Acute Myeloid Leukemia, RelapsedPhase 2/3NCT07366801
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
A Phase 2/3 study testing Cyclosporine A (CsA), Sirolimus and Ruxolitinib (JAKAVI®) in people with Acute Myeloid Leukemia, Relapsed and Acute Lymphoblastic Leukemia, High Risk, run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology at 1 site in Russia.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 year to 25 years
- Conditions the study is for
- Acute Myeloid Leukemia, Relapsed, Acute Lymphoblastic Leukemia, High Risk, Acute Myeloid Leukemia, High Risk
- What is being tested?
- Cyclosporine A (CsA), Sirolimus and Ruxolitinib (JAKAVI®)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 1 siteRussia
- Study dates
- 3 Sep 2025to 3 Feb 2028
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 1 year to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Informed consent signed by the patient (age 14 to 25 years) and/or his/her legal representative (age 0 to 18 years).
- The patient has an indication for allogeneic hematopoietic stem cell transplantation (HSCT) established in accordance with the current regulatory framework
- Planned HSCT from a haploidentical donor
- The Karnofsky or Lansky score is more than 70%
- Life expectancy of at least 8 weeks
- Heart function: ejection fraction of at least 40%
- Consent to continue follow-up for 3 years
Who cannot join
- Acute viral hepatitis or acute HIV infection
- Hypoxemia with SaO2 <90%
- Bilirubin >3 normal
- Creatinine >3 norms
- Pregnancy and lactation
- Life-threatening infection
- Severe (>?) pathology of the central nervous system (epilepsy, dementia, organic damage to the central nervous system)
- Karnofsky score or Lansky score <70%
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Russia1 site, 1 recruiting
- Recruiting
National medical research center of pediatric haematology, oncology and immulogy named after Dmytriy Rogachyov, Moscow, 117198
Moscow
Lena Smirnova, +7(985)130-61-03, lena.smirnova@dgoi.ru
About this study
Official title: 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).
Read the detailed description
- Infusion of ex-vivo T-depleted peripheral blood hematopoietic stem cells (CD3 depletion product)
- Infusion of donor lymphocytes enriched with T regulatory lymphocytes (CD25 selective product)
- Drug therapy (pharmacological prophylaxis of GVHD)
- Cyclosporine A
- Sirolimus o Ruxolitinib o Abatacept
- Conditions
- Acute Myeloid Leukemia, Relapsed, Acute Lymphoblastic Leukemia, High Risk, Acute Myeloid Leukemia, High Risk, Acute Lymphoblastic Leukemia, Relapse
- Being tested
- Cyclosporine A (CsA), Sirolimus, Ruxolitinib (JAKAVI®), Abatacept
- Keywords
- Acute myeloid leukemia, Acute lymphoblastic leukemia, relapse, T regulatory, CD3 depletion, allogeneic HSCT
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cyclosporine A (CsA)Drug
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).
SirolimusDrug
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
Ruxolitinib (JAKAVI®)Drug
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
AbataceptDrug
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
| Study group | Role | What they receive |
|---|---|---|
| Cyclosporine A | Active comparator | Four groups corresponding to the pharmacological prophylaxis of GVHD: 1) Cyclosporine A Receives: Drug: Cyclosporine A (CsA) |
| Sirolimus | Active comparator | Drug therapy (pharmacological prophylaxis of GVHD) 2) Sirolimus Receives: Drug: Sirolimus |
| Ruxolitinib | Active comparator | Drug therapy (pharmacological prophylaxis of GVHD) Ruxolitinib Receives: Drug: Ruxolitinib (JAKAVI®) |
| Abatacept | Active comparator | Drug therapy (pharmacological prophylaxis of GVHD) Abatacept Receives: Drug: Abatacept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
proportion of patients who received an infusion of the planned dose of regulatory T lymphocytes (at least 80%)
Measured: day 30
Safety- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Cumulative risk of GVHD Grade III-IV (target < 5%)
Measured: 120 days after HSCT
Secondary outcomes (10)
Cumulative probability of engraftment
Measured: up to 100 day
Time to engraftment of neutrophils and platelets
Measured: 100 day after HSCT
Cumulative risk of acute GVHD Grade II-IV
Measured: up to 100 days after HSCT
Cumulative risk of viral
(CMV, ADV, EBV, HHV-6) DNA detection in blood, peak viral DNA load and the duration of detectable viral DNA (each virus separately)
Measured: at 120 day after HSCT
cumulative risk of developing severe chronic GVHD
Severity of chronic GVHD
Measured: at 2 years
Cumulative risk of leukemia relapse
Measured: at 2 years
Cumulative risk of non-relapse mortality
Measured: at 100 days and 2 years
Overall survival
Measured: at 3 years
Event-free survival
Measured: at 3 years
GVHD- and relapse-free survival
Measured: at 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2026
- Study start3 Sep 2025
- Primary completion1 Jun 2027 (estimated)
- Study completion3 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia, RelapsedStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Single blind
- Who is masked
- Care provider
- Enrolment
- 64 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07366801
- Sponsor's protocol ID
- NCHPOI- 2025-14
- Status last verified
- Dec 2025
- First submitted
- 7 Jan 2026
- Last updated
- 26 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07366801
- Acronym / sponsor ID
- NCHPOI- 2025-14
- Conditions
- Acute Myeloid Leukemia, Relapsed, Acute Lymphoblastic Leukemia, High Risk, Acute Myeloid Leukemia, High Risk, Acute Lymphoblastic Leukemia, Relapse
- Keywords
- Acute myeloid leukemia, Acute lymphoblastic leukemia, relapse, T regulatory, CD3 depletion, allogeneic HSCT
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 64 participants (estimated)
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and ImmunologyUniversity, hospital or other
- Study start
- 3 Sep 2025 (actual)
- Primary completion
- 1 Jun 2027 (estimated)
- Study completion
- 3 Feb 2028 (estimated)
- First posted
- 26 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
- Infusion of ex-vivo T-depleted peripheral blood hematopoietic stem cells (CD3 depletion product)
- Infusion of donor lymphocytes enriched with T regulatory lymphocytes (CD25 selective product)
- Drug therapy (pharmacological prophylaxis of GVHD)
- Cyclosporine A
- Sirolimus o Ruxolitinib o Abatacept
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Single blind (care provider)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cyclosporine A | Active comparator | Drug: Cyclosporine A (CsA)Four groups corresponding to the pharmacological prophylaxis of GVHD: 1) Cyclosporine A |
| Sirolimus | Active comparator | Drug: SirolimusDrug therapy (pharmacological prophylaxis of GVHD) 2) Sirolimus |
| Ruxolitinib | Active comparator | Drug: Ruxolitinib (JAKAVI®)Drug therapy (pharmacological prophylaxis of GVHD) Ruxolitinib |
| Abatacept | Active comparator | Drug: AbataceptDrug therapy (pharmacological prophylaxis of GVHD) Abatacept |
Who may qualify
- Ages
- 1 year to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Informed consent signed by the patient (age 14 to 25 years) and/or his/her legal representative (age 0 to 18 years).
- The patient has an indication for allogeneic hematopoietic stem cell transplantation (HSCT) established in accordance with the current regulatory framework
- Planned HSCT from a haploidentical donor
- The Karnofsky or Lansky score is more than 70%
- Life expectancy of at least 8 weeks
Key exclusion criteria (8 listed)
- Acute viral hepatitis or acute HIV infection
- Hypoxemia with SaO2 <90%
- Bilirubin >3 normal
- Creatinine >3 norms
- Pregnancy and lactation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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). | Drug | Cyclosporine A |
| SirolimusPharmacological 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 | Drug | Sirolimus |
| 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 | Drug | Ruxolitinib |
| AbataceptPharmacological 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 | Drug | Abatacept |
Primary outcome
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%)
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%)
10 secondary outcomes
Cumulative probability of engraftment
Time frame: up to 100 day
Time to engraftment of neutrophils and platelets
Time frame: 100 day after HSCT
Cumulative risk of acute GVHD Grade II-IV
Time frame: up to 100 days after HSCT
Cumulative risk of viral
Time frame: at 120 day after HSCT
cumulative risk of developing severe chronic GVHD
Time frame: at 2 years
Cumulative risk of leukemia relapse
Time frame: at 2 years
Cumulative risk of non-relapse mortality
Time frame: at 100 days and 2 years
Overall survival
Time frame: at 3 years
Event-free survival
Time frame: at 3 years
GVHD- and relapse-free survival
Time frame: at 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Cyclosporine A (CsA), Sirolimus and Ruxolitinib (JAKAVI®) in people with Acute Myeloid Leukemia, Relapsed and Acute Lymphoblastic Leukemia, High Risk, run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology at 1 site in Russia.
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 7 inclusion and 8 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: looking for participants now, as of the sponsor's last update on 26 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 Russia. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Michael Maschan, Prof (+79166512145, mmaschan@yandex.ru), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Feasibility- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 (time frame: day 30). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 26 Jan 2026.