Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT06752694

Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia

Led by Fred Hutchinson Cancer Center · Updated on 2026-03-12

20

Participants Needed

1

Research Sites

118 weeks

Total Duration

On this page

Sponsors

F

Fred Hutchinson Cancer Center

Lead Sponsor

I

Incyte Corporation

Collaborating Sponsor

AI-Summary

What this Trial Is About

This phase II trial tests how well a ruxolitinib-based graft versus host disease (GVHD) prevention (prophylaxis) regimen works before, during, and after bone marrow/stem cell transplantation (hematopoietic cell transplantation \[HCT\]) in patients with acquired aplastic anemia. Acquired aplastic anemia (AA) is a condition in which the bone marrow is unable to produce blood cells. Affected patients typically present with infections due to abnormally low number of neutrophils, bleeding due to low platelet count, and/or fatigue due to a lower-than-normal number of red blood cells (anemia). Its incidence varies with age, occurring most frequently in patients aged 2-5 years, 20-25 years, and 55 years and older. Treatment of AA includes either immunosuppressive therapy (IST) or bone marrow/stem cell transplantation (HCT) with first-line therapy in younger adults often being HCT, while adults over 40 still frequently trial IST first due to the morbidity and mortality concerns with HCT. GVHD is a common complication after donor stem cell transplantation, resulting from donor immune cells recognizing recipients' cells and attacking them. Ruxolitinib, a drug in a class of oral medications called JAK inhibitors has been approved for the treatment of acute and chronic GVHD. It has also been shown to decrease GVHD when used in the prevention setting in patients with myelofibrosis. The current study aims to assess whether adding ruxolitinib to a standard GVHD prevention regimen may reduce the risk of Grade II-IV acute and chronic GVHD after bone marrow/stem cell transplantation in older patients with acquired aplastic anemia.

CONDITIONS

Official Title

Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age over 40 years or ages 18 to 40 with a specific transplant comorbidity score above 3 or inability to tolerate antithymocyte globulin (ATG)
  • Diagnosis of severe acquired aplastic anemia with bone marrow hypoplasia and at least two criteria: low neutrophils, low platelets, or low reticulocytes
  • Diagnosis of non-severe acquired aplastic anemia with hypocellular marrow and transfusion dependence
  • Does not meet World Health Organization criteria for myelodysplastic syndrome
  • Ability and willingness to sign informed consent
  • Potential candidate for hematopoietic stem cell transplant as determined by a physician
  • Karnofsky performance score of 70 or higher
  • Creatinine clearance over 60 ml/min
  • Total serum bilirubin below 2 mg/dL unless due to Gilbert's disease or hemolysis
  • Transaminases less than three times the upper limit of normal
  • Evaluation and acceptable status for liver disease without severe liver failure or complications
  • Diffusing capacity for carbon monoxide corrected above 60% of normal
  • Not on supplemental oxygen
  • Left ventricular ejection fraction above 40% or shortening fraction above 26%
  • Prior treatment for aplastic anemia allowed but not required before transplant
Not Eligible

You will not qualify if you...

  • Known allergy or hypersensitivity to ruxolitinib or similar drugs
  • History of heart attack, stroke, or unprovoked pulmonary embolism or deep vein thrombosis within the past 6 months
  • Previous allogeneic transplant
  • Active or recent infection without infectious disease specialist approval
  • Untreated tuberculosis
  • HIV infection
  • Pregnant or breastfeeding
  • History of cancer with more than 20% risk of returning within 5 years
  • Lack of a suitable HLA-identical sibling donor or a matching unrelated donor meeting transplant criteria

AI-Screening

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Trial Site Locations

Total: 1 location

1

Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, United States, 98109

Actively Recruiting

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Research Team

R

Rachel B. Salit, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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