Individualized Application of Anti-Thymocyte Globulin(ATG)in Unrelated Donor Hematopoietic Stem Cell Transplantation

June 11, 2026 updated by: Daihong Liu

Application of Anti-Thymocyte Globulin(ATG) Individualized Dosing Model in Unrelated Donor Hematopoietic Stem Cell Transplantation

This study aims to compare individualized anti-thymocyte globulin (ATG) dosing versus conventional fixed-dose regimens in unrelated donor peripheral blood stem cell transplantation (URD-PBSCT).

This study notes that URD-HSCT is a key treatment for malignant hematologic diseases and severe bone marrow failure, with rapid expansion in China. However, this study identifies post-transplant CMV infection as a major challenge, adversely affecting survival and quality of life. This study finds that CMV infection compromises immunity and causes multi-organ complications. Given the high costs, long treatment cycles, and limited efficacy of current interventions, this study considers optimizing CMV prevention to be of greater value than expanding treatment options. This study asserts that effective prevention can reduce infection rates and improve overall survival (OS) and long-term prognosis.

This study recognizes that ATG is widely used in URD-HSCT to prevent graft-versus-host disease (GVHD), but its dosage is significantly linked to CMV risk. This study indicates that inadequate ATG exposure increases GVHD risk, while excessive exposure raises viral reactivation (e.g., CMV, EBV) and may cause relapse. This study thus identifies balancing GVHD prevention and infection control as a key clinical goal. This study cites Remberger et al. (2004), who compared ATG doses (4-10 mg/kg) in 162 URD-HSCT patients, finding lower doses increased acute GVHD (aGVHD) and 10 mg/kg raised infection-related mortality, suggesting 6-8 mg/kg as a balanced range. This study also references Bacigalupo et al. (2001), who found no survival differences across doses but noted higher doses reduced severe aGVHD at the cost of increased infection. Therefore, this study concludes that optimal ATG dosing requires balancing GVHD, infection, and relapse.

This study acknowledges that ATG pharmacokinetics (PK) are complex, influenced by dose, body weight, and absolute lymphocyte count (ALC). This study points out that even with fixed dosing, internal exposure (active ATG-AUC) varies greatly among individuals, indicating that fixed dosing is suboptimal and individualized strategies are needed. This study notes that Admiraal et al. developed an ALC-based individualized ATG model, improving immune reconstitution, reducing viral infections, and enhancing OS. However, this study observes that this model was designed for non-myeloablative conditioning and is not applicable to myeloablative conditioning (MAC), which is standard in China.

To address this, this study states that our team initiated ATG PK studies in 2019. This study explains that under MAC, ALC is nearly eliminated, making traditional models unsuitable. By monitoring active ATG-AUC in 106 haploidentical HSCT (haplo-HSCT) patients and using machine learning, this study identified an optimal exposure window of 100-148.5 UE·day/mL. This study found that patients within this window had lower CMV/EBV reactivation without increased GVHD. This study developed a protocol adjusting doses on days -3 and -2 based on ATG concentrations measured on days -5 and -4. This study confirmed through a prospective single-arm study in haplo-HSCT that this regimen reduces CMV/EBV infection and improves disease-free survival (DFS) and OS while maintaining GVHD control.

Given the consistency between URD-PBSCT and haplo-PBSCT in conditioning, GVHD prophylaxis, and CMV prevention-and that CMV infection rates in Chinese URD-PBSCT patients reach 65%-70%-this study extends the individualized ATG protocol to URD-PBSCT to validate its universality across donor sources.

In summary, building on prior haploidentical transplant research, this study applies individualized ATG dosing to URD-PBSCT. This study aims to precisely regulate ATG exposure to reduce CMV infection while maintaining GVHD prophylaxis. This study seeks to improve patient survival and outcomes, laying the foundation for a population PK model and advancing HSCT toward precision medicine.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
324

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
    • Beijing Municipality
      • Beijing, Beijing Municipality, China, 100853
        • Recruiting
        • Chinese PLA General Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Patients with indications for allogeneic hematopoietic stem cell transplantation, with malignant hematologic diseases in CR1 or CR2 before transplantation.
  2. Have an HLA-matched sibling, unrelated, or haploidentical donor.
  3. Age ≥ 14 years and ≤ 65 years.
  4. Liver function: ALT and AST ≤ 2.5 × upper limit of normal, bilirubin ≤ 2 × upper limit of normal.
  5. Renal function: creatinine ≤ upper limit of normal.
  6. No uncontrolled infection or severe mental or psychological disorders.
  7. ECOG performance status score of 0-2.
  8. Signed informed consent.

Exclusion Criteria:

- 1.No HLA-matched donor. 2.Malignant hematologic disease in CR3 or higher disease stage, or refractory/relapsed status.

3.Patient age < 14 years or > 65 years. 4.Pregnancy of either the donor or the recipient. 5.Presence of mental illness or other conditions that preclude compliance with the protocol.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: Control group
Drug: Anti-Thymocyte Globulin
Patients receiving individualized dosing of ATG + standard GVHD prophylaxis regimen
Drug: Anti-Thymocyte Globulin
Patients receiving fixed-dose ATG + standard GVHD prophylaxis regimen
Experimental: Experimental group
Drug: Anti-Thymocyte Globulin
Patients receiving individualized dosing of ATG + standard GVHD prophylaxis regimen
Drug: Anti-Thymocyte Globulin
Patients receiving fixed-dose ATG + standard GVHD prophylaxis regimen

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
1-year GVHD-free and relapse-free survival rate(1-year-GRFS)
Time Frame: 1 years after treatment
The 1-year GVHD-free and relapse-free survival rate (1-year GRFS) is the proportion of patients who, within one year post-treatment, have not experienced: grade III-IV acute graft-versus-host disease (GVHD), moderate-to-severe chronic GVHD requiring systemic immunosuppression, or disease relapse or progression.
1 years after treatment

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
overall survival (OS)
Time Frame: 1 years after treatment
Overall survival (OS) refers to the time from the start of treatment to the death of the patient for any reason.
1 years after treatment
+180 days CMV virus reactivation rate
Time Frame: 180 days post-transplant
+180 days CMV virus reactivation rate refers to the time from randomization to CMV virus reactivation, relapse or death within 180 days post-transplant. The actual incidence rate is calculated as the number of patients with events / total number of cases × 100%.
180 days post-transplant
Acute GVHD incidence
Time Frame: 100 days post-transplant
Acute GVHD incidence refers to the time from randomization to acute GVHD onset, relapse or death within 100 days post-transplant. The actual incidence rate is calculated as the number of patients with events / total number of cases × 100%.
100 days post-transplant
disease free survival (DFS)
Time Frame: 1 years after treatment
Disease free survival (DFS) refers to the time from treatment to the first lymphoma recurrence.
1 years after treatment
Cumulative incidence of relapse (CIR)
Time Frame: 1 years after treatment
Cumulative incidence of relapse (CIR) refers to the number of patients with hematologic or MRD relapse from randomization to the last follow-up.
1 years after treatment
Cumulative non-relapse mortality (NRM)
Time Frame: 1 years after treatment
Cumulative non-relapse mortality (NRM) refers to death due to non-relapse causes while in complete remission (CR), measured as the number of patients with NRM from randomization to the last follow-up.
1 years after treatment
Treatment-related safety indicators
Time Frame: 1 years after treatment
Treatment-related safety indicators mainly include the cumulative incidence of bacterial infection, viral infection, fungal infection, PTLD, and chronic GVHD from randomization to the last follow-up.
1 years after treatment
Immune reconstitution status
Time Frame: 1 years after treatment
Immune reconstitution status refers to the actual incidence rate calculated as the number of patients with immune reconstitution / total number of cases × 100% from randomization to the last follow-up.
1 years after treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Daihong Liu

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Peking University People's Hospital
The First Affiliated Hospital of Zhengzhou University

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Chair: Dai-Hong Liu, Dr., Chinese PLA General Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 1, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 30, 2029

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 30, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 9, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 11, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 15, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 15, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 11, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • S2025-1112-01

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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