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

NCT05998408 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 145

Last updated 2026-05-14

No results posted yet for this study

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.

Conditions

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

Interventions

DRUG

Ruxolitinib

Subjects will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food)

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    lead NIH

Principal Investigators

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

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-02-20
Primary Completion
2025-07-22
Completion
2032-06-03
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05998408 on ClinicalTrials.gov