Ignite Creation Date:
2025-12-24 @ 5:01 PM
Ignite Modification Date:
2025-12-28 @ 2:29 PM
Study NCT ID:
NCT07297550
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-12-22
First Post:
2025-12-09
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia
Sponsor:
Institute of Hematology & Blood Diseases Hospital, China
Organization:
Study Overview
Official Title:
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
Status Verified Date:
2025-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
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.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: