Low-dose Baricitinib Plus Danazol for Steroid-resistant/Relapse Immune Thrombocytopenia
- Conditions
- Immune Thrombocytopenia
- Interventions
- Registration Number
- NCT05852847
- Lead Sponsor
- Peking University People's Hospital
- Brief Summary
This is a prospective, multicenter, randomized, controlled phase 2 trial to compare the efficacy and safety profiles in ITP patients receiving baricitinib plus danazol to those receiving danazol alone.
- Detailed Description
This is a prospective, multicenter, randomized, controlled design of 216 adult patients with steroid-resistant/relapse ITP in China. Patients are randomly assigned at a 1:1 ratio to receive baricitinib plus danazol or danazol alone. Patients in the combination therapy group receive oral baricitinib at a dose of 2 mg daily and oral danazol at a dose of 200 mg twice a day. Those in the monotherapy group receive oral danazol at 200 mg twice daily. The treatment lasts for 6 months. Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request. The primary endpoint is durable response, defined as the maintenance of platelet count ≥ 30,000/μL, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 216
- Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia;
- Patients with chronic low platelet count (<30,000/μL) for 6 months who have failed at least one treatment for chronic low platelet count;
- 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;
- Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with clinically significant bleeding symptoms at the enrollment;
- Willing and able to provide written informed consent, and agreeable to the schedule of assessment.
- Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection or patients with confirmed autoimmune disease);
- Active or a history of malignancy;
- Pregnancy or lactation;
- Current or recent (<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
- A history of symptomatic herpes zoster infection within 12 weeks prior to screening;
- Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
- 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;
- 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;
- Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure;
- 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;
- Any of the following specific abnormalities on screening laboratory tests:
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Low-dose baricitinib plus danazol Baricitinib 2 MG [Olumiant] Oral baricitinib is given at a dose of 2 mg daily for 6 months. Danazol is given at a dose of 200 mg twice a day for 6 months. Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request. Low-dose baricitinib plus danazol Danazol Oral baricitinib is given at a dose of 2 mg daily for 6 months. Danazol is given at a dose of 200 mg twice a day for 6 months. Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request. Danazol Danazol Danazol is given at a dose of 200 mg twice a day for 6 months. Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request.
- Primary Outcome Measures
Name Time Method Durable response 6 months The maintenance of a platelet count ≥30,000/μL, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
- Secondary Outcome Measures
Name Time Method Adverse events 6 months Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.
Time to response 6 months The time from starting treatment to time of achievement of CR or R.
Initial response 28 days Achievement of CR or R at day 28
Bleeding events 6 months Clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
Health-related quality of life (HRQoL) 6 months ITP-PAQ is used to assess the Health Related Quality of Life (HRQoL) before and after treatment.
Complete response (CR) 1 month A platelet count over 100,000/μL and absence of bleeding.
Response (R) 1 month A platelet count over 30,000/μL and at least 2-fold increase of the baseline count and absence of bleeding.
Trial Locations
- Locations (10)
The Sixth Medical Center of PLA General Hospital
🇨🇳Beijing, China
Peking University Insititute of Hematology, Peking University People's Hospital
🇨🇳Beijing, Beijing, China
Beijing Friendship Hospital
🇨🇳Beijing, China
Beijing Hospital
🇨🇳Beijing, China
China-Japan Friendship Hospital
🇨🇳Beijing, China
Chinese PLA General Hospital
🇨🇳Beijing, China
Beijing Luhe Hospital
🇨🇳Beijing, China
Beijing Tsinghua Changgeng Hospital
🇨🇳Beijing, China
Peking University Third Hospital
🇨🇳Beijing, China
Peking University First Hospital
🇨🇳Beijing, China