Combining low-dose Baricitinib with high-dose Dexamethasone for treating newly diagnosed Immune Thrombocytopenia

Low-dose Baricitinib Plus High-dose Dexamethasone as First-line Treatment for Patients With Newly Diagnosed Immune Thrombocytopenia: a Multicenter, Open-label, Randomized, Controlled, Phase 2 Trial

This study is testing if a combination of low-dose Baricitinib and high-dose Dexamethasone can help adults with newly diagnosed immune thrombocytopenia better than just Dexamethasone alone.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 132 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Peking University People's Hospital (Academic / other)
• Drugs / interventions: chemotherapy, baricitinib
• Locations: 10 sites (Beijing and 9 other locations)
• Trial ID: NCT05932524 on ClinicalTrials.gov

What this trial studies

This phase 2 trial investigates the effectiveness and safety of low-dose Baricitinib combined with high-dose Dexamethasone compared to Dexamethasone alone in adults with newly diagnosed immune thrombocytopenia (ITP). Patients will be randomly assigned to one of the two treatment groups, and the primary goal is to achieve a durable response defined by specific platelet count criteria and the absence of bleeding. The study is conducted across multiple centers in China, ensuring a diverse patient population. Treatment will be halted if severe adverse events occur or at the patient's request.

Who should consider this trial

Good fit: Ideal candidates are adults with newly diagnosed, treatment-naive immune thrombocytopenia and a platelet count below 30,000/μL.

Not a fit: Patients with a history of other autoimmune diseases or those who have previously received ITP-modifying therapies will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment combination could provide a more effective first-line therapy for patients with immune thrombocytopenia.

How similar studies have performed: While there have been studies on ITP treatments, this specific combination of Baricitinib and high-dose Dexamethasone is a novel approach.

Eligibility criteria

Inclusion Criteria:

1. Confirmed newly-diagnosed, treatment-naive ITP;
2. A platelet count \<30,000/μL, or a platelet count \<50,000/μL with clinically significant bleeding symptoms (WHO bleeding scale 2 or above) at the enrollment;
3. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
2. Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;
3. Active or a history of malignancy;
4. Pregnancy or lactation;
5. Received first-line and second-line ITP-modifying therapy;
6. Previously received corticosteroids or immunosuppressive agents for non-ITP diseases within 6 months before enrollment;
7. A history of clinically significant adverse reactions to previous corticosteroid therapy;
8. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);
9. Current or recent (\<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
10. A history of symptomatic herpes zoster infection within 12 weeks prior to screening;
11. Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
12. Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB;
13. Have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled;
14. Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure;
15. A history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data;
16. Any of the following specific abnormalities on screening laboratory tests:

1) ALT or AST \>2 x ULN, or total bilirubin ≥1.5 x ULN 2) hemoglobin \<9 g/dL, or total white blood cell (WBC) count \<2,500/µL, or neutropenia (absolute neutrophil count \<1,200/µL), or lymphopenia (lymphocyte count \<750/µL) 3) eGFR \<50 mL/min/1.73 m\^2.

Where this trial is running

Beijing and 9 other locations

• Peking University Insititute of Hematology, Peking University People's Hospital — Beijing, China (Recruiting)
• Beijing Friendship Hospital — Beijing, China (Recruiting)
• Beijing Hospital — Beijing, China (Recruiting)
• Beijing Luhe Hospital — Beijing, China (Recruiting)
• Beijing Tsinghua Changgeng Hospital — Beijing, China (Recruiting)
• China-Japan Friendship Hospital — Beijing, China (Recruiting)
• Chinese PLA General Hospital — Beijing, China (Recruiting)
• Peking University First Hospital — Beijing, China (Recruiting)
• Peking University Third Hospital — Beijing, China (Recruiting)
• The Sixth Medical Center of PLA General Hospital — Beijing, China (Recruiting)

Study contacts

• Principal investigator: Xiaohui Zhang — Peking University Institute of Hematology, Peking University People's Hospital
• Study coordinator: Xiaohui Zhang

Conditions Immune Thrombocytopenia; Baricitinib; High-dose dexamethasone