Persistent ThrombocytopeniaPhase 1/2BAPTNCT07535645
Baricitinib for Post-HSCT Persistent Thrombocytopenia
A Phase 1/2 study testing Baricitinib in people with Persistent Thrombocytopenia and Allogeneic Hematopoietic Stem Cell Transplantation, run by Peking University People's Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Persistent Thrombocytopenia, Allogeneic Hematopoietic Stem Cell Transplantation
- What is being tested?
- Baricitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteChina
- Study dates
- 9 Mar 2026to 15 Sep 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking University People's Hospital
Beijing, Beijing Municipality, 100044
Yue Jin, +86-18161502996, jinyue@pku.edu.cn
About this study
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
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.
Read the 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.
- Being tested
- Baricitinib
- Keywords
- Persistent thrombocytopenia, Allogeneic Hematopoietic Stem Cell Transplantation, Baricitinib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BaricitinibDrug
Baricitinib, an orally administered, selective, reversible JAK1/2 inhibitor.
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: Drug: Baricitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events in the Ib part
The incidence and severity of adverse events are assessed using the criteria of CTCAE 5.0.
Measured: 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.
Measured: 12 weeks
Secondary outcomes (11)
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.
Measured: 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.
Measured: 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.
Measured: 24 weeks
Time to response
The time from the date of the first dose of baricitinib to the date of OR or CR.
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 24 weeks
Overall Survival (OS)
The overall survival of patients who received at least one dose of baricitinib in the study.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 104 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2026
- Study start9 Mar 2026
- Primary completion31 Dec 2027 (estimated)
- Study completion15 Sep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Persistent ThrombocytopeniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Peng Zhao, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07535645
- Sponsor's protocol ID
- 2026PHD005-BAPT
- Acronym
- BAPT
- Status last verified
- Sep 2026
- First submitted
- 6 Apr 2026
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07535645
- Acronym / sponsor ID
- BAPT / 2026PHD005-BAPT
- Keywords
- Persistent thrombocytopenia, Allogeneic Hematopoietic Stem Cell Transplantation, Baricitinib
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 28 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 9 Mar 2026 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 15 Sep 2029 (estimated)
- First posted
- 17 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Baricitinib | Experimental | Drug: BaricitinibGroup 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) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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;
Key exclusion criteria (14 listed)
- Relapse of hematologic malignancy or MRD positivity;
- Active infection;
- Active graft-versus-host disease;
- Thrombotic microangiopathy;
- Primary graft failure or poor graft function;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BaricitinibBaricitinib, an orally administered, selective, reversible JAK1/2 inhibitor. | Drug | Baricitinib |
Primary outcome
Adverse events in the Ib part
Time frame: 24 weeks
The incidence and severity of adverse events are assessed using the criteria of CTCAE 5.0.
Overall response rate (ORR) for the IIa part
Time frame: 12 weeks
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.
11 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Baricitinib in people with Persistent Thrombocytopenia and Allogeneic Hematopoietic Stem Cell Transplantation, run by Peking University People's Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Peng Zhao (+86-18810323668, zpeng702@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events in the Ib part (time frame: 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2026; AzTrials last refreshed its copy on 26 Oct 2026.