NCT02099747

hATG+CsA vs hATG+CsA+Eltrombopag for SAA

A Prospective Randomized Multicenter Study Comparing Horse Antithymocyte Globuline (hATG) + Cyclosporine A (CsA) With or Without Eltrombopag as Front-line Therapy for Severe Aplastic Anemia Patients.

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 202 (actual)
• Sponsor: European Society for Blood and Marrow Transplantation · Network
• Sex: All
• Age: 15 Years
• Healthy volunteers: Not accepted

Summary

The null hypothesis of no difference in CR% at 3 months between the arms will be tested against the alternative of a difference in CR% at an alpha level of .05 by assessing the odds ratio for arm yielded by this model.

Detailed description

This is a superiority trial aiming to increase the 3 month complete response rate. The sample size is calculated on the hypothesis that the experimental treatment will increase the 3 months response rate up to 21% (by 3 folds, based on the 7% reported in Scheinberg et al \[17\]). Under these assumptions, the sample size to reject the null hypothesis is n=96 patients for each treatment arm, increased by 4% for possibly not evaluable patients (total number of 200 patients, 100 each treatment arm). Statistical design for sample size calculation: increase from 7% (control arm) to 21% (investigational arm) in 3 month complete response rate (two-sided binomial test); alpha-error 0.05; power 0.8.

Conditions

• Severe Aplastic Anemia

Interventions

Type	Name	Description
DRUG	hATG
DRUG	CsA
DRUG	Eltrombopag

Timeline

• Start date: 2015-07-01
• Primary completion: 2020-12-01
• Completion: 2020-12-01
• First posted: 2014-03-31
• Last updated: 2020-12-22

Locations

29 sites across 6 countries: France, Italy, Netherlands, Spain, Switzerland, United Kingdom