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Not yet recruiting NCT07535645

Baricitinib for Post-HSCT Persistent Thrombocytopenia

Phase I / Phase II Interventional Persistent Thrombocytopenia Allogeneic Hematopoietic Stem Cell Transplantation

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Baricitinib.
Who it may be relevant to
Registry conditions: Persistent Thrombocytopenia, Allogeneic Hematopoietic Stem Cell Transplantation. Basic parameters: 18 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

Safety and Efficacy of Baricitinib in Thrombopoietin-Receptor-Agonist-Refractory Persistent Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplantation: A Phase Ib/II Study

Overview

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.

Detailed description

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.

Interventions

  • Drug Baricitinib
    Baricitinib, an orally administered, selective, reversible JAK1/2 inhibitor.

Primary outcome measures

  • Adverse events in the Ib part [Time frame: 24 weeks]
  • Overall response rate (ORR) for the IIa part [Time frame: 12 weeks]
Secondary outcome measures (11)
  • Overall response rate (ORR) for the Ib part [Time frame: 12 weeks]
  • Complete response (CR) [Time frame: 12 weeks]
  • Durable response [Time frame: 24 weeks]
  • Time to response [Time frame: 12 weeks]
  • Bleeding events [Time frame: 24 weeks]
  • Rescue medication [Time frame: 24 weeks]
  • Adverse events in the IIa part [Time frame: 24 weeks]
  • Overall Survival (OS) [Time frame: 104 weeks]
  • Transplantation-related mortality (TRM) [Time frame: 104 weeks]
  • Relapse or progression of underlying disease [Time frame: 104 weeks]
  • Graft-versus-host disease [Time frame: 104 weeks]

Eligibility criteria

Inclusion criteria

  • Aged 18-70 years;
  • Underwent allo-HSCT;
  • Meet the diagnostic criteria for delayed platelet engraftment (DPE) or secondary failure of platelet recovery (SFPR);
  • Have platelet counts consistently <20 ×10\^9/L or transfusion-dependent within 14 days prior to enrollment;
  • Have received adequate corticosteroid and TPO-RA therapy for persistent thrombocytopenia for no less than 4 weeks, with treatment failure or intolerance;
  • Complete donor chimerism.

Exclusion criteria

  • Relapse of hematologic malignancy or MRD positivity;
  • Active infection;
  • Active graft-versus-host disease;
  • Thrombotic microangiopathy;
  • Primary graft failure or poor graft function;
  • Presence of other factors that may lead to secondary thrombocytopenia at the time of PT diagnosis;
  • History of systemic herpes zoster infection within 12 weeks prior to enrollment screening;
  • Acute or chronic infection with HBV, HCV, or HIV;
  • Evidence of active tuberculosis, or history of active tuberculosis without documented standard anti-tuberculosis treatment, or close contact with active tuberculosis without documented standard tuberculosis prophylaxis;
  • Receipt of a live vaccine within 12 weeks prior to enrollment screening, or planned receipt of a live vaccine during the study period;
  • Clinically significant thromboembolic event within 24 weeks prior to enrollment screening, or current use of anticoagulant medications deemed by the investigator to carry an uncontrollable risk;
  • Estimated glomerular filtration rate <50 mL/min/1.73 m\^2;
  • Severe pre-existing or current conditions involving the cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, nervous, or neuropsychiatric systems, or other severe or unstable illnesses or laboratory abnormalities that will make the study drug unacceptable for the patient or can interfere study data;
  • Participation in another clinical trial within 30 days prior to enrollment.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • Peking University People's Hospital — Beijing

Identifiers

NCT: NCT07535645 · 2026PHD005-BAPT

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗