TPO-RA Plus Baricitinib vs. TPO-RA for ITP

TPO-RA Plus Baricitinib vs. TPO-RA in Patients With ITP : A Randomized, Open-label Trial

This is a prospective, randomized, controlled trial. ITP patients who failed prior full-does TPO-RA monotheray for 14 days. Patients are randomly assigned at a 1:1 ratio to receive baricitinib plus TPO-RA or TPO-RA alone. Patients are randomly assigned at a 1:1 ratio to receive baricitinib plus TPO-RA or TPO-RA alone. The primary endpoint was the 14-day overall response rate without any rescue therapy.

Study Overview

• Status: Not yet recruiting
• Conditions: ITP - Immune Thrombocytopenia
• Intervention / Treatment: Drug: Baricitinib; Drug: TPO-RA

Detailed Description

This is a prospective, randomized, controlled trial.Eligible patients were at least 18 years old, had a diagnosis of primary ITP and did not respond after receiving TPO-RA (hetrombopag or eltrombopag) at the full dose (hetrombopag 7.5mg per day or eltrombopag 75 mg per day) for 14 days (platelet count below 30×10^9/L or a value less than a 2-fold increase from their baseline platelet count). Patients are randomly assigned at a 1:1 ratio to receive baricitinib plus TPO-RA or TPO-RA alone. Both groups will continue their prior full-dose TPO-RA therapy for 14 days (day 1-14), while baricitinib was given orally at a dose of 2 mg twice daily concomitantly in the combination group for 14 days (day 1-14). The primary endpoint was the 14-day overall response rate without bleeding and any rescue therapy. The secondary endpoints included the 28-day overall response rate, 14-day complete response rate, the 28-day complete response rate, time to response, WHO bleeding scores, health-related quality of life, and adverse events.

• Study Type: Interventional
• Enrollment (Estimated): 100
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. ≥18 years old;
2. Patients diagnosed with primary ITP who failed to achieve a response after 14 days of full-dose TPO-RA therapy;
3. Patients with baseline platelet count less than 30×10⁹/L, or those with baseline platelet count ranging from 30×10⁹/L to 50×10⁹/L accompanied by clinically significant bleeding (WHO bleeding score ≥2).

Exclusion Criteria:

• Pregnant or lactating women, and who were possibly pregnant, planning to become pregnant, or who had partners planning to become pregnant;
• With active malignancy or a history of malignant tumor;
• Having experienced severe bacterial, viral, fungal or parasitic infection within the past 4 weeks;
• With a history of symptomatic herpes zoster infection within 12 weeks prior to screening;
• Active or chronic HBV, HCV or HIV infection;
• Evidence of active tuberculosis; or previous evidence of active tuberculosis without appropriate and documented treatment; or household contact with patients with active tuberculosis without appropriate and documented tuberculosis prophylaxis;
• Receipt of live vaccines within the past 12 weeks, or planned live vaccination during the study period;
• Prior baricitinib therapy;
• History of solid organ transplant or planned surgery;
• Myelodysplastic syndrome, aplastic anemia or myelofibrosis;
• Patients with other diseases were undergoing treatment with immunosuppressants;
• Clinically significant thromboembolic events within the past 24 weeks, or ongoing anticoagulant treatment, who are deemed ineligible for the study by the investigator;
• History or presence of myocardial infarction, unstable ischemic heart disease, stroke, or NYHA Class IV heart failure;
• History or active manifestations of severe or unstable cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, neuropsychiatric, or other medical conditions that, in the investigator's judgment, could confer unacceptable safety risks with the investigational product or confound the interpretation of study data;
• AST > 2 times the upper limit of normal (ULN), ALT > 2×ULN, TBIL ≥ 1.5×ULN;
• eGFR < 50 mL/min/1.73m²;
• Other patients deemed unsuitable for enrollment in this study by the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Combined therapy; Oral baricitinib is given at a dose of 2 mg twice daily for 14 days (day 1-14); prior full-dose TPO-RA therapy (hetrombopag 7.5mg once daily or eltrombopag 75mg once daily) was continued for 14 days (day 1-14).	Drug: Baricitinib; Oral baricitinib is given at a dose of 2 mg twice daily for 14 days.; Drug: TPO-RA; Hetrombopag is given at an initial dose of 7.5 mg once daily for 14 days; eltrombopag is given at an initial dose of 75 mg once daily for 14 days; Other Names: eltrombopag; hetrombopag
Active Comparator: Monotherapy; Prior full-dose TPO-RA (hetrombopag 7.5mg once daily or eltrombopag 75mg once daily) was continued.	Drug: TPO-RA; Hetrombopag is given at an initial dose of 7.5 mg once daily for 14 days; eltrombopag is given at an initial dose of 75 mg once daily for 14 days; Other Names: eltrombopag; hetrombopag

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
14-day Overall response rate	Overall response was defined as platelet count over 30,000/μL and at least a 2-fold increase of the baseline count in the absence of bleeding and rescue therapy.	From enrollment to the end of treatment at 14 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
14-day Complete response (CR) rate	Complete response (CR) was defined as platelet count over 100,000/μL and absence of bleeding.	From enrollment to the end of treatment at 14 days
28-day ovrall response rate	Overall response was defined as platelet count over 30,000/μL and at least a 2-fold increase of the baseline count and absence of bleeding.	From enrollment to the end of treatment at 28 days
28-day CR rate	Complete response (CR) was defined as platelet count over 100,000/μL and absence of bleeding.	From enrollment to the end of treatment at 28 days
Time to response (TTR)	The time from treatment initiation to achieve a CR or a R.	From the start of study treatment (Day 1) up to day 14
Bleeding events	Bleeding was assessed with the WHO bleeding scale (grade 0, no bleeding; grade 1, petechiae; grade 2, mild blood loss; grade 3, gross blood loss; grade 4, debilitating blood loss).	From the start of study treatment (Day 1) to the end of day 14
Health-related quality of life (HRQoL)	ITP-patient assessment questionnaire was used to assess the HRQoL before and after treatment.	From the start of study treatment (Day 1) to the end of day 14
AE	Adverse events	From enrollment to the end of treatment at 14 days

Collaborators and Investigators

• Sponsor: Peking University People's Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): August 31, 2028

Study Registration Dates

• First Submitted: June 20, 2026
• First Submitted That Met QC Criteria: June 20, 2026
• First Posted (Actual): June 25, 2026

Study Record Updates

• Last Update Posted (Actual): June 25, 2026
• Last Update Submitted That Met QC Criteria: June 20, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: baricitinib; immune thrombocytopenia; TPO-RA
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic; eltrombopag; baricitinib; hetrombopag
• Other Study ID Numbers: SEN-TPO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes