Co-infusion of Treg-enriched Donor Lymphocytes With CD3-depleted Hematopoietic Stem Cell Graft to Prevent Graft-versus Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation Among Children With Hematologic Malignancies

January 16, 2026 updated by: Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Pilot Study of Co-Infusion of Donor Lymphocytes Enriched With Regulatory T Lymphocytes With Ex-vivo CD3-Depleted Hematopoietic Stem Cell Graft for the Prevention of Graft-versus-Host Disease in Children With Hematopoietic and Lymphoid Tissue Neoplasms

Two key methods of GVHD prevention in allogeneic HSCT have a number of limitations: ex vivo T depletion is associated with an excess of infectious complications, and pharmacological immunosuppression with insufficient efficacy of GVHD prevention. Modern graft engineering technologies make it possible to create a graft with a balanced cell composition, reducing the risk of adverse events, in particular, severe forms of acute and chronic GVHD, while preserving the immunological function of the graft. In the proposed concept, enrichment of the T graft with regulatory cells will reduce the risk of GVHD and preserve a sufficient number of T lymphocytes in the graft for the formation of protective anti-infective immunity in the early stages after HSCT. The combination of partial T depletion and pharmacological immunosuppression minimized in volume and duration will combine the advantages of T depletion (early engraftment, low risk of GVHD, low risk of organ complications) and pharmacological prophylaxis (restoration of anti-infective immunity).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

  1. Infusion of ex-vivo T-depleted peripheral blood hematopoietic stem cells (CD3 depletion product)
  2. Infusion of donor lymphocytes enriched with T regulatory lymphocytes (CD25 selective product)

  3. Drug therapy (pharmacological prophylaxis of GVHD)

    • Cyclosporine A
    • Sirolimus o Ruxolitinib o Abatacept

Study Type

Interventional

Enrollment (Estimated)

64

Phase

  • Phase 2
  • Phase 3

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 Locations

  • Russia
      • Moscow, Russia
        • Recruiting
        • National medical research center of pediatric haematology, oncology and immulogy named after Dmytriy Rogachyov, Moscow, 117198
        • 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

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Informed consent signed by the patient (age 14 to 25 years) and/or his/her legal representative (age 0 to 18 years).
  2. The patient has an indication for allogeneic hematopoietic stem cell transplantation (HSCT) established in accordance with the current regulatory framework
  3. Planned HSCT from a haploidentical donor
  4. The Karnofsky or Lansky score is more than 70%
  5. Life expectancy of at least 8 weeks
  6. Heart function: ejection fraction of at least 40%
  7. Consent to continue follow-up for 3 years

Exclusion Criteria:

  1. Acute viral hepatitis or acute HIV infection
  2. Hypoxemia with SaO2 <90%
  3. Bilirubin >3 normal
  4. Creatinine >3 norms
  5. Pregnancy and lactation
  6. Life-threatening infection
  7. Severe (>?) pathology of the central nervous system (epilepsy, dementia, organic damage to the central nervous system)
  8. Karnofsky score or Lansky score <70%

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: Sequential Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Cyclosporine A
Four groups corresponding to the pharmacological prophylaxis of GVHD: 1) Cyclosporine A
Drug: Cyclosporine A (CsA)
The combination of partial T depletion and pharmacological immunosuppression minimized in volume and duration will combine the advantages of T depletion (early engraftment, low risk of GVHD, low risk of organ complications) and pharmacological prophylaxis (restoration of anti-infective immunity).
Active Comparator: Sirolimus
Drug therapy (pharmacological prophylaxis of GVHD) 2) Sirolimus
Drug: Sirolimus
Pharmacological prophylaxis of GVHD is one of the key subjects of evaluation in the current study. Since the optimal pharmacological approach is not known, the allocation of the patients to study groups will be by randomization procedure, although not for the purpose of direct comparison, but for unbiased descriptive analysis. There will be four main groups and an additional group to be open for allocation based on the main group closing for fitting the stopping rules. The details of pharmacological GVHD prevention are Sirolimus 1 mg -3 till +30 4-8 ng/ml
Active Comparator: Ruxolitinib
Drug therapy (pharmacological prophylaxis of GVHD) Ruxolitinib
Drug: Ruxolitinib (JAKAVI®)
Pharmacological prophylaxis of GVHD is one of the key subjects of evaluation in the current study. Since the optimal pharmacological approach is not known, the allocation of the patients to study groups will be by randomization procedure, although not for the purpose of direct comparison, but for unbiased descriptive analysis. There will be four main groups and an additional group to be open for allocation based on the main group closing for fitting the stopping rules. The details of pharmacological GVHD prevention regimens are Ruxolitinib 5 mg -2 till +30
Active Comparator: Abatacept
Drug therapy (pharmacological prophylaxis of GVHD) Abatacept
Drug: Abatacept
Pharmacological prophylaxis of GVHD is one of the key subjects of evaluation in the current study. Since the optimal pharmacological approach is not known, the allocation of the patients to study groups will be by randomization procedure, although not for the purpose of direct comparison, but for unbiased descriptive analysis. There will be four main groups and an additional group to be open for allocation based on the main group closing for fitting the stopping rules. Abatacept 10 mg/kg -1, +7, +14, +28

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Feasibility- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time Frame: day 30
proportion of patients who received an infusion of the planned dose of regulatory T lymphocytes (at least 80%)
day 30
Safety- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time Frame: 120 days after HSCT
Cumulative risk of GVHD Grade III-IV (target < 5%)
120 days after HSCT

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Cumulative probability of engraftment
Time Frame: up to 100 day
up to 100 day
Time to engraftment of neutrophils and platelets
Time Frame: 100 day after HSCT
100 day after HSCT
Cumulative risk of acute GVHD Grade II-IV
Time Frame: up to 100 days after HSCT
up to 100 days after HSCT
Cumulative risk of viral
Time Frame: at 120 day after HSCT
(CMV, ADV, EBV, HHV-6) DNA detection in blood, peak viral DNA load and the duration of detectable viral DNA (each virus separately)
at 120 day after HSCT
cumulative risk of developing severe chronic GVHD
Time Frame: at 2 years
Severity of chronic GVHD
at 2 years
Cumulative risk of leukemia relapse
Time Frame: at 2 years
at 2 years
Cumulative risk of non-relapse mortality
Time Frame: at 100 days and 2 years
at 100 days and 2 years
Overall survival
Time Frame: at 3 years
at 3 years
Event-free survival
Time Frame: at 3 years
at 3 years
GVHD- and relapse-free survival
Time Frame: at 3 years
at 3 years

Collaborators and Investigators

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

Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

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)

September 3, 2025

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

February 3, 2028

Study Registration Dates

First Submitted

January 7, 2026

First Submitted That Met QC Criteria

January 16, 2026

First Posted (Actual)

January 26, 2026

Study Record Updates

Last Update Posted (Actual)

January 26, 2026

Last Update Submitted That Met QC Criteria

January 16, 2026

Last Verified

December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NCHPOI- 2025-14

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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