Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia
NCT ID: NCT06752694
Last Updated: 2025-09-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE2
20 participants
INTERVENTIONAL
2025-09-25
2027-06-01
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes
NCT07228624
Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis
NCT04384692
HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis
NCT06001385
Ruxolitinib Phosphate, Tacrolimus and Sirolimus in Preventing Acute Graft-versus-Host Disease During Reduced Intensity Donor Hematopoietic Cell Transplant in Patients With Myelofibrosis
NCT02528877
Dose-Expansion Study of Low Dose Post-Transplant Cyclophosphamide/Tacrolimus/Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis in Myeloablative Allogeneic Peripheral Blood Stem Cell Transplantation
NCT07249346
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
CONDITIONING REGIMEN: Patients receive fludarabine intravenously (IV) over 30 minutes once daily (QD) on days -4, -3, and -2 and undergo total body irradiation (TBI) in one or two fractions on day -1.
TRANSPLANT: Patients undergo peripheral blood stem cell (PBSC) or bone marrow transplant on day 0.
GVHD PROPHYLAXIS: Cyclosporine, Sirolimus, mycophenolate mofetil (MMF), Ruxolitinib
HUMAN LEUKOCYTE ANTIGEN (HLA)-MATCHED: Patients receive cyclosporine orally (PO) every 12 hours (Q12H) on days -3 to 96 with taper beginning on day 97 until day 180 (until day 150 for patients with unrelated donors), ruxolitinib PO twice daily (BID) or QD on day -5 to 365 and mycophenolate mofetil (MMF) PO 4-6 hours after transplant and then every 8 hours (Q8H) until day 29, then reduced to Q12H on days 30-40. Patients with unrelated donors also receive sirolimus PO QD on days -3 to 150 with taper beginning on day 151 until day 180. Patients also begin granulocyte colony-stimulating factor (G-CSF) subcutaneously (SC) on day 1 to continue until absolute neutrophil count (ANC) \> 1000/mm\^3 x 3 days. Patients also undergo multi-gated acquisition scan (MUGA)/echocardiogram (ECHO) and computed tomography (CT) during screening, as well as collection of blood samples and bone marrow aspiration and biopsy throughout the study.
HLA-MISMATCHED: Patients receive cyclosporine PO Q12 on days -3 to 150 with taper beginning on day 151 until day 180, sirolimus PO QD on days -3 to 180 with taper beginning on day 181 until day 365, ruxolitinib PO BID or QD on day -5 to 365, and MMF PO 4-6 hours after transplant and then Q8 until day 29, then reduced to Q12H on days 30-40. Patients also begin G-CSF SC on day 1 to continue until ANC \> 1000/mm\^3 x 3 days.
Patients also undergo MUGA/ECHO and CT during screening, as well as collection of blood samples and bone marrow aspiration and biopsy throughout the study.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Prevention (conditioning, transplant, GVHD prophylaxis)
See Detailed Description.
Biospecimen Collection
Undergo blood sample collection
Bone Marrow Aspiration
Undergo bone marrow biopsy and aspiration
Bone Marrow Biopsy
Undergo bone marrow biopsy and aspiration
Bone Marrow Transplantation
Undergo bone marrow transplant
Computed Tomography
Undergo CT
Cyclosporine
Given PO
Fludarabine
Given IV
Granulocyte Colony-Stimulating Factor
Given SC
Mycophenolate Mofetil
Given PO
Peripheral Blood Stem Cell Transplantation
Undergo PBSC transplantation
Ruxolitinib
Given PO
Sirolimus
Given PO
Total-Body Irradiation
Undergo TBI
Multigated Acquisition Scan
Undergo MUGA
Echocardiography
Undergo ECHO
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Biospecimen Collection
Undergo blood sample collection
Bone Marrow Aspiration
Undergo bone marrow biopsy and aspiration
Bone Marrow Biopsy
Undergo bone marrow biopsy and aspiration
Bone Marrow Transplantation
Undergo bone marrow transplant
Computed Tomography
Undergo CT
Cyclosporine
Given PO
Fludarabine
Given IV
Granulocyte Colony-Stimulating Factor
Given SC
Mycophenolate Mofetil
Given PO
Peripheral Blood Stem Cell Transplantation
Undergo PBSC transplantation
Ruxolitinib
Given PO
Sirolimus
Given PO
Total-Body Irradiation
Undergo TBI
Multigated Acquisition Scan
Undergo MUGA
Echocardiography
Undergo ECHO
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Diagnosis of severe acquired aplastic anemia defined as a bone marrow hypoplasia (\< 25% or 25-50% with \< 30% residual hematopoietic cells) shown by a biopsy and at least two of the three following criteria: absolute neutrophil count (ANC) \< 0.5×10\^9/L, platelets \< 20×10\^9/L, or absolute reticulocytes \< 40×10\^9/L or
* Non-severe acquired aplastic anemia defined as a hypocellular marrow and transfusion dependent (red cells and/or platelets)
* Does not meet World Health Organization (WHO) criteria for myelodysplastic syndrome (MDS)
* Ability to understand and the willingness to sign a written informed consent document
* Patient must be a potential hematopoietic stem cell transplant candidate as assessed by the consenting physician
* Karnofsky ≥ 70
* Calculated creatinine clearance using the Cockcroft-Gault formula or 24 hr urine creatinine clearance must be \> 60 ml/min
* Total serum bilirubin must be \< 2 mg/dL unless the elevation is thought to be due to Gilbert's disease or hemolysis
* Transaminases must be \< 3x the upper limit of normal
* Patients with clinical or laboratory evidence of liver disease will be evaluated for the cause of liver disease, its clinical severity in terms of liver function, and the degree of portal hypertension. Patients with fulminant liver failure, cirrhosis with evidence of portal hypertension or bridging fibrosis, alcoholic hepatitis, hepatic encephalopathy, or correctable hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin \> 3mg/dL, and symptomatic biliary disease will be excluded
* Diffusing capacity for carbon monoxide (DLCO) corrected \> 60% normal
* May not be on supplemental oxygen
* Left ventricular ejection fraction \> 40% OR shortening fraction \> 26%
* Patients may have received prior treatment for their AA but they are NOT required to have received immune suppression prior to consideration for transplant
Exclusion Criteria
* Patients with history of myocardial infarction (MI), cerebrovascular accident (CVA) or unprovoked pulmonary embolism (PE)/deep vein thrombosis (DVT) in past 6 months
* History of prior allogeneic transplant
* Active or recent infection without infectious disease (ID) consult and approval
* History of untreated tuberculosis (TB)
* History of HIV infection
* Pregnant or breastfeeding
* History of prior malignancy with \> 20% risk of recurrence in the next 5 years
* Patients without an HLA-identical sibling donor, 10 of 10 HLA-matched or 9 of 10 mismatched unrelated donor that meet transplant criteria
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Incyte Corporation
INDUSTRY
Fred Hutchinson Cancer Center
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Rachel B. Salit, MD
Role: PRINCIPAL_INVESTIGATOR
Fred Hutch/University of Washington Cancer Consortium
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
NCI-2024-06524
Identifier Type: REGISTRY
Identifier Source: secondary_id
20575
Identifier Type: OTHER
Identifier Source: secondary_id
RG1124040
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.