NCT07297550

Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia

Phase Ib/II Study to Evaluate Safety and Preliminary Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 42 (estimated)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study is designed as a Phase Ib/II trial. The phase Ib cohort will enroll patients with severe aplastic anemia (SAA) who have failed to respond to intensified immunosuppressive therapy. In contrast, the phase II cohort will include newly diagnosed and treatment-naïve patients with SAA. A Safety Review Committee (SRC), chaired by the principal investigators, will be established to oversee patient safety throughout the study. Suppose the Phase Ib results demonstrate acceptable safety and tolerability. In that case, the data will be submitted to the Ethics Committee for review, and, upon approval, the study will advance to Phase II. Phase Ib uses a 3+3 dose-escalation design with two cohorts: 150 mg golidocitinib orally every other day (low dose) or once daily (high dose). Phase II is a single-arm trial with Simon's two-stage optimal design.

Conditions

• Severe Aplastic Anemia
• Refractory Aplastic Anemia
• Newly Diagnosed Aplastic Anemia

Interventions

Type	Name	Description
DRUG	Golidocitinib Capsules	In phase Ib, the low-dose cohort will receive golidocitinib 150 mg orally every other day (qod), and the high-dose cohort will receive golidocitinib 150 mg orally once daily (qd).

Timeline

• Start date: 2025-12-15
• Primary completion: 2026-12-31
• Completion: 2027-07-01
• First posted: 2025-12-22
• Last updated: 2025-12-22