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

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Study ID
NCT05998408
Phase
PHASE1/PHASE2
Status
Active Not Recruiting

Conditions

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

Eligibility Criteria

Sex
ALL
Age
18 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • Ruxolitinib — DRUG
    Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.

Study Details

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.

Key Dates

First listed
Aug 21, 2023
Start date
Feb 20, 2024
Status verified
Jul 2026
Primary completion
Jul 22, 2025
Completion
Jun 3, 2032

Study Design

Enrollment
13 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1: PParticipants with Severe Aplastic Anemia (SAA)
    Participants diagnosed with Severe Aplastic Anemia (SAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
  • Experimental: Cohort 2: Participants with Moderate Aplastic Anemia (MAA)
    Participants diagnosed with Moderate Aplastic Anemia (MAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
  • Experimental: Cohort 3: Participants with Unilineage Bone Marrow Failure Disorder
    Participants diagnosed with Unilineage Bone Marrow Failure Disorder will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
  • Experimental: Cohort 4: Participants with T-cell large granular lymphocyte (T-LGL) leukemia
    Participants diagnosed with T-cell large granular lymphocyte (T-LGL) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily
  • Experimental: Cohort 5: Participants with hypoplastic myelodysplastic syndrome (hMDS)
    Participants diagnosed with hypoplastic myelodysplastic syndrome (hMDS) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.

Primary Outcome Measure

Number of Participants Who Completed a Full Course of Ruxolitinib Without Discontinuation Due to Hematologic Toxicity [ Time Frame: 6 months ]

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892-

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By research site
National Institutes of Health Clinical Center· Bethesda, MD

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