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

NCT05998408 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: 10001667001667-H
• Study Type: interventional
• Target: 145
• Locations: 1 country
• Sites: 1

Brief 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

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

Keywords

Hematologic toxicity; Ruxolitinib

Outcome Measures

Primary Outcomes (2)

• The number of patients who complete a full course of ruxolitinib without cessation is required by hematologic toxicity.

  The primary safety endpoint will be the number of patients who complete a full course of ruxolitinib without cessation required by hematologic toxicity in the 6 months following treatment initiation.

  6 months

• Overall Response Rate. Patients who develop a Complete response at 3 months and stop the drug will also be deemed responders even if they subsequently relapse.

  The primary efficacy endpoint is the OR rate by 6 months. Patients who develop CR at 3 months and stop the drug will also be deemed responders even if they subsequently relapse.

  6 months

Secondary Outcomes (8)

• Hematological response

  3, 12 months, and yearly thereafter

• Depth of response

  3, 6 months

• Rate of clonal evolution

  Variable

• Rate of relapse

  Variable

• Time to transfusion independence

  Variable

Study Arms (5)

Subjects with hypoplastic MDS

EXPERIMENTAL

Subjects are defined as patients with a diagnosis of hMDS clinically confirmed by a licensed physician or an advanced practitioner who meets the inclusion and exclusion criteria and can provide informed consent.

Drug: Ruxolitinib

Subjects with MAA

EXPERIMENTAL

Subjects are defined as patients with a diagnosis of MAA clinically confirmed by a licensed physician or an advanced practitioner who meets the inclusion andexclusion criteria and can provide informed consent.

Drug: Ruxolitinib

Subjects with PRCA

EXPERIMENTAL

Subjects are defined as patients with a diagnosis of PRCA clinically confirmed by a licensed physician or advanced practitioners who meet the inclusion and exclusion criteria and can provide informed consent.

Drug: Ruxolitinib

Subjects with SAA

EXPERIMENTAL

Subjects are defined as patients with a diagnosis of SAA clinically confirmed by a licensed physician oran advanced practitioner who meets the inclusion andexclusion criteria and can provide informed consent.

Drug: Ruxolitinib

Subjects with TLGL

EXPERIMENTAL

Subjects are defined as patients with a diagnosis of TLGL clinically confirmed by a licensed physician or advanced practitioner who meets the inclusion and exclusion criteria and can provide informed consent.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

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

Subjects with MAA; Subjects with PRCA; Subjects with SAA; Subjects with TLGL; Subjects with hypoplastic MDS

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

• 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 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)
• COHORT 1: RELAPSED/REFRACTORY SAA:
• Meet all 3 criteria below:
• Severe aplastic anemia\*:
• Bone marrow cellularity \<30% excluding lymphocytes

Sponsors & Collaborators

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

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Emma M Groarke, M.D.

  National Heart, Lung, and Blood Institute (NHLBI)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ivana Darden, R.N.

CONTACT

(301) 827-2988; ivana.darden@nih.gov

Emma M Groarke, M.D.

CONTACT

(301) 496-5093; emma.groarke@nih.gov

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 17, 2023
• First Posted: August 21, 2023
• Study Start: February 20, 2024
• Primary Completion: July 22, 2025
• Study Completion (Estimated): June 3, 2032
• Last Updated: April 24, 2026

Record last verified: 2026-04-22

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)