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NCT05998408 · ClinicalTrials.gov registry record · Phase 1
JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure
A Phase 1 study of Severe Aplastic Anemia and Hypo-Plastic MDS, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Recruiting
- Registry status
- Phase 1
- Development phase
- 145
- Enrollment target
- 1
- Study location
NCT05998408: Recruiting Phase 1 study of Severe Aplastic Anemia and Hypo-Plastic MDS, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT05998408 is a Phase 1 study of Severe Aplastic Anemia and Hypo-Plastic MDS that is actively recruiting participants, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 145 participants, above the 118-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (23% higher). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05998408, a Phase 1 study of Severe Aplastic Anemia and Hypo-Plastic MDS, is actively recruiting participants, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 145 participants
- Enrollment target
- 1
- Study location
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.
Primary Outcome
Numbers of participants who complete a full course of ruxolitinib without discontinuation due to hematologic toxicity in the 6 months following treatment initiation. Discontinuation due to hematologic toxicity is defined as those participants that remain off drug for 6 consecutive weeks due to ongoing hematologic toxicity. Hematologic toxicity for this study will be defined as follows: * Greater than 50% increase in transfusion needs in participants who were transfusion dependent prior to ruxo
Conditions Studied
Interventions
- DRUG Ruxolitinib
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 145 participants |
| Start Date | 2024-02-20 |
| Est. Completion | 2032-06-03 |
| Phase | Phase 1 |
What NCT05998408 shows while recruiting
NCT05998408 is an interventional study that assigns participants to a tested intervention. The registered 145 participants enrollment target is mid-sized for trials with a published cap, above the 118-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (23% higher).
The record links to 3 conditions, with Severe Aplastic Anemia appearing as the primary indexed condition, and to 1 intervention - of which Ruxolitinib is the first listed.
NCT05998408 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT05998408 about?
NCT05998408 is a clinical study titled "JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure". 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...
What is the current status of trial NCT05998408?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 145 participants. The study started on 2024-02-20. Estimated completion is 2032-06-03.
What conditions does trial NCT05998408 study?
This clinical trial studies the following conditions: Severe Aplastic Anemia, Hypo-Plastic MDS, Single Lineage Cytopenias, T-LGL.
What interventions are being tested in trial NCT05998408?
The interventions under investigation include: Ruxolitinib (DRUG).
Who is sponsoring clinical trial NCT05998408?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05998408 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT05998408's enrollment target sits among peer trials
145 3rd of 16 higher than 14 of 16 other Severe Aplastic Anemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Severe Aplastic Anemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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