NCT06045052

Eltrombopag for Treatment of Fanconi Anemia

An Open-label, Phase II Study to Assess the Efficacy and Safety of Eltrombopag for the Treatment of Children and Adolescents With Fanconi Anemia.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Julian Sevilla · Academic / Other
• Sex: All
• Age: 4 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Fanconi Anemia

Interventions

Type	Name	Description
DRUG	Eltrombopag	Eltrombopag tablets by mouth once daily for 24 weeks (6 months) with the dose depending on race, age and weight.

Timeline

• Start date: 2020-12-02
• Primary completion: 2023-06-19
• Completion: 2023-08-29
• First posted: 2023-09-21
• Last updated: 2023-09-21

Locations

1 site across 1 country: Spain