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 range2 Years

SexALL

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.

What they're measuring

Participants That Responded to Eltrombopag

Timeframe: 6 months (24 weeks +/- 14 days)

Time (Weeks) to Response

Timeframe: 6 months (24 weeks +/- 14 days)

Number of Adverse Events

Timeframe: 6 months (24 weeks +/- 14 days)

Trial details

NCT IDNCT04269889

SponsorNational Heart, Lung, and Blood Institute (NHLBI)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2022-08-16

Results submitted2023-06-16

View on ClinicalTrials.gov More Anemia, Diamond-Blackfan trials

Plain-language summary

Who can participate

Age range2 Years

SexALL

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.