHD post HSCT 1y and 2y	day +1y and +2y
TRM post day +1y and +2y	compre the incidence of TRM post HSCT day 1y and 2y	day +1y and +2y

Collaborators and Investigators

• Sponsor: Sijia Gu
• Investigators: Principal Investigator: Jing Chen, PhD, Shanghai Children's Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): May 26, 2024
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): March 31, 2028

Study Registration Dates

• First Submitted: May 15, 2024
• First Submitted That Met QC Criteria: May 15, 2024
• First Posted (Actual): May 21, 2024

Study Record Updates

• Last Update Posted (Actual): May 29, 2024
• Last Update Submitted That Met QC Criteria: May 26, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: SCMCIRB-K2024030-2

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