Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag (NCT04269889) | Clinical Trial Compass
CompletedPhase 1/2
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
United States15 participantsStarted 2020-12-01
Plain-language 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....
Who can participate
Age range
2 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Exclusion criteria
-For pediatric patients 17-years-old or younger, GFR shall be used. This can be estimated using the bedside Schwartz equation, the Counahan-Barratt method, or a similar methodology. Direct measurement including, but not limited to, 24-hour urine creatinine clearance or radiographic methods is recommended for patients with stage 3 disease (GFR less than or equal to 45 mL/min/1.73 m(2)).
-Physiologic steroid replacement for adrenal insufficiency or other similar conditions is not exclusive of trial participation
-HIV infection is not exclusive of trial participation if the infection is effectively controlled with medications not known to interfere with eltrombopag metabolism or be metabolized by pathways known to be altered by eltrombopag. HIV RNA viral load must be undetectable at the time of enrollment, and CD4 cell count must be greater than or equal to 200/microliter. Patients must remain on antiretroviral therapy throughout study participation and must be periodically monitored for suppression of viral load and CD4 cell count. If drug-drug interactions between antiretroviral medications and eltrombopag are suspected, these must be addressed by a qualified clinical pharmacist or pharmacologist, and any changes to antiretroviral therapy need to be approved in consultation with an Infectious Disease and/or HIV specialist prior to enrollment.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Participants That Responded to Eltrombopag
Timeframe: 6 months (24 weeks +/- 14 days)
2
Time (Weeks) to Response
Timeframe: 6 months (24 weeks +/- 14 days)
3
Number of Adverse Events
Timeframe: 6 months (24 weeks +/- 14 days)
Trial details
NCT IDNCT04269889
SponsorNational Heart, Lung, and Blood Institute (NHLBI)