Trial Profile

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

Status: Completed

Phase of Trial: Phase I/II

Latest Information Update: 07 May 2025

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At a glance

Drugs Eltrombopag (Primary)
Indications Diamond-Blackfan syndrome
Focus Adverse reactions; Therapeutic Use

Most Recent Events

03 Dec 2024 Status changed from active, no longer recruiting to completed.
13 Dec 2022 Results assessing the safety and efficacy of daily, fixed-dose eltrombopag starting at 150 mg daily or pediatric weight-based equivalent for 6 months in patients with DBA, presented at the 64th American Society of Hematology Annual Meeting and Exposition.
05 Oct 2022 Planned End Date changed from 1 May 2025 to 30 Jun 2027.

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