NCT07522996

Enarodustat+CsA vs CsA in the Treatment of Newly-diagnosed TD-NSAA

Enarodustat Plus CsA Versus CsA Monotherapy in the Treatment of Newly-diagnosedTD-NSAA: a Single-center Randomized Trial

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 90 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study aimed to compare the efficacy and safety of enarodustat combined with cyclosporine versus cyclosporine alone in the treatment of TD-NSAA.

Detailed description

For TD-NSAA patients without HLA-matched donors, the firstline therapy is immunosuppressive therapy (IST) combined with thrombopoietin receptor agonists (TPO-RAs). However, some patients only have partial hematological responses, and their hemoglobin levels cannot be effectively increased. Inflammatory factors such as IFN-γ, TNF-α, and IL-6 are significantly elevated in the bone marrow and peripheral blood of AA patients. Even in patients who respond effectively to IST, the proportion of CD3+ IFN+ and CD3+ TNF+ lymphocytes is still higher than that in healthy controls, and these cytokines may be involved in inhibiting the recovery of hemoglobin in patients. HIF-PHI prevent the hydroxylation of HIF-α, thereby enabling the transcription and expression of genes related to erythropoiesis, such as those related to erythropoietin and iron transport. Roxadustat and enarodustat have been approved for the treatment of anemia in chronic kidney disease. Studies have shown that roxadustat can significantly reduce the levels of inflammatory factors such as IFN-γ, TNF-α, and IL-6 in the serum of patients with chronic kidney disease. A preliminary study has shown that roxadustat monotherapy in patients with insufficient erythroid response after IST can achieve a red blood cell response rate of 71.4%. Enarodustat has a similar mechanism to roxadustat, and in a rat model of inflammatory anemia, it was found that enarodustat can stimulate erythropoiesis by increasing iron utilization and improving inflammatory anemia. However, there are currently no studies on the use of enarodustat in AA patients. Thus, this study aims to conduct a single-center, prospective, randomized controlled trial to compare the efficacy and safety of CsA+enarodustat with CsA monotherapy in newly diagnosed TD-NSAA patients.

Conditions

• Transfusion-dependent Non-severe Aplastic Anemia

Interventions

Type	Name	Description
DRUG	Enarodustat	Enarodustat 8mg qd
DRUG	Cyclosporin (CSA)	CsA 3-5mg/kg/d

Timeline

• Start date: 2026-04-01
• Primary completion: 2028-12-01
• Completion: 2029-12-01
• First posted: 2026-04-13
• Last updated: 2026-04-13