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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
216
Oral baricitinib was given at a dose of 2 mg daily for 6 months. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patients' request.
Danazol was given at a dose of 200 mg bid for 6 months
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, China
RECRUITINGBeijing Friendship Hospital
Beijing, China
RECRUITINGDurable response
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.
Time frame: 6 months
Complete response (CR)
A platelet count over 100,000/μL and absence of bleeding.
Time frame: 1 month
Response (R)
A platelet count over 30,000/μL and at least 2-fold increase of the baseline count and absence of bleeding.
Time frame: 1 month
Time to response
The time from starting treatment to time of achievement of CR or R.
Time frame: 6 months
Initial response
Achievement of CR or R at day 28
Time frame: 28 days
Bleeding events
Clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
Time frame: 6 months
Health-related quality of life (HRQoL)
ITP-PAQ is used to assess the Health Related Quality of Life (HRQoL) before and after treatment.
Time frame: 6 months
Adverse events
Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.
Time frame: 6 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Beijing Hospital
Beijing, China
Beijing Luhe Hospital
Beijing, China
RECRUITINGBeijing Tsinghua Changgeng Hospital
Beijing, China
RECRUITINGChina-Japan Friendship Hospital
Beijing, China
RECRUITINGChinese PLA General Hospital
Beijing, China
RECRUITINGPeking University First Hospital
Beijing, China
RECRUITINGPeking University Third Hospital
Beijing, China
RECRUITINGThe Sixth Medical Center of PLA General Hospital
Beijing, China
RECRUITING