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Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
NCT04269889 · View on ClinicalTrials.gov ↗
Study Summary
Background: Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work. Other patients must get blood transfusions regularly which are time consuming and can have significant side effects. The drug eltrombopag can increase red blood cells. Researchers want to see if it can help people with DBA and, if so, for how long. Objective: To study the safety and efficacy of eltrombopag in people with DBA who have not responded to steroids or could not take them. Eligibility: People ages 2 and older with DBA who did not respond to steroids or could not take them, or their disease has returned despite taking them Design: Participants will be screened with: Medical and medicine history Physical exam MRI: Participants will lie in a machine that takes pictures of the liver. Blood and urine tests Bone marrow biopsy: A thin needle will remove a marrow sample from the participant's hip bone. Electrocardiogram Participants will take eltrombopag pills once daily for 24 weeks. They will have blood taken every 2 weeks. Participants will have visits 6 months. At 6 months, they will repeat all the screening tests and also have: Quality-of-life questionnaire Neurodevelopmental test (for participants younger than 18 years) If participants blood cell counts improve, they may keep taking eltrombopag for up to 3 more years. If so, they will have blood taken every 4 weeks. They will visit NIH every 6 months and repeat the above tests. Participants will be monitored for up to 3 years after they stop taking eltrombopag. They will visit NIH 6 months after treatment ends. If participants blood counts go down after treatment ends, they may restart the drug....
Conditions Studied
Interventions
- DRUG Eltrombopag
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center — Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2020-12-01 |
| Est. Completion | 2024-02-16 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04269889
The ClinicalTrials.gov registry entry for NCT04269889 describes a study currently listed as completed. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 15 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Heart, Lung, and Blood Institute (NHLBI), which has 381 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Anemia, Diamond-Blackfan appearing as the primary indexed condition, and to 1 intervention — of which Eltrombopag is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04269889 reports 1 study location spanning 1 distinct geographic area — top geographies include Maryland. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04269889 about?
NCT04269889 is a clinical study titled "Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag". Background: Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work. Other patients must get blood transfusions regularly which are time consuming and can have significant side effects. The drug eltrombopag can increase red blood cells. R...
What is the current status of trial NCT04269889?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2020-12-01. Estimated completion is 2024-02-16.
What conditions does trial NCT04269889 study?
This clinical trial studies the following conditions: Anemia, Diamond-Blackfan. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04269889?
The interventions under investigation include: Eltrombopag (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04269889?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 381 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04269889 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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