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AzTrials

Haploidentical Hematopoietic Stem Cell TransplantationPhase 3NCT05166967

Targeted Dose Study of ATG in Haploidentical Hematopoietic Stem Cell Transplantation

A Phase 3 study testing Individual Antithymocyte globulin and Antithymocyte globulin in people with Haploidentical Hematopoietic Stem Cell Transplantation, run by Chinese PLA General Hospital at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is to determine the response and toxicity rate of two different dosages (Individualized dosage VS. fixed dosage) of ATG as a prophylaxis for acute GVHD in haploidentical peripheral blood stem cell transplantation (haplo-PBSCT). Read more about this study

Who is it for?
Ages 14 to 65 years
What is being tested?
Individual Antithymocyte globulin and Antithymocyte globulin
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
204 participants
Where
1 siteChina
Study dates
1 Jan 2022to 29 Jul 2025
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 14 to 65 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 1. All patients should have the indication of Haploidentical hematopoietic stem cell transplant.
  • 2. All patients should sign an informed consent document indicating that they understand the purpose of and procedures required for the study and be willing to participate in the study.

Who cannot join

  • 1.Patients with any conditions not suitable for the trial (investigators' decision).

Trial locations and contacts

1 site in 1 country.

China1 site

  • Chinese PLA General Hospital

    Beijing, Beijing Municipality, 100853

Other trials in China

About this study

Official title: Impact of Optimal Doses of Antithymocyte Globulin Conditioning on Graft Versus-host Disease and Virus Reactivation in Haploidentical Hematopoietic Stem Cell Transplantation

The purpose of this study is to determine the response and toxicity rate of two different dosages (Individualized dosage VS. fixed dosage) of ATG as a prophylaxis for acute GVHD in haploidentical peripheral blood stem cell transplantation (haplo-PBSCT).

Read the detailed description

Acute graft-versus-host disease (aGvHD) is an important complication of haploHSCT. The Seattle group initially introduced the use of ATG as a treatment for acute graft-versus-host disease (aGVHD) in allogeneic hematopoietic stem cell transplantation (haplo-PBSCT) recipients. Presently, in both myeloablative and reduced-intensity conditioning (RIC) haplo-PBSCT, ATG is part of post engraftment immunosuppressive regimens. The regimens for prophylaxis of GVHD based on 10mg/kg rabbit anti-human thymocyte immunoglobin (ATG, Thymoglobin®, Genzyme Polyclonals S.A.S) effectively reduced the occurrence of grade II-IV aGvHD. Howevre, the incidence of cytomegalovirus (CMV) and EB virus (EBV) reactivation were higher due to a slower immune reconstitution. The 100-day cumulative incidence of CMV and EBV viremia were both over 70% in our unmanipulated haplo-PBSCT program. The optimal dose of ATG balancing the efficacy of GVHD prophylaxis and the risk of virus reactivation in haplo-PBSCT remains unknown.

Reports on the pharmacokinetics of Thymoglobulin in allo-HSCT revealed a high variability. Recent pharmacokinetic studies have shown that the half-life of total ATG after transplant is longer than the active ATG (which is available to bind to human lymphocytes and causes the desired immunological effects). And active ATG appears more associated with pharmacodynamics effects. In our previous cohort study, we found that virus reactivation and acute GVHD were highly affected by ATG exposure (area under the curve, AUC). We have found an optimal range of active ATG range is 110-148.5UE/ml.day the efficacy of GVHD prophylaxis and the risk of virus reactivation. The cumulative incidence of CMV reactivation and persistent CMV hyperemia at 180 days after transplantation in the optimal total AUC group was 60.57% and 31.52% respectively. Significantly lower than 77.08% and 56.25% in the non-optimal total AUC group.

The results suggested that Individualized dosing of ATG has a potential advantage in balancing the efficacy of GVHD prophylaxis and the risk of virus reactivation in haplo-PBSCT. This may improve the survival and quality of life of patients undergoing haplo-PBSCT. A prospective randomized trial is required to compare the efficacy of Individualized dosage of ATG as a prophylaxis for acute GVHD in haplo-PBSCT.

Being tested
Individual Antithymocyte globulin, Antithymocyte globulin
Keywords
ATG, Haplo-HSCT

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Individual Antithymocyte globulinDrug

    Individual dose of ATG was Intravenous infused every day from day -5 to day -2 (total ATG dose was calculated based on pharmacokinetic index, within a range of 6 mg/kg to 13mg/kg). Prophylaxis against graft versus-host disease (GVHD) was performed with cyclosporine A (CsA), mycophenolate mofetil (MMF) and short-term methotrexate.

  • Antithymocyte globulinDrug

    A 10mg/kg total dose of antithymocyte globulin (ATG) was added to conditioning regimens for 4 days (from day -5 to day -2). Prophylaxis against graft versus-host disease (GVHD) was performed with cyclosporine A (CsA), mycophenolate mofetil (MMF) and short-term methotrexate.

Study groups in NCT05166967
Study groupRoleWhat they receive
Individual dose of ATGExperimentalIndividual dose of ATG: Individual dose of ATG was Intravenous infused every day from day -5 to day -2 (total ATG dose was calculated based on pharmacokinetic index, within a range of 6 mg/kg to 13mg/kg), and the active ATG concentration ranges from 110 to 148.5UE/ml.
Receives: Drug: Individual Antithymocyte globulin
Fixed dose of ATGActive comparatorA total amount of 10mg/kg ATG was divided into 4 days (from day -5 to day -2). The specific usage: 1.5mg/kg for day -5, 2.5mg/kg for day -4 and day -3, 3.5 mg/kg for day -2.
Receives: Drug: Antithymocyte globulin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. GVHD-free and relapse-free survival (GRFS)

    GRFS is defined as a composite endpoint of death from any cause, disease relapse, grade III-IV acute GVHD, or moderate to severe chronic GVHD requiring systemic immunosuppression therapy.

    Measured: 12 months after transplantation

Secondary outcomes (12)

  1. Cumulative incidences of CMV reactivation

    CMV reactivation was defined as DNA load ≥ 1×104 copies /mL or ≥ 1×103 copies/mL in two consecutive tests. The cumulative incidence of CMV reactivation by Day +180 post-transplantation was defined as the proportion of CMV reactivation occurring at any monitoring point during 6 months post-transplant.

    Measured: 6 months after transplantation

  2. Incidence of CMV disease

    The cumulative incidences of CMV disease in participants after transplantation,

    Measured: 6 months after transplantation

  3. Cumulative incidences of EBV reactivation

    The cumulative incidences of EBV reactivation in participants after transplantation, tested by EBV realtime PCR.

    Measured: 6 months after transplantation

  4. Cumulative incidences of PTLD(posttransplant lymphoproliferative disorders)

    The cumulative incidences of PTLD in participants after transplantation

    Measured: 6 months after transplantation

  5. Cumulative incidences of aGVHD

    The diagnosis and grading of aGVHD are based on the modified Glucksberg grading standard.

    Measured: 365 days after transplantation

  6. Cumulative incidences of cGVHD

    Chronic GVHD can be classified as "limited" or "extensive" according to the Seattle criteria, and also be classified as "mild" or "moderate" or "severe" according to the National Institutes of Health (NIH) criteria.

    Measured: 365 days after transplantation

  7. Neutrophil engraftment

    Neutrophil engraftment is defined as the first of 3 consecutive days with an absolute neutrophil count > 0.5 × 10^9/L.

    Measured: 1 month after transplantation

  8. Platelet engraftment

    Platelet engraftment is defined as the first of 7 consecutive days with an absolute platelet count > 20 × 10^9/L independent from transfusion

    Measured: 1 month after transplantation

  9. Overall survival (OS)

    Overall survival (OS) is defined as the time from randomization to death resulting from any cause.

    Measured: 365 days after transplantation

  10. Disease-free survival (DFS)

    Disease-free survival (DFS) is defined as the time from enrollment to relapse of primary disease or death from any cause, whichever occurred first.

    Measured: 365 days after transplantation

  11. Nonrelapse mortality (NRM)

    Non-relapse mortality (NRM) is defined as the time from enrollment to death of any causes other than hematologic disease relapse.

    Measured: 365 days after transplantation

  12. Infection rate

    Infection rate is defined as the proportion of participants who developed all kinds of infection

    Measured: 365 days after transplantation

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Dec 2021
  2. Study start1 Jan 2022
  3. Primary completion16 Feb 2024
  4. Study completion29 Jul 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Random Group Assignment
Masking
None (open label)
Enrolment
204 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05166967
Sponsor's protocol ID
S2020-484-02
Status last verified
Jul 2025
First submitted
22 Nov 2021
Last updated
21 Jan 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Dou L, Wang N, Zhang H, Yang J, Du J, Liu Q, Zhang X, Li F, Boelens JJ, Wang H, Cao W, Zhang X, Liu D. Therapeutic-drug-monitoring-based ATG Targeted Dosing Strategy in Unmanipulated Haploidentical Haematopoietic Stem Cell Transplantation: a randomized, multicenter, phase 3 clinical trial. Cancer Lett. 2026 Jul 27;658:218766. doi: 10.1016/j.canlet.2026.218766. Online ahead of print.

    PubMed 42508748

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Individual Antithymocyte globulin and Antithymocyte globulin in people with Haploidentical Hematopoietic Stem Cell Transplantation, run by Chinese PLA General Hospital at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 21 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

GVHD-free and relapse-free survival (GRFS) (time frame: 12 months after transplantation). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05166967, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jan 2026.

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