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Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure

This study is testing a drug called ruxolitinib for people with immune bone marrow failure, a condition where your immune system attacks your bone marrow cells. This can lead to serious blood disorders like severe aplastic anemia or hypoplastic MDS. Researchers want to see if ruxolitinib can improve blood counts and is safe to use. You might be able to join if you are an adult (18-99 years old) with one of these immune bone marrow failure conditions that has come back or hasn't responded to other treatments. The study aims to see how many people can take ruxolitinib for 6 months without serious side effects and how many show an overall improvement in their condition. The study is currently unclear on its recruitment status and plans to enroll 13 participants.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It is a non-randomized study, and the phase is not specified.
What's involved
You will take ruxolitinib by mouth twice daily for up to 6 months, with your dose gradually increasing. You will have a physical exam, give blood and saliva samples, and undergo a bone marrow biopsy.
Compensation
Not stated in the trial record.
Follow-up
The study measures outcomes at 6 months, but it is not specified how long participants are followed after treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05998408

JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure

Active, Not Recruiting
PHASE1Ages 18–99InterventionalTreatment
National Heart, Lung, and Blood Institute (NHLBI)
~13 participants
Updated 2026-08-18 on ClinicalTrials.gov
What's tested:Ruxolitinib

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Participants Who Completed a Full Course of Ruxolitinib Without Discontinuation Due to Hematologic Toxicity
Measured over 6 months
+1 more outcome measured

Conditions

Severe Aplastic Anemia
Single Lineage Cytopenias, T-LGL
Hypoplastic MDS

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Where it's being run

1 sites across 1 states
Maryland1

Study leadership

  • Emma M Groarke, M.D. · PRINCIPAL_INVESTIGATOR · National Heart, Lung, and Blood Institute (NHLBI)

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Ability of the participant or legally authorized representative (LAR) to understand and be willing to sign a written informed consent document
Age 18 or older
For females of childbearing potential, stated willingness to use an accepted method of contraception for the duration of the study. Accepted methods of contraception are:
Total abstinence
Use of an implanted or intrauterine hormonal device for at least 30 consecutive days before study drug administration
Use of oral, patch or injectable contraceptives or a vaginal hormonal device for at least 30 consecutive days before study drug infusion
Use of a non-hormonal intrauterine device for at least 30 consecutive days before study drug administration
Two barrier methods such as a diaphragm with spermicide or a condom with spermicide
For sexually active males with a female partner of childbearing potential, stated willingness to agree to use a condom with spermicide for the duration of the study.
Diagnosis of immune bone marrow failure (see specific cohort)
Bone marrow cellularity \<30% excluding lymphocytes
Absolute neutrophil count \< 0.5 x 10\^9/L
Platelet count \< 20 x 10\^9/L
Absolute Reticulocyte count \< 60 x 10\^9/L
Relapsed or refractory disease as evidenced by a course of at least 1 prior therapy.
Not suitable for transplant due to age, co-morbidities, lack of suitable donor, or participant choice.
Patients who have a documented historic diagnosis of SAA and have received an ATG-based therapy in the past and are now relapsed / refractory may be included in this cohort even if documentation of original CBC and bone marrow are unavailable.
Aplastic anemia (hypocellular bone marrow for age) with no evidence for other disease processes causing marrow failure, and depression of at least two out of three blood counts below the normal values but not fulfilling the criteria for SAA:
Absolute neutrophil count \<= l.2 x 10\^9/L
Platelet count \<= 70 x 10\^9/L
Anemia with hemoglobin \<= 9 g/dL and absolute reticulocyte count \< 60 x 10\^9/L or transfusion dependence
Relapsed or refractory disease as evidenced by a course of at least 1 prior therapy.
Erythroid lineage: Hemoglobin \<= 9 g/dL and reticulocyte count \< 60 x 10\^9/L or red cell transfusion dependence and bone marrow with absent or reduced red cell precursors
No evidence of viral or drug suppression of the marrow, T-LGL, dysplasia, or underproduction anemias secondary to B12, folate, iron or other reversible causes.
Relapsed or refractory disease as evidenced by a course of at least 1 prior therapy.
Clinical history supportive of the diagnosis of T-LGL leukemia (i.e., a history of cytopenias with peripheral blood morphologic evidence of LGLs).
Immunophenotypic studies of peripheral blood showing an increased population of T-LGLs (suggested by staining with CD3+, CD8+ and CD16+ or CD57+) or gamma-delta T cells.
Restricted or clonal rearrangement of the T-cell receptor by PCR
Relapsed or refractory disease as evidenced by a course of at least 1 prior therapy.
A diagnosis of hypoplastic MDS by WHO 2016, WHO 2022, or ICC criteria with significant cytopenias defined as:
Neutropenia: Absolute neutrophil count \< 0.5 x 10\^9/L
Thrombocytopenia: Platelet count \< 30 x 10\^9/L or platelet transfusion dependence
Anemia: Hemoglobin \< 9g/dL or red cell transfusion-dependence or absolute reticulocyte count \<60 x 10\^9/L
Relapsed or refractory disease as evidenced by a course of at least 1 prior therapy

Exclusion

Known diagnosis or high suspicion of constitutional marrow failure syndrome
Evidence of a clonal disorder with poor risk cytogenetics per R-IPSS criteria involving chromosome 7 (-7del/-7), chromosome 3 (inv 3/del3/t(3)) or three or more chromosomal abnormalities (complex)
MDS with EB-1, EB-2, AML, chronic myelomonocytic leukemia (CMML), MDS/MPN
For MDS: Has received hypomethylating agent, chemotherapy, or immunomodulatory therapy within 8 weeks prior to study entry
History of progressive multifocal leuko-encephalopathy (PML)
Infection not adequately responding to appropriate therapy
Participants with untreated or poorly controlled HIV, Hepatitis B or C
Participants with cancer who are on active chemotherapeutic treatment
Presence of severely impaired renal function defined by CrCl (as calculated by eGFR) less than 15 mL/min not requiring renal dialysis
Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during this study
Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the participant s ability to tolerate protocol therapy, or that death within 7-10 days is likely
Inability to understand the investigational nature of the study or to give informed consent or does not have a legally authorized representative or surrogate that can provide informed consent
Hypersensitivity to ruxolitinib or its components
Inability to swallow pills
Currently breastfeeding
Active non-melanoma skin cancer
Acute thrombosis (myocardial infarction, ischemic heart disease requiring stents, stroke, pulmonary embolism, or deep venous thrombosis) within the last 6 months
Patients with a PNH clone \>50% who are not taking anticoagulation or anticomplement therapy

What this trial measures

  • Number of Participants Who Completed a Full Course of Ruxolitinib Without Discontinuation Due to Hematologic Toxicity6 months

    Numbers of participants who complete a full course of ruxolitinib without discontinuation due to hematologic toxicity in the 6 months following treatment initiation. Discontinuation due to hematologic toxicity is defined as those participants that remain off drug for 6 consecutive weeks due to ongoing hematologic toxicity. Hematologic toxicity for this study will be defined as follows: * Greater than 50% increase in transfusion needs in participants who were transfusion dependent prior to ruxolitinib therapy, lasting for more than 12 weeks * Need for any transfusion for more than 12 weeks in participants who were transfusion independent prior to ruxolitinib therapy. This excludes transfusions given for Hb \>7g/dL or platelets \>10 x 109 or those given for procedures. * Worsening in peripheral cytopenias \>50% compared to pre-treatment levels in participants with a pre-treatment ANC \>500 or platelets \>50 Drop in ANC to \<200 in participants with a pre-treatment ANC \<500

  • Number of Participants Who Achieved an Overall Response6 months

    Participants who had a CR at 3 months and discontinued study drug were considered responders, even if they subsequently relapsed. • Cohort 1: Response: No Camitta SAA criteria; ≥2 of ANC ≥0.5 × 10⁹/L, platelets ≥20 × 10⁹/L, reticulocytes ≥60 × 10⁹/L on 2 counts ≥1 week apart Complete Response (CR): ANC ≥1 × 10⁹/L, platelets ≥100 × 10⁹/L, Hb ≥10 g/dL Partial Response (PR): Response but not CR • Cohorts 3: Response: ≥1 evaluable lineage response Erythroid: Hb ↑ \>1.5 g/dL, ≥4 fewer RBC transfusions/8 weeks, or reticulocytes \>60 × 10⁹/L Platelet: ↑ ≥30 × 10⁹/L if baseline ≥20 × 10⁹/L, or \<20 to \>20 × 10⁹/L and ≥100% ↑ Neutrophil: ≥100% ↑ and absolute ↑ \>0.5 × 10⁹/L CR: ANC ≥1 × 10⁹/L, platelets ≥100 × 10⁹/L, Hb ≥10 g/dL • Cohort 4: Response: ≥1 evaluable lineage response CHR: ANC \>1.5 × 10⁹/L, platelets \>150 × 10⁹/L, lymphocytes \<4 × 10⁹/L PHR: Improvement in ≥1 affected parameter but not CHR CMR: No clonal T-cell detection and CHR CR: CHR and CMR