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NCT05998408
JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure
Phase 1, PHASE2 trial testing Ruxolitinib in Severe Aplastic Anemia in 145 participants. Currently enrolling.
3 June 2030
Quick facts
| Lead sponsor | National Heart, Lung, and Blood Institute (NHLBI) |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 145 |
| Start date | 20 February 2024 |
| Primary completion | 3 June 2030 |
| Estimated completion | 3 June 2032 |
| Sites | 1 location across United States |
Drugs / interventions tested
- Ruxolitinib (RUXOLITINIB) — full drug profile →
Conditions studied
- Severe Aplastic Anemia — all drugs for Severe Aplastic Anemia →
- Single Lineage Cytopenias, T-LGL — all drugs for Single Lineage Cytopenias, T-LGL →
- Hypoplastic MDS — all drugs for Hypoplastic MDS →
Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Who can join
Adults 18 to 99, any sex, with Severe Aplastic Anemia or Single Lineage Cytopenias, T-LGL. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT05998408
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Related trials
Other trials of Ruxolitinib
Trials testing the same drug.
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- NCT07249346 — Dose-Expansion Study of Low Dose Post-Transplant Cyclophosphamide/Tacrolimus/Ruxolitinib for Graft-versus-Host Disease ( · Phase 2 · recruiting
- NCT07311746 — Phase Ib/II Trial of Cladribine/Ruxolitinib/Venetoclax in Patients With Relapsed/Refractory T-cell Prolymphocytic Leukem · Phase 1, PHASE2 · not yet recruiting
- NCT07340138 — Study of Pelabresib add-on to Ruxolitinib in Japanese Adult Patients With Myelofibrosis · Phase 1 · not yet recruiting
- NCT07424222 — Ruxolitinib for Immune Effector Cell Associated Hemophagocytic Lymphohistiocytosis-like Syndrome (RISE) · Phase 1 · not yet recruiting
Other recruiting trials for Severe Aplastic Anemia
Currently open trials in the same condition.
- NCT06622694 — CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia · Phase 1 · recruiting
- NCT06517641 — Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia · Phase 2 · recruiting
- NCT06695741 — Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia · Phase 2 · recruiting
- NCT06412497 — MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Se · Phase 2 · recruiting
- NCT06378060 — Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia · Phase 2 · recruiting
Other National Heart, Lung, and Blood Institute (NHLBI) trials
Trials by the same sponsor.
- NCT07566494 — Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease · Phase 1 · not yet recruiting
- NCT07137455 — EDEN Intracardiac Electrogram Recording and Classifying System · NA · enrolling by invitation
- NCT05372627 — NHLBI-Emory Advanced Cardiac CT Reconstruction · not yet recruiting
- NCT07516379 — GRAfT 2.0. A Multimodal Prospective Approach to Define the Mechanisms and Clinical Features of Acute and Chronic Rejecti · not yet recruiting
- NCT06948097 — Syk Inhibition in MItigating Lung Allograft Rejection (SIMILAR): A Trial to Evaluate the Safety and Tolerability of Fost · Phase 1 · not yet recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT05998408 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by National Heart, Lung, and Blood Institute (NHLBI)
- Last refreshed: 3 March 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05998408.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing