A randomized, open-label, multicenter, phase 2 trial to compare the efficacy and safety of baricitinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
This is a parallel group, multicenter, randomized, controlled trial of patients with ITP in China. Patients were randomly assigned to receive baricitinib plus high-dose dexamethasone or high-dose dexamethasone monotherapy. 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 and 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
132
Baricitinib 2 mg q.d., p.o., for 6 consecutive months.
Dexamethasone 40 mg q.d. for 4 consecutive days (the 4-day course of dexamethasone will be repeated in the case of lack of response by day 10)
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, China
RECRUITINGBeijing Friendship Hospital
Beijing, China
RECRUITINGBeijing Hospital
Durable 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
Duration of response
Duration of response at 6-month follow up.
Time frame: 6 months
Early response
Achievement of CR or R at day 7
Time frame: 7 days
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: From the start of study treatment (Day 1) to the end of week 26
Health-related quality of life (HRQoL)
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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
RECRUITINGITP-PAQ is used to assess the Health Related Quality of Life (HRQoL) before and after treatment.
Time frame: From the start of study treatment (Day 1) to the end of week 26
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: From the start of study treatment (Day 1) to the end of week 26