NCT04328727

Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia

A Non-randomized, Open Label, Multi-center, Phase II Study to Assess the Safety and Efficacy of Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin (r-ATG) and Cyclosporine A (CsA) in East-Asian Patients With Treatment Naive Severe Aplastic Anemia (REACTS)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 36 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 6 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

This study was designed to evaluate the efficacy and safety of eltrombopag when added to r-ATG and CsA in treatment naive East-Asian adult and pediatric patients with severe aplastic anemia (SAA).

Detailed description

This was a non-randomized, open label, single arm, multi-center, Phase II study to evaluate the efficacy and safety of eltrombopag in combination with immunosuppressive therapy (IST) regimen of r-ATG + CsA in East-Asian patients with severe aplatsic anemia who had not received prior IST. Eligible participants were enrolled into the study and received eltrombopag (from Day 1 to Week 26) concomitantly with r-ATG (on Days 1-5) and CsA (from Day 1 to Week 26) in the core treatment part. Participants who were assessed as responders (meeting complete (CR) or partial (PR) response criteria) at Week 26 were eligible for the extension part of the study and continued treatment with eltrombopag and CsA after Week 26 up to Week 52. During the extension part, eltrombopag treatment was provided up to Week 52. CsA was maintained or tapered at the investigator's discretion according to local practice, with a total duration of at least 2 years (18 months after Week 26). There was a 30 days after end of treatment safety follow-up at the end of the extension part. All participants were offered to enter the long term follow-up after the discontinuation of eltrombopag, with yearly efficacy and clonal evolution assessments up to Year 3 (Week 156).

Conditions

• Severe Aplastic Anemia (SAA)

Interventions

Type	Name	Description
DRUG	eltrombopag	Tablet 25mg and 12.5mg
DRUG	rabbit anti-thymocyte globulin (r-ATG)	r-ATG 25 mg sterile lyophilized powder in 10 mL vials for IV use
DRUG	cyclosporine A (CsA)	CsA 25mg Capsule or CsA 5.0g/50mL solution for oral use

Timeline

• Start date: 2020-11-04
• Primary completion: 2022-06-10
• Completion: 2024-12-06
• First posted: 2020-03-31
• Last updated: 2026-01-13
• Results posted: 2025-07-17

Locations

12 sites across 4 countries: China, Japan, South Korea, Taiwan