Clinical trial NCT05369364: The Combination of Zanubrutinib and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia
Official title: The Combination of Oral Zanubrutinib and High-dose Dexamethasone vs High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia: A Multicenter, Randomized, Open-label Trial
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Keywords: BTK inhibitor
High-dose Dexamethasone
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
Collaborators: name: Beijing Hospital
name: Navy General Hospital, Beijing
name: Beijing Aerospace General Hospital
name: Qilu Hospital of Shandong University
name: Beijing Tongren Hospital
First posted: 2022-05-11
Enrollment: 150
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Randomized, open-label, multicenter study to compare the efficacy and safety of zanubrutinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).

Detailed description: The investigators are undertaking a parallel group, multicenter, randomized controlled trial of patients with ITP in China. Patients were randomized to Zanubrutinib + high-dose dexamethasone and high-dose dexamethasone monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Eligibility criteria: Inclusion Criteria:

1. Confirmed newly-diagnosed, treatment-naive ITP;
2. Platelet counts \<30×10\^9/L ;
3. Platelet counts \< 50×10\^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
4. 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. Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ;
3. Current HIV infection or hepatitis B virus or hepatitis C virus infections;
4. Active infection;
5. Maligancy;
6. Severe medical condition (lung, hepatic or renal disorder) other than chronic ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);
7. Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period; a history of clinically significant adverse reactions to previous corticosteroid therapy;
8. 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.

Primary outcomes:
measure: Sustained response
timeFrame: 12 months
Secondary outcomes:
measure: Initial complete response
timeFrame: 1 month
measure: Initial response
timeFrame: 1 month
measure: Number of patients with bleeding
timeFrame: 12 months
measure: Number of patients with adverse events
timeFrame: 12 months
measure: Time to response (TTR)
timeFrame: 12 months
measure: Duration of response (DOR)
timeFrame: 12 months
measure: Loss of response
timeFrame: 12 month
measure: Durable response
timeFrame: 6 months
measure: Early response
timeFrame: 1 week
measure: Remission
timeFrame: 12 months
Other outcomes:
Locations:
Study officials:
affiliation: Peking University People's Hospital
name: Xiaohui Zhang, md
role: PRINCIPAL_INVESTIGATOR
References: