A Phase I/II Study of the JAK1/2 Inhibitor Ruxolitinib for Relapsed / Refractory Immune Bone Marrow Failure
National Heart, Lung, and Blood Institute (NHLBI)
Summary
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.
Description
Study Description: This is a prospective, non-randomized, phase I/II study in which participants with relapsed/refractory immune marrow failure (severe aplastic anemia, moderate aplastic anemia, single lineage cytopenias, T-LGL, and hypoplastic MDS) will be treated with the JAK1/2 inhibitor ruxolitinib. Our hypothesis is that JAK1/2 inhibition with ruxolitinib will result in hematologic improvement in participants with immune marrow failure. Objectives: The primary objectives are to assess safety and efficacy of the JAK1/2 inhibitor ruxolitinib in immune marrow failure. The secondary objec…
Eligibility
- Age range
- 18–99 years
- Sex
- All
- Healthy volunteers
- No
* INCLUSION/EXCLUSION CRITERIA: Participants of both sexes will be considered for inclusion in this study. There will be no racial, ethnic, or sex discrimination. To be eligible to participate in the treatment portion of this study, an individual must meet all of the following inclusion criteria and none of the following exclusion criteria: INCLUSION CRITERIA: ALL COHORTS: * 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 willingne…
Interventions
- DrugRuxolitinib
Subjects will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food)
Location
- National Institutes of Health Clinical CenterBethesda, Maryland