A Phase 1/2 interventional study of Baricitinib in Persistent Thrombocytopenia and Allogeneic Hematopoietic Stem Cell Transplantation, sponsored by Peking University People's Hospital. Recruiting at 1 site in China. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-09-16.
Sponsored by Peking University People's Hospital · Phase 1/2, Interventional, and Treatment
This is a prospective, open-label phase 1b/2 clinical trial to explore the safety and efficacy profiles of baricitinib in patients with thrombopoietin-receptor-agonist-refractory persistent thrombocytopenia after allogeneic hematopoietic stem cell transplantation.
Phase 1 part:
The phase 1b part will use a standard 3+3 design to explore the safety profiles and to establish the recommended phase 2 dose (RP2D) of baricitinib. The initial dose is 2 mg once daily, and the maximum dose is 4 mg once daily. Additional patients may be enrolled to further explore a selected dose defined by dose escalation cohorts (up to 9 patients in each dose level).
Phase 2 part:
The phase 2 part is a single-arm, open-label study to assess the efficacy and safety of baricitinib at RP2D in patients with thrombopoietin-receptor-agonist-refractory persistent thrombocytopenia after allogeneic hematopoietic stem cell transplantation. Patients in phase 1b who were treated with baricitinib at the RP2D will be included in the phase 2 efficacy endpoint analyses.
Exclusion Criteria:
Group A: Open label baricitinib at 2 mg daily (Phase 1) Group B: Open label baricitinib at 4 mg daily (Phase 1) Group C: Open label baricitinib at the RP2D (Phase 2)
Drug: Baricitinib
Baricitinib, an orally administered, selective, reversible JAK1/2 inhibitor.
Adverse events in the Ib part
The incidence and severity of adverse events are assessed using the criteria of CTCAE 5.0.
Time frame: 24 weeks
Overall response rate (ORR) for the IIa part
The proportion of patients achieving an overall response (OR), defined as a platelet count ≥20×10\^9/L maintained for more than 7 days without transfusion support. Platelet counts obtained within 4 weeks after rescue therapy were not included in the efficacy assessment.
Time frame: 12 weeks
Overall response rate (ORR) for the Ib part
The proportion of patients achieving an overall response (OR), defined as a platelet count ≥20×10\^9/L maintained for more than 7 days without transfusion support. Platelet counts obtained within 4 weeks after rescue therapy were not included in the efficacy assessment.
Time frame: 12 weeks
Complete response (CR)
The proportion of patients achieving a complete response (CR), defined as a platelet count ≥50×10\^9/L maintained for more than 7 days without transfusion support. Platelet counts obtained within 4 weeks after rescue therapy were not included in the efficacy assessment.
Time frame: 12 weeks
Durable response
The proportion of patients achieving a durable response (DR), defined as a platelet count ≥20×10\^9/L maintained for more than 8 weeks without transfusion. Platelet counts obtained within 4 weeks after rescue therapy were not included in the efficacy assessment.
Time frame: 24 weeks
Time to response
The time from the date of the first dose of baricitinib to the date of OR or CR.
Time frame: 12 weeks
Bleeding events
Clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale: 0, no bleeding; 1, petechiae; 2, mild blood loss; 3, gross blood loss; and 4, debilitating blood loss.
Time frame: 24 weeks
Rescue medication
Time and type of rescue medications, defined as any additional treatment intended to prevent bleeding or raise the platelet counts, including a dose increase of more than 10% above baseline of the concomitant medication and any additional PT-modifying agents (e.g., corticosteroids, intravenous immunoglobulin, and platelet transfusions).
Time frame: 24 weeks
Adverse events in the IIa part
Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.
Time frame: 24 weeks
Overall Survival (OS)
The overall survival of patients who received at least one dose of baricitinib in the study.
Time frame: 104 weeks
Transplantation-related mortality (TRM)
All deaths without relapse or disease progression occurring after transplantation as a direct or indirect consequence of the transplant procedure or associated complications in patients who received at least one dose of baricitinib in the study.
Time frame: 104 weeks
Relapse or progression of underlying disease
Relapse or progression of underlying disease of patients who received at least one dose of baricitinib in the study.
Time frame: 104 weeks
Graft-versus-host disease
The incidence and severity of acute graft-versus-host disease and chronic graft-versus-host disease in patients who received at least one dose of baricitinib in the study.
Time frame: 104 weeks
Plan to share: Undecided
No publications or documents are linked to this record.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Peking University People's Hospital