Clinical trial NCT04747080: The Combination of Tacrolimus and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia
Official title: The Combination of High-dose Dexamethasone and Tacrolimus Versus High-dose Dexamethasone as the First-Line Treatment of Newly-diagnosed Immune Thrombocytopenia: A Randomized, Controlled, Multicenter, Open-label Trial
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: ITP
Immune Thrombocytopenia
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
Collaborators: name: Beijing Hospital
name: Beijing Friendship Hospital
name: China-Japan Friendship Hospital
name: The First Affiliated Hospital of Zhengzhou University
name: First Affiliated Hospital of Chongqing Medical University
name: Shanxi Bethune Hospital
First posted: 2021-02-10
Enrollment: 120
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 Combination of High-dose Dexamethasone and Tacrolimus versus High-dose Dexamethasone 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 100 adults with ITP in China. Patients were randomized to tacrolimuis plus 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 \<30×109/L ;
3. Platelet counts \< 50×109/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;
9. Patients who are deemed unsuitable for the study by the investigator.

Primary outcomes:
measure: Sustained（Durable） response
timeFrame: 6 months
Secondary outcomes:
measure: Complete response (CR)
timeFrame: Day 14
measure: Response (R)
timeFrame: Day 14
measure: Time to response
timeFrame: 6 months
measure: Duration of response (DOR)
timeFrame: 6 months
measure: Loss of response
timeFrame: 6 months
measure: Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale
timeFrame: From the start of study treatment (Day 1) up to the end of week 24.
measure: Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)
timeFrame: Time Frame: From the start of study treatment (Day 1) up to the end of week 24.
measure: Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F)
timeFrame: Time Frame: From the start of study treatment (Day 1) up to the end of week 24.
measure: Number of Participants with side effects of the drugs
timeFrame: Time Frame: From the start of study treatment (Day 1) up to the end of week 24.
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University People's Hospital
Study officials:
References: