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NCT04269889 · ClinicalTrials.gov registry record · Phase 1
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
A Phase 1 study of Anemia, Diamond-Blackfan, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
- 1
- Study location
NCT04269889: Completed Phase 1 study of Anemia, Diamond-Blackfan, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT04269889 is a Phase 1 study of Anemia, Diamond-Blackfan that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 15 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (75% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04269889, a Phase 1 study of Anemia, Diamond-Blackfan, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
- 1
- Study location
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....
Primary Outcome
Participants that responded to Eltrombopag as defined by: Response to treatment will be defined by one or more of the following: * Erythroid response for subjects with a pretreatment hemoglobin less than 9 G/dL will be defined as an increase in hemoglobin by \>1.5 G/dL from enrollment baseline, and/or * A reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment - compared with the pretreatment transfusion number in the previous
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 |
What the finished NCT04269889 record still lists
NCT04269889 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (75% lower).
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.
NCT04269889 reports a single indexed study location in Maryland.
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.
What interventions are being tested in trial NCT04269889?
The interventions under investigation include: Eltrombopag (DRUG).
Who is sponsoring clinical trial NCT04269889?
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 NCT04269889 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 NCT04269889's enrollment target sits among peer trials
15 1798th of 2000 higher than 157 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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.
Source ClinicalTrials.gov registry export · 2026-08-08
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