NCT04403321

Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 114 (actual)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 14 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

This is a randomized, open-label, phase II study to compare the efficacy of eltrombopag combined with tacrolimus to eltrombopag alone in Chinese subjects with refractory or relapsed aplastic anemia. The safety would also be evaluated. Patients would be randomized to receive eltrombopag alone or eltrombopag combined with tacrolimus. Treatment with eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day, or the best response was achieved. Tacrolimus will be given at 1mg bid with the target trough concentration of 4-10 ng/mL throughout the study. The hematological response rate and safety will be recorded and compared at 3, 6 months and 1 year after starting the study treatment (Week 13, 26 and 52).

Conditions

• Aplastic Anemia
• Drug Effect

Interventions

Type	Name	Description
DRUG	Tacrolimus	Tacrolimus will be given at 1mg bid with the target trough concentration to be 4-10 ng/mL.
DRUG	Placebo (for Tacrolimus)	placebo will be given at 1mg bid.

Timeline

• Start date: 2020-07-01
• Primary completion: 2022-07-30
• Completion: 2022-07-31
• First posted: 2020-05-27
• Last updated: 2023-01-19

Locations

1 site across 1 country: China