NCT07535645

Brief Summary

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.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
28

participants targeted

Target at P25-P50 for phase_1

Timeline
41mo left

Started Apr 2026

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress2%
Apr 2026Sep 2029

First Submitted

Initial submission to the registry

April 6, 2026

Completed
9 days until next milestone

Study Start

First participant enrolled

April 15, 2026

Completed
2 days until next milestone

First Posted

Study publicly available on registry

April 17, 2026

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2027

Expected
1.7 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 15, 2029

Last Updated

April 17, 2026

Status Verified

April 1, 2026

Enrollment Period

1.7 years

First QC Date

April 6, 2026

Last Update Submit

April 14, 2026

Conditions

Persistent ThrombocytopeniaAllogeneic Hematopoietic Stem Cell Transplantation

Keywords

Persistent thrombocytopeniaAllogeneic Hematopoietic Stem Cell TransplantationBaricitinib

Outcome Measures

Primary Outcomes (2)

  • Adverse events in the Ib part

    The incidence and severity of adverse events are assessed using the criteria of CTCAE 5.0.

    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.

    12 weeks

Secondary Outcomes (11)

  • Overall response rate (ORR) for the Ib part

    12 weeks

  • Complete response (CR)

    12 weeks

  • Durable response

    24 weeks

  • Time to response

    12 weeks

  • Bleeding events

    24 weeks

  • +6 more secondary outcomes

Study Arms (1)

Baricitinib

EXPERIMENTAL

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

Interventions

BaricitinibDRUG

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

Baricitinib

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Peking University People's Hospital

Beijing, Beijing Municipality, 100044, China

Location

MeSH Terms

Interventions

baricitinib

Central Study Contacts

Peng Zhao

CONTACT

+86-18810323668zpeng702@163.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Research Fellow

Study Record Dates

First Submitted

April 6, 2026

First Posted

April 17, 2026

Study Start

April 15, 2026

Primary Completion (Estimated)

December 31, 2027

Study Completion (Estimated)

September 15, 2029

Last Updated

April 17, 2026

Record last verified: 2026-04

Locations

CN(1)