New drug combo aims to make bone marrow transplants safer for older aplastic anemia patients
NCT ID NCT06752694
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tests whether adding the drug ruxolitinib to standard care can prevent graft-versus-host disease (GVHD) in older adults with acquired aplastic anemia who receive a bone marrow transplant. GVHD is a common and serious complication where donor immune cells attack the recipient's body. The study enrolls patients over 40 or those unable to tolerate standard treatments, and aims to see if this approach lowers the risk of moderate to severe GVHD.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Ruxolitinib
- What this could lead to
- If successful, this could offer a safer way for older adults with aplastic anemia to receive a bone marrow transplant with a lower risk of serious graft-versus-host disease.
- What could go wrong
- This is a very early phase 2 trial with only one participant enrolled so far, so results are highly preliminary. Ruxolitinib may not reduce GVHD as hoped, and there are always risks of infection or other side effects from the drug and transplant.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
1 person
The number who actually took part.
- Started
-
Sep 2025
- Expected to finish
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Oct 2026
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Age \> 40 years or ages 18 - 40 years with Hematopoietic Cell Transplantation - Specific Comorbidity Index (HCT-CI) score \> 3 necessitating a low intensity transplant or determined inability to tolerate antithymocyte globulin (ATG) * 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: * Contraindication to receiving ruxolitinib including: patients who have known hypersensitivity to JAK inhibitors and excipients * 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Half-Matched stem cells tested as a cure for severe aplastic anemia
- Can a pill revive bone marrow in aplastic anemia?
- Can a drug revive blood cell production in children with aplastic anemia?
- Could a platelet-boosting drug shorten recovery after stem cell transplants?
- A common antioxidant may help platelets recover faster after stem cell transplant
- Could a cancer antibody reboot blood production in aplastic anemia?