A Phase 2 interventional study of Ruxolitinib and Mesna in Hematologic Malignancy and Myeloid Malignancy, sponsored by St. Jude Children's Research Hospital. Terminated at 1 site in United States. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2025-10-02.
Sponsored by St. Jude Children's Research Hospital · Phase 2, Interventional, and Treatment
The participants are being asked to take part in this clinical trial because the participant have a lymphoid or myeloid based cancer diagnosis that requires a bone marrow transplant.
Primary Objectives
To estimate the incidence of severe acute GVHD (saGVHD) using a prophylaxis regimen with no calcineurin inhibitors after day +60 post first allogeneic Human Leukocyte antigen (HLA)-matched sibling or unrelated donor HCT for hematological malignancies.
Secondary objective
Determine the cumulative incidence of relapse, NRM, chronic GVHD, and OS in study participants at one year post-transplant.
Exploratory objectives
The investigator propose to employ two preparative regimens based on the underlying hematological malignancy. For hematological malignancies of the lymphoid lineage we will use a standard preparative regimen consisting of Total Body Irradiation and cyclophosphamide (TBI/Cy), unless TBI is contraindicated. For myeloid malignancies we will use thiotepa, busulfan, and fludarabine (TBF), a preparative regimen that has been associated with a reduced risk of relapse and trend for improved survival with comparable NRM in comparison to busulfan and cyclophosphamide (BuCy), our current regimen. All HCT recipients will receive cyclosporine in combination with methotrexate and ruxolitinib as GVHD prophylaxis. Recipients of MUD HCT will receive rATG for additional immunosuppression as is standard for unrelated donor transplants
1,464 studies on the registry are indexed under Hematologic Neoplasms; 433 are open to participants now.
This study's enrollment of 3 is below the median of 45 across 1,068 interventional studies indexed under Hematologic Neoplasms.
Browse Hematologic Neoplasms studies →St. Jude Children's Research Hospital is the lead sponsor of 434 studies on the registry; 99 are open to participants now.
Of its 60 completed or terminated interventional studies of FDA-regulated products, 35 (58%) have results posted.
Counted across the registry records on this site, refreshed daily.
Diagnosis:
Patients must have a related or unrelated donor matched at 12 of 12 HLA alleles.
Patient must have a Karnofsky/Lansky score of 70 or higher.
Patients must be 12 years of age or older.
Patients must have a shortening fraction >26% or left ventricular ejection fraction >40%.
Patients must have bilirubin less than or equal to 2.5 mg/dL and alanine aminotransferase (ALT) less than or equal to 5 times the upper limit of normal.
Patients must have creatinine clearance, or a glomerular filtration rate (GFR), greater than 70 mL/min/1.73m2.
Patients must be free of severe infection that upon determination of principal investigator precludes BMT.
Patients must have FVC >50% predicted OR, if unable to perform pulmonary function testing, must maintain pulse oximetry oxygen saturation >92% on room air.
Female patients of childbearing age must have a negative pregnancy test.
Exclusion criteria
Drug: Ruxolitinib · Drug: Mesna · Drug: Anti-thymocyte globulin (ATG) · Drug: Cyclosporine · Drug: Cyclophosphamide · Drug: Fludarabine · Drug: Methotrexate · Radiation: Total Body Irradiation (radiation treatment) · Drug: Bone marrow infusion · Drug: Busulfan · Drug: Thiotepa
Day +40 Dosage and Route of Administration-Ruxolitinib tablets should be administered orally BID, approximately every 12 hours, continuously, Oral
Also known as: (Jakafi®)
Days -3 and -2 Dosage and Route of Administration-Mesna is dosed based on the cyclophosphamide dose and generally administered in fractionated doses at approximately 20% of the total cyclophosphamide dose, Intravenous.
Also known as: (Mesnex)
Days -3, -2 and -1 Dosage and Route of Administration-(7 mg/kg total dose); intravenous.
Also known as: (Thymoglobulin®, rabbit ATG)
Day -1 Dosage and Route of Administration-3 mg/kg; dose will be adjusted to maintain a steady concentration between 250-350 ng/mL or trough concentration between 175-250 ng/mL when transitioned to intermittent dosing.
Also known as: (Gengraf)
Days -3 and -2 Dosage and Route of Administration-60 mg/kg for 2 consecutive days, (120 mg/kg total dose); intravenous.
Also known as: (Cytoxan)
Days -4, -3 and -2 Dosage and Route of Administration-50 mg/m2 daily for 3 consecutive days (150 mg/m2 total dose) intravenous
Also known as: (Fludara)
Days +1, +3, +6 and +11 Dosage and Route of Administration-10 mg/m2/dose, intravenous
Also known as: MTX, Amethopterin
Days -7, -6, -5 and -4 6.2.3 Target Dose 1200 cGy total dose delivered 150 cGy per treatment fraction delivered BID over 4 days with 6 MV photons. Dose rate should be \< 10 cGy/min in patients treated at extended SSD (450-500 cm) in the TBI couch and will be less than 15 cGy/min in young children and infants treated at extended SSD (200-220 cm) on the floor. Intra-fraction interval should be 6 hours. Custom posteriorly placed (PA only) partial transmission lung shields (blocks) will be used to reduce the average dose to the lung to approximately 1000 cGy total dose. An additional 400 cGy supplemental testicular radiation delivered in 2 fractions of 200 cGy delivered for males with lymphoid lineage leukemia.
Also known as: TBI
Day 0
Days -4, -3 and -2 Dosage and Route of Administration-3.2 mg/kg/day; intravenous.
Also known as: Busulfex)
Days -6 and -5 Dosage and Route of Administration-(10 mg/kg total dose); intravenous
Also known as: Triplex by Immunex, TESPA, TSPA
Proportion of saGVHD Using a ProphylaxisRegimen With no Calcineurin Inhibitors After Day 100 Post First Allogeneic HLA-Matched Sibling or Unrelated Donor HCT for Hematological Malignancies.
Development of Severe Acute GVHD (saGVHD) at or before Day 100 post transplant is considered as an event. Severe acute GVHD is defined as grade II-IVGVHD. Acute graft-vs-host disease will be evaluated using the standard grading criteria.
Time frame: 100 days post transplant
Cumulative Incidence of Overall Survival (OS)
The one-year survival is defined by the participant who has not died within one year after post transplantation. The rate is calculated by computing the ratio between total number of one year survival patients and the total number of patients.
Time frame: One-year post-transplantation.
Cumulative Incidence of Relapse
Bone marrow studies for disease status evaluation will be performed at 1-year post-transplant. Testing will include a research evaluation for minimal residual disease.
Time frame: One-year post-transplantation.
Cumulative Incidence of Non Relapse Mortality (NRM)
Non-relapse mortality is death without evidence of disease relapse or progression. The rate is calculated by computing the ratio between total number of NRM patients and the total number of patients.
Time frame: One-year post-transplantation.
Cumulative Incidence of Chronic GVHD
Chronic graft-vs-host disease will be evaluated using the standard grading criteria.
Time frame: One-year post-transplantation.
| Milestone | Total Body Irradiation (TBI)/Cyclophosphamide (Cy) | Thiotepa, Busulfan, and Fludarabine (TBF) |
|---|---|---|
| Started | 2 | 1 |
| Completed | 1 | 1 |
| Not completed | 1 | 0 |
Development of Severe Acute GVHD (saGVHD) at or before Day 100 post transplant is considered as an event. Severe acute GVHD is defined as grade II-IVGVHD. Acute graft-vs-host disease will be evaluated using the standard grading criteria.
| Participants | Total Body Irradiation (TBI)/Cyclophosphamide (Cy) | Thiotepa, Busulfan, and Fludarabine (TBF) |
|---|---|---|
| Proportion of saGVHD Using a ProphylaxisRegimen With no Calcineurin Inhibitors After Day 100 Post First Allogeneic HLA-Matched Sibling or Unrelated Donor HCT for Hematological Malignancies. | 0 | 0 |
The one-year survival is defined by the participant who has not died within one year after post transplantation. The rate is calculated by computing the ratio between total number of one year survival patients and the total number of patients.
| Participants | Total Body Irradiation (TBI)/Cyclophosphamide (Cy) | Thiotepa, Busulfan, and Fludarabine (TBF) |
|---|---|---|
| Cumulative Incidence of Overall Survival (OS) | 1 | 1 |
Bone marrow studies for disease status evaluation will be performed at 1-year post-transplant. Testing will include a research evaluation for minimal residual disease.
| Participants | Total Body Irradiation (TBI)/Cyclophosphamide (Cy) | Thiotepa, Busulfan, and Fludarabine (TBF) |
|---|---|---|
| Cumulative Incidence of Relapse | 0 | 0 |
Non-relapse mortality is death without evidence of disease relapse or progression. The rate is calculated by computing the ratio between total number of NRM patients and the total number of patients.
| Participants | Total Body Irradiation (TBI)/Cyclophosphamide (Cy) | Thiotepa, Busulfan, and Fludarabine (TBF) |
|---|---|---|
| Cumulative Incidence of Non Relapse Mortality (NRM) | 0 | 0 |
Chronic graft-vs-host disease will be evaluated using the standard grading criteria.
| Participants | Total Body Irradiation (TBI)/Cyclophosphamide (Cy) | Thiotepa, Busulfan, and Fludarabine (TBF) |
|---|---|---|
| Cumulative Incidence of Chronic GVHD | 0 | 0 |
Collected over Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first year post-transplant. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| TBI/Cy Regimen | 1/2 (50%) | 2/2 (100%) | 2/2 (100%) |
| TBF Regimen | 0/1 (0%) | 1/1 (100%) | 1/1 (100%) |
| Event | TBI/Cy Regimen | TBF Regimen |
|---|---|---|
| FeverGeneral disorders | 0/2 | 1/1 |
| Sinusoidal obstruction syndromeHepatobiliary disorders | 0/2 | 1/1 |
| Neutrophil count decreasedInvestigations | 0/2 | 1/1 |
| Platelet count decreasedInvestigations | 0/2 | 1/1 |
| Multi-organ failureGeneral disorders | 1/2 | 0/1 |
| Infections and infestations - Other, specify (Disseminated Adenovirus)Infections and infestations | 1/2 | 0/1 |
| Lung infectionInfections and infestations | 1/2 | 0/1 |
| TracheitisInfections and infestations | 1/2 | 0/1 |
| Weight lossInvestigations | 1/2 | 0/1 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 1/2 | 0/1 |
| Event | TBI/Cy Regimen | TBF Regimen |
|---|---|---|
| Blood and lymphatic system disorders - Other, specify (Thrombotic Microangiopathy)Blood and lymphatic system disorders | 1/2 | 1/1 |
| Febrile neutropeniaBlood and lymphatic system disorders | 0/2 | 1/1 |
| EnterocolitisGastrointestinal disorders | 0/2 | 1/1 |
| Mucositis oralGastrointestinal disorders | 0/2 | 1/1 |
| NauseaGastrointestinal disorders | 2/2 | 1/1 |
| Cytomegalovirus infection reactivationInfections and infestations | 1/2 | 1/1 |
| Epstein-Barr virus infection reactivationInfections and infestations | 2/2 | 1/1 |
| Lung infectionInfections and infestations | 0/2 | 1/1 |
| SepsisInfections and infestations | 2/2 | 0/1 |
| Infusion related reactionInjury, poisoning and procedural complications | 2/2 | 0/1 |
Participants meeting eligibility criteria and who completed study therapy: Allogeneic Human Leukocyte antigen (HLA)-matched sibling or unrelated donor hematopoietic cell transplant (MSD/MUD HCT) for hematological malignancies. Hematological malignancies of the lymphoid lineage will use a standard preparative regimen consisting of Total Body Irradiation and cyclophosphamide (TBI/Cy), unless TBI is contraindicated. Myeloid malignancies will use thiotepa, busulfan, and fludarabine (TBF) regimen.
| Age, Continuous(years) | Total Body Irradiation (TBI)/Cyclophosphamide (Cy) | Thiotepa, Busulfan, and Fludarabine (TBF) | Total |
|---|---|---|---|
| Mean | 15.5 ± 4.95 | 17 ± 0 | 16.25 ± 3.6 |
| Sex: Female, Male(Participants) | Total Body Irradiation (TBI)/Cyclophosphamide (Cy) | Thiotepa, Busulfan, and Fludarabine (TBF) | Total |
|---|---|---|---|
| Female | 1 | 1 | 2 |
| Male | 1 | 0 | 1 |
| Ethnicity (NIH/OMB)(Participants) | Total Body Irradiation (TBI)/Cyclophosphamide (Cy) | Thiotepa, Busulfan, and Fludarabine (TBF) | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 1 | 2 |
| Not Hispanic or Latino | 1 | 0 | 1 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Total Body Irradiation (TBI)/Cyclophosphamide (Cy) | Thiotepa, Busulfan, and Fludarabine (TBF) | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 2 | 1 | 3 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.
Supporting information: Study protocol, Sap, Icf
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