Graft-versus-host-diseasePhase 2PTCyRuxoNCT04669210
PTCy and Ruxolitinib vs PTCy, Tacrolimus and MMF in MUD and Haploidentical HSCT
A Phase 2 study testing Ruxolitinib, Tacrolimus and Mycophenolate Mofetil in people with Graft-versus-host-disease and Stem Cell Transplant Complications, run by St. Petersburg State Pavlov Medical University at 2 sites in Russia.
Trial summary
This is multicenter investigator-initiated randomized open-label phase II clinical trial to compare prophylaxis of graft versus host disease treated with tacrolimus and mycophenolate mofetil versus ruxolitinib after post-transplant cyclophosphamide. In total 128 patients will be included in the study. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Graft-versus-host-disease, Stem Cell Transplant Complications, Acute Myeloid Leukemia
- What is being tested?
- Ruxolitinib, Tacrolimus and Mycophenolate Mofetil
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 128 participants
- Where
- 2 sitesRussia
- Study dates
- 3 Nov 2020to 28 Mar 2025
- Phase
- Phase 2
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Informed consent to participate in the study, signed by the patient;
- Diagnosis: acute lymphoblastic or acute myeloblastic leukemia;
- Morphological remission, defined as less than 5% of blasts by microscopy or flow cytometry with a peripheral leukocyte level of more than 1.500 μL. It is acceptable to include patients without restored platelets or erythrocytes;
- Indications for performing allogeneic hematopoietic stem cell transplantation, determined by the participating center in accordance with local medical practice;
- Unrelated or haploidentical donor;
- Age 18-70 years;
- Functional status according to ECOG scale 0-2 score.
Who cannot join
- Repeated allogeneic transplantation, regardless of the indications for its implementation;
- Source of graft - umbilical cord stem cells;
- Any ex vivo modification of the graft with the exception of separation or washing of red blood cells;
- The presence of more than 5% of clonal tumor cells according to flow cytometry in the presence of morphological remission;
- Diagnosis: acute promyelocytic leukemia;
- Severe organ failure: creatinine more than 2 ULN; ALT, AST more than 5 ULN; bilirubin more than 1.5 ULN; respiratory failure more than 1 grade;
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- Unstable hemodynamics, requiring the introduction of vasopressors;
- Uncontrolled bacterial or fungal infection at the time of randomization, determined by the level of CRP> 70 mg/l with adequate antibacterial or antifungal therapy;
- Rhythm disturbances that persist despite adequate antiarrhythmic therapy: a tachysystolic form of atrial fibrillation, ventricular arrhythmias V gradation according to Laun, AV block of III degree;
- Decrease in ejection fraction according to echocardiography less than 40%;
- Angina of more than II functional class or unstable angina;
- Another severe concomitant pathology, which according to the attending physician does not allow the patient to be included in the study;
- Pulmonary pathology with a decrease in FEV1 of less than 60% or pulmonary diffusion capacity of less than 60%;
- Inability to quit smoking for up to 6 months after transplantation;
- Pregnancy or refusal to perform highly effective contraception for 6 months after transplantation.
- Highly effective contraceptive methods include:
- Total abstinence: if it corresponds to the preferred and customary way of life of the patient. Periodic abstinence (for example, calendar, ovulation, symptothermal, postovulation methods) and interrupted sexual intercourse are not considered acceptable methods of contraception;
- Female sterilization (surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy or tubal ligation at least 6 weeks before the start of the therapy being studied. In the case of ovariectomy only, the reproductive status of the woman must be confirmed using a subsequent analysis of hormones;
- Sterilization of the male partner (at least 6 months before screening). For women participating in the study, the sexual partner after a vasectomy should be the only partner;
- Use of oral, injectable or implanted hormonal contraceptive drugs, intrauterine devices or contraceptive systems, or other forms of hormonal contraception with similar efficacy (failure rate less than 1%), for example, hormonal vaginal rings or transdermal hormonal contraceptives.
- Somatic or mental pathology not allowing to sign informed consent.
Trial locations and contacts
2 sites in 1 country.
Russia2 sites
National Hematology Research Center
Moscow, 125167
RM Gorbacheva Research Institute
Saint Petersburg, 197022
About this study
Official title: A Randomized, Multicenter, Open-label Phase II Trial to Compare Prophylaxis of Graft Versus Host Disease With Tacrolimus and Mycophenolate Mofetil Versus Ruxolitinib After Post-transplant Cyclophosphamide
This is multicenter investigator-initiated randomized open-label phase II clinical trial to compare prophylaxis of graft versus host disease treated with tacrolimus and mycophenolate mofetil versus ruxolitinib after post-transplant cyclophosphamide.
In total 128 patients will be included in the study. After inclusion into the study and performing of transplantation patients will be randomized in 1:1 proportion in two arms (64 patients per arm): arm A will include patients who will be treated with cyclophosphamide and ruxolitinib for GVHD prophylaxis; arm B will include patients who will be treated with cyclophosphamide, tacrolimus and MMF for GVHD prophylaxis. After the end of the treatment patients will be followed-up during two years.
- Conditions
- Graft-versus-host-disease, Stem Cell Transplant Complications, Acute Myeloid Leukemia, Acute Lymphoid Leukemia
- Being tested
- Ruxolitinib, Tacrolimus, Mycophenolate Mofetil
- Keywords
- Graft-versus-host-disease, Prophylaxis, ruxolitinib, tacrolimus, MMF, PTCY, cyclophosphamide, unrelated donor, haploidentical donor, randomized trial
- Medical subject headings
- Graft vs Host Disease, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Also called Jakavi
Ruxolitinib administered during conditioning 5 mg tid before allogeneic hematopoietic stem cell transplantation, 5 mg tid days 5-21 and 5 mg bid days 22-150 after transplantation instead of tacrolimus and MMF.
TacrolimusDrug
Also called Prograf
Tacrolimus 0.03 mg/kg adjusted to concentrations 5-15 ng/ml from day+5 to +100
Also called CellCept
Mycophenolate mofetil 30 mg/kg from day +5 to +35
| Study group | Role | What they receive |
|---|---|---|
| PTCY tacrolimus MMF | Active comparator | Conditioning:
fludarabine 180 mg/m2 busulfan 8-14 mg/kg per os
GVHD prophylaxis:
cyclophosphamide 50 mg/kg day+3, +4 tacrolimus 0.03 mg/kg from day+5 to 100 mycophenolate mofetil 30 mg/kg from day+5 to 35 Receives: Drug: Tacrolimus; Drug: Mycophenolate Mofetil |
| PTCY ruxolitinib | Experimental | Conditioning:
fludarabine 180 mg/m2 busulfan 8-14 mg/kg per os ruxolitinib 5 mg tid days -7 to -2
GVHD prophylaxis:
cyclophosphamide 50 mg/kg day+3, +4 ruxolitinib 5 mg tid days +5 to +21 ruxolitinib 5 mg bid days +22 to +150 Receives: Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of acute GVHD grade II-IV
Proportion of patients with acute GVHD II-IV grade
Measured: 125 days
Secondary outcomes (8)
Non-relapse mortality
Cumulative incidence of patients with mortality without hematological relapse of malignancy
Measured: 2 years
Relapse incidence
Cumulative incidence of patients with relapse
Measured: 2 years
Incidence of moderate and severe chronic GVHD
Cumulative incidence of patients with moderate and severe chronic GVHD according to NIH 2015 criteria
Measured: 2 years
Overall survival
Kaplan-Meier estimate of death from all causes
Measured: 2 years
Event-free survival
Kaplan-Meier estimate of death or relapse
Measured: 2 years
Incidence of HSCT-associated adverse events (safety and toxicity)
Toxicity assessment is based on NCI CTC AE 5.0 grades. Veno-occlusive disease incidence and severity assessment is based on EBMT criteria 2016. Transplant-associated microangiopathy incidence assessment is based on Cho et al. criteria. All toxicity measurements will be aggregated as severity scores.
Measured: 125 days
Primary or secondary graft failure
Cumulative incidence of patients with primary or secondary graft failure defined by the absence of donor chimerism.
Measured: 2 years
Incidence of infections
Number of patients with bacteremia before engraftment, bacteremia after engraftment, severe sepsis (presence of multiple organ failure), pneumonia, soft tissue infection, invasive mycosis (probable or proven invasive aspergillosis, candidaemia, zygomycosis), reactivation of cytomegalovirus, other opportunistic viral infections
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2020
- Study start3 Nov 2020
- Primary completion27 Feb 2023
- Study completion28 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Graft-versus-host-diseaseStudies 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
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 128 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Petersburg State Pavlov Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ivan S Moiseev, St. Petersburg State Pavlov Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04669210
- Sponsor's protocol ID
- CINC424ARU01T
- Acronym
- PTCyRuxo
- Status last verified
- Jul 2025
- First submitted
- 26 Nov 2020
- Last updated
- 4 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Publications
Moiseev I, Drokov M, Kitaeva Y, Rudakova T, Barabanshchikova M, Chernyshova D, Bondarenko S, Kuzmina L, Rodionov G, Svetkina E, Lapin S, Kholopova I, Morozova E, Vasilyeva V, Vlasova Y, Zhogolev D, Konstantinova T, Parovichnikova E, Kulagin A. A randomized, multicenter phase II trial to compare prophylaxis of graft versus host disease with tacrolimus and mycophenolate mofetil versus ruxolitinib after post-transplant cyclophosphamide. Transplant Cell Ther. 2026 Apr 30:S2666-6367(26)00333-7. doi: 10.1016/j.jtct.2026.04.042. Online ahead of print.
PubMed 42066960
Study at a glance
From the registry record, last updated by the sponsor on 4 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Multicenter, Open-label Phase II Trial to Compare Prophylaxis of Graft Versus Host Disease With Tacrolimus and Mycophenolate Mofetil Versus Ruxolitinib After Post-transplant Cyclophosphamide
- ClinicalTrials.gov ID
- NCT04669210
- Acronym / sponsor ID
- PTCyRuxo / CINC424ARU01T
- Conditions
- Graft-versus-host-disease, Stem Cell Transplant Complications, Acute Myeloid Leukemia, Acute Lymphoid Leukemia
- Keywords
- Graft-versus-host-disease, Prophylaxis, ruxolitinib, tacrolimus, MMF, PTCY, cyclophosphamide, unrelated donor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 128 participants (actual)
- Lead sponsor
- St. Petersburg State Pavlov Medical UniversityUniversity, hospital or other
- Study start
- 3 Nov 2020 (actual)
- Primary completion
- 27 Feb 2023 (actual)
- Study completion
- 28 Mar 2025 (actual)
- First posted
- 16 Dec 2020 (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.
This is multicenter investigator-initiated randomized open-label phase II clinical trial to compare prophylaxis of graft versus host disease treated with tacrolimus and mycophenolate mofetil versus ruxolitinib after post-transplant cyclophosphamide.
In total 128 patients will be included in the study. After inclusion into the study and performing of transplantation patients will be randomized in 1:1 proportion in two arms (64 patients per arm): arm A will include patients who will be treated with cyclophosphamide and ruxolitinib for GVHD prophylaxis; arm B will include patients who will be treated with cyclophosphamide, tacrolimus and MMF for GVHD prophylaxis. After the end of the treatment patients will be followed-up during two years.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PTCY tacrolimus MMF | Active comparator | Drug: Tacrolimus, Drug: Mycophenolate MofetilConditioning: fludarabine 180 mg/m2 busulfan 8-14 mg/kg per os GVHD prophylaxis: cyclophosphamide 50 mg/kg day+3, +4 tacrolimus 0.03 mg/kg from day+5 to 100 mycophenolate mofetil 30 mg/kg from day+5 to 35 |
| PTCY ruxolitinib | Experimental | Drug: RuxolitinibConditioning: fludarabine 180 mg/m2 busulfan 8-14 mg/kg per os ruxolitinib 5 mg tid days -7 to -2 GVHD prophylaxis: cyclophosphamide 50 mg/kg day+3, +4 ruxolitinib 5 mg tid days +5 to +21 ruxolitinib 5 mg bid days +22 to +150 |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Informed consent to participate in the study, signed by the patient;
- Diagnosis: acute lymphoblastic or acute myeloblastic leukemia;
- Morphological remission, defined as less than 5% of blasts by microscopy or flow cytometry with a peripheral leukocyte level of more than 1.500 μL. It is acceptable to include patients without restored platelets or erythrocytes;
- Indications for performing allogeneic hematopoietic stem cell transplantation, determined by the participating center in accordance with local medical practice;
- Unrelated or haploidentical donor;
Key exclusion criteria (21 listed)
- Repeated allogeneic transplantation, regardless of the indications for its implementation;
- Source of graft - umbilical cord stem cells;
- Any ex vivo modification of the graft with the exception of separation or washing of red blood cells;
- The presence of more than 5% of clonal tumor cells according to flow cytometry in the presence of morphological remission;
- Diagnosis: acute promyelocytic leukemia;
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 |
|---|---|---|
| RuxolitinibAlso: JakaviRuxolitinib administered during conditioning 5 mg tid before allogeneic hematopoietic stem cell transplantation, 5 mg tid days 5-21 and 5 mg bid days 22-150 after transplantation instead of tacrolimus and MMF. | Drug | PTCY ruxolitinib |
| TacrolimusAlso: PrografTacrolimus 0.03 mg/kg adjusted to concentrations 5-15 ng/ml from day+5 to +100 | Drug | PTCY tacrolimus MMF |
| Mycophenolate MofetilAlso: CellCeptMycophenolate mofetil 30 mg/kg from day +5 to +35 | Drug | PTCY tacrolimus MMF |
Primary outcome
Incidence of acute GVHD grade II-IV
Time frame: 125 days
Proportion of patients with acute GVHD II-IV grade
8 secondary outcomes
Non-relapse mortality
Time frame: 2 years
Relapse incidence
Time frame: 2 years
Incidence of moderate and severe chronic GVHD
Time frame: 2 years
Overall survival
Time frame: 2 years
Event-free survival
Time frame: 2 years
Incidence of HSCT-associated adverse events (safety and toxicity)
Time frame: 125 days
Primary or secondary graft failure
Time frame: 2 years
Incidence of infections
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jul 2025.
- Derived from this study: PubMed 42066960Moiseev I, Drokov M, Kitaeva Y, Rudakova T, Barabanshchikova M, Chernyshova D, Bondarenko S, Kuzmina L, Rodionov G, Svetkina E, Lapin S, Kholopova I, Morozova E, Vasilyeva V, Vlasova Y, Zhogolev D, Konstantinova T, Parovichnikova E, Kulagin A. A randomized, multicenter phase II trial to compare prophylaxis of graft versus host disease with tacrolimus and mycophenolate mofetil versus ruxolitinib after post-transplant cyclophosphamide. Transplant Cell Ther. 2026 Apr 30:S2666-6367(26)00333-7. doi: 10.1016/j.jtct.2026.04.042. Online ahead of print.
- Individual participant dataSharing planned: Yes
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ruxolitinib, Tacrolimus and Mycophenolate Mofetil in people with Graft-versus-host-disease and Stem Cell Transplant Complications, run by St. Petersburg State Pavlov Medical University at 2 sites 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 21 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 4 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 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.
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?
Incidence of acute GVHD grade II-IV (time frame: 125 days). 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 NCT04669210, 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 4 Jul 2025.