Actively Recruiting
JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-05-14
145
Participants Needed
1
Research Sites
432 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Immune bone marrow failure is a condition that occurs when a person s immune system attacks the cells of the bone marrow. This can lead to diseases including different types of anemias and blood cancers. Some of these diseases can be deadly. Better treatments are needed. Objective: To test a drug (ruxolitinib) in people with different types of immune bone marrow failure. Eligibility: Adults aged 18 and older with an immune bone marrow failure. Design: Participants will be screened. They will have a physical exam. They will give samples of blood and saliva. They will have a bone marrow biopsy: A large needle will be inserted into a small cut to remove a sample of the soft tissue inside the bone. Some participants may have a skin biopsy: A small piece of skin will be removed. Some may have a computed tomography (CT) scan: They will lie on a table that slides into a donut-shaped machine that uses X-rays to make pictures of the inside of the body. Ruxolitinib is a tablet taken by mouth. Participants will take the drug twice a day for up to 6 months. Participants will have blood tests every week while they are taking the drug. These tests can be done by the participant s own physician and the results sent to the researchers. Participants will have clinic visits after taking the drug for 3 months and 6 months and then after 1, 2, and 3 years. The blood tests and bone marrow biopsy will be repeated. Participants who improve while taking the drugs may go on to an extension phase of the study.
CONDITIONS
Official Title
JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ability to understand and sign informed consent or have a legally authorized representative
- Age 18 years or older
- For females of childbearing potential, willingness to use accepted contraception methods during the study
- For sexually active males with female partners of childbearing potential, willingness to use condoms with spermicide during the study
- Diagnosis of immune bone marrow failure
- Relapsed or refractory disease with at least one prior therapy
- Specific blood count and bone marrow criteria depending on cohort (severe aplastic anemia, moderate aplastic anemia, unilineage bone marrow failure, T-LGL leukemia, or hypoplastic MDS)
- Not suitable for transplant due to age, co-morbidities, donor availability, or participant choice
You will not qualify if you...
- Known or suspected constitutional marrow failure syndrome
- High-risk clonal disorders with specific poor-risk cytogenetics
- MDS with EB-1, EB-2, AML, CMML, or MDS/MPN
- Recent treatment with hypomethylating agents, chemotherapy, or immunomodulatory therapy within 8 weeks
- History of progressive multifocal leukoencephalopathy
- Active infection not responding to therapy
- Untreated or poorly controlled HIV, Hepatitis B or C
- Active cancer on chemotherapy
- Severe renal impairment (eGFR less than 15 mL/min) not on dialysis
- Current pregnancy or unwillingness to use birth control if of childbearing potential
- Severe concurrent disease likely to limit ability to tolerate treatment or cause death within 7-10 days
- Inability to give informed consent or lack of legally authorized representative
- Allergy to ruxolitinib or its components
- Inability to swallow pills
- Currently breastfeeding
- Active non-melanoma skin cancer
- Acute thrombosis within the last 6 months
- PNH clone greater than 50% without anticoagulation or anticomplement therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
I
Ivana Darden, R.N.
CONTACT
E
Emma M Groarke, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
5
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