Eltrombopag for Treatment of Fanconi Anemia

Status Completed

Clinical Trial Summary

An open-label, phase II study to assess the efficacy and safety of eltrombopag for the treatment of children and adolescents with Fanconi anemia.

Clinical Trial Description

Open-label, phase II study to assess the efficacy and safety of eltrombopag in the treatment of patients diagnosed with Fanconi anemia who have no immediate curative treatment for their bone marrow failure (n=10). The primary objective of this open-label, phase II proof of concept study is to assess the efficacy and safety of using eltrombopag for the treatment of patients with FA before conducting a larger phase II/III study. Specific objectives: - Primary: To assess the efficacy of eltrombopag on hematopoiesis by measuring changes in peripheral platelet, hemoglobin and neutrophil counts. - Secondary: To assess the safety of eltrombopag in patients with Fanconi anemia. If the preliminary efficacy and safety results from the study are positive, we will propose its expansion to a multi-institutional Phase II/III study within the framework of the Spanish Fanconi Anemia Research Network. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT06045052
Study type	Interventional
Source	Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Contact
Status	Completed
Phase	Phase 2
Start date	December 2, 2020
Completion date	August 29, 2023

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See also
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