AzTrials
AzTrials

Aplastic AnemiaPhase 2NCT06752694

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

A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Aplastic Anemia, run by Fred Hutchinson Cancer Center at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Aplastic Anemia
What is being tested?
Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy
Could I receive a placebo?
No, everyone receives an active treatment
How many people
1 participants
Where
1 siteUnited States
Study dates
25 Sep 2025to 15 Oct 2026
Phase
Phase 2
Are there results?
Not yet: the study is under way

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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 15Show fewer
  • 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

Who cannot join

  • 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
Show all 9Show fewer
  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Fred Hutch/University of Washington Cancer Consortium

    Seattle, Washington, 98109

Other trials in United States

About this study

Official title: Ruxolitinib Based GVHD Prophylaxis Regimen for Older Adults Receiving Non-ATG Containing Non-Myeloablative Hematopoietic Cell Transplantation for Acquired Aplastic Anemia

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.

Read the detailed description

OUTLINE:

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
Aplastic Anemia
Being tested
Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Bone Marrow Transplantation, Computed Tomography
Medical subject headings
Anemia, Aplastic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo blood sample collection

  • Bone Marrow AspirationProcedure or surgery

    Undergo bone marrow biopsy and aspiration

  • Bone Marrow BiopsyProcedure or surgery

    Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow

    Undergo bone marrow biopsy and aspiration

  • Bone Marrow TransplantationProcedure or surgery

    Also called Blood and Bone Marrow Transplant, BMT, Bone Marrow, Bone Marrow Grafting

    Undergo bone marrow transplant

  • Computed TomographyProcedure or surgery

    Also called CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography

    Undergo CT

  • CyclosporineDrug

    Also called 27-400, Ciclosporin, CsA, CyA

    Given PO

  • FludarabineDrug

    Also called Fluradosa

    Given IV

  • Granulocyte Colony-Stimulating FactorDrug

    Also called Colony Stimulating Factor 3, Colony-Stimulating Factor (Granulocyte), Colony-Stimulating Factor 3, CSF3

    Given SC

  • Mycophenolate MofetilDrug

    Also called CellCept, MMF

    Given PO

  • Peripheral Blood Stem Cell TransplantationProcedure or surgery

    Also called PBPC transplantation, PBSCT, Peripheral Blood, Peripheral Blood Progenitor Cell Transplantation

    Undergo PBSC transplantation

  • RuxolitinibDrug

    Also called INCB 018424, INCB-018424, INCB-18424, INCB18424

    Given PO

  • SirolimusDrug

    Also called AY 22989, AY-22989, AY22989, RAPA

    Given PO

  • Total-Body IrradiationRadiation

    Also called SCT_TBI, TBI, Total Body Irradiation, Whole Body

    Undergo TBI

  • Multigated Acquisition ScanProcedure or surgery

    Also called MUGA, MUGA Scan, Radionuclide ventriculography

    Undergo MUGA

  • EchocardiographyProcedure or surgery

    Also called EC

    Undergo ECHO

Study groups in NCT06752694
Study groupRoleWhat they receive
Prevention (conditioning, transplant, GVHD prophylaxis)ExperimentalSee Detailed Description.
Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Procedure: Bone Marrow Transplantation; Procedure: Computed Tomography; Drug: Cyclosporine; Drug: Fludarabine; Drug: Granulocyte Colony-Stimulating Factor; Drug: Mycophenolate Mofetil; Procedure: Peripheral Blood Stem Cell Transplantation; Drug: Ruxolitinib; Drug: Sirolimus; Radiation: Total-Body Irradiation; Procedure: Multigated Acquisition Scan; Procedure: Echocardiography

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of grades II-IV acute graft-versus-host disease (GVHD)

    Will be estimated as simple proportions and informally compared to the historical controls at Fred Hutch. (i.e., estimates presented descriptively, but no formal statistical comparisons will be made).

    Measured: At day 100

Secondary outcomes (5)

  1. Incidence of grade III-IV acute GVHD

    Will be estimated as simple proportions and informally compared to the historical controls at Fred Hutch. (i.e., estimates presented descriptively, but no formal statistical comparisons will be made).

    Measured: At day 100

  2. Incidence of chronic GVHD

    Will be estimated as simple proportions and informally compared to the historical controls at Fred Hutch. (i.e., estimates presented descriptively, but no formal statistical comparisons will be made).

    Measured: At 1 year

  3. Incidence of primary graft failure

    Will be defined as absence of 3 consecutive days with neutrophils ≥ 500/ul combined with host CD3 peripheral blood chimerism ≥ 50% at day 42; absence of 3 consecutive days with neutrophils ≥ 500/ul under any circumstances at day 55; death after day 28 with neutrophil count <100/ul without any evidence of engraftment (< 5% donor CD3) and; primary autologous count recovery with < 5% donor CD3 peripheral blood chimerism at count recovery and without relapse.

    Measured: At 2 years

  4. Nonrelapse mortality

    Kaplan-Meier curve will be generated with point estimates and 95% confidence intervals, and Log-rank test will be conducted.

    Measured: At day 100

  5. Overall survival

    Kaplan-Meier curve will be generated with point estimates and 95% confidence intervals, and Log-rank test will be conducted.

    Measured: At 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Dec 2024
  2. Study start25 Sep 2025
  3. Primary completion23 Jan 2026
  4. Study completion15 Oct 2026 (estimated)

Resources

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
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
1 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06752694
Sponsor's protocol ID
RG1124040
Other IDs
NCI-2024-06524, 20575
Status last verified
May 2026
First submitted
23 Dec 2024
Last updated
22 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Aplastic Anemia, run by Fred Hutchinson Cancer Center at 1 site in United States.

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 15 inclusion and 9 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: under way, not looking for participants, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. 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?

This study is still under way. Results are not available in the source record yet.

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 grades II-IV acute graft-versus-host disease (GVHD) (time frame: At day 100). 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 NCT06752694.

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 22 May 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in