Combining low-dose Baricitinib with Danazol for treating immune thrombocytopenia

Low-dose Baricitinib Plus Danazol Versus Danazol for Patients With Steroid-resistant/Relapse Immune Thrombocytopenia: A Multicenter, Randomized, Open-label Phase 2 Trial

This study is testing if a combination of low-dose baricitinib and danazol can help adults with hard-to-treat immune thrombocytopenia improve their platelet counts and avoid bleeding.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 216 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Peking University People's Hospital (Academic / other)
• Drugs / interventions: baricitinib
• Locations: 10 sites (Beijing, Beijing and 9 other locations)
• Trial ID: NCT05852847 on ClinicalTrials.gov

What this trial studies

This phase 2 trial investigates the effectiveness and safety of combining low-dose baricitinib with danazol in adults suffering from steroid-resistant or relapsed immune thrombocytopenia (ITP). A total of 216 patients will be randomly assigned to receive either the combination therapy or danazol alone for a duration of six months. 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 to ensure a diverse patient population.

Who should consider this trial

Good fit: Ideal candidates are adults with chronic immune thrombocytopenia who have not responded to previous treatments and have low platelet counts.

Not a fit: Patients with secondary immune thrombocytopenia or those with active malignancies will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new effective option for patients with difficult-to-treat immune thrombocytopenia.

How similar studies have performed: While the combination of baricitinib and danazol is a novel approach, previous studies have shown promise in treating immune thrombocytopenia with other therapies.

Eligibility criteria

Inclusion Criteria:

1. Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia;
2. Patients with chronic low platelet count (\<30,000/μL) for 6 months who have failed at least one treatment for chronic low platelet count;
3. Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation;
4. Patients with a platelet count \<30,000/μL or a platelet count \<50,000/μL with clinically significant bleeding symptoms at the enrollment;
5. Willing and able to provide written informed consent, and agreeable to the schedule of assessment.

Exclusion Criteria:

1. Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection or patients with confirmed autoimmune disease);
2. Active or a history of malignancy;
3. Pregnancy or lactation;
4. Current or recent (\<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
5. A history of symptomatic herpes zoster infection within 12 weeks prior to screening;
6. Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
7. 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;
8. 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;
9. Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure;
10. 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;
11. 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, Beijing and 9 other locations

• Peking University Insititute of Hematology, Peking University People's Hospital — Beijing, 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, MD — Peking University Institute of Hematology, Peking University People's Hospital
• Study coordinator: Xiaohui Zhang, MD

Conditions Immune Thrombocytopenia; baricitinib