The Combination of Tacrolimus and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia

Immune Thrombocytopenia Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04747080
• Other study ID #: ITP-PKU022
• Status: Recruiting
• Phase: Phase 2
• Start date: March 1, 2021
• Est. completion date: July 1, 2023

Study information

• Verified date: February 2021
• Source: Peking University People's Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 120
• Est. completion date: July 1, 2023
• Est. primary completion date: March 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• ITP
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Dexamethasone
Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone is repeated in the case of lack of response by day 14).
• Tacrolimus
Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.

Locations

Country	Name	City	State
China	Peking University People's Hospital	Beijing

Sponsors (7)

Lead Sponsor	Collaborator
Peking University People's Hospital	Beijing Friendship Hospital, Beijing Hospital, China-Japan Friendship Hospital, First Affiliated Hospital of Chongqing Medical University, Shanxi Bethune Hospital, The First Affiliated Hospital of Zhengzhou University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Sustained(Durable) response	The maintenance of platelet count = 30 x 10^9/L, 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.	6 months
Secondary	Complete response (CR)	Complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.	Day 14
Secondary	Response (R)	Response (R) as platelet count more than 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.	Day 14
Secondary	Time to response	The time from starting treatment to time of achievement of CR or R.	6 months
Secondary	Duration of response (DOR)	Duration of response at 6-month follow up.	6 months
Secondary	Loss of response	Platelet counts below 100 x 109/L or bleeding (from CR) or platelet counts below 30 x 109/L, less than 2-fold increase of baseline platelet count or bleeding (from R)	6 months
Secondary	Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale	The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.	From the start of study treatment (Day 1) up to the end of week 24.
Secondary	Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)	In all participants ,use ITP-PAQ to assess the Health Related Quality of Life(HRQoL) before and after treatment.	Time Frame: From the start of study treatment (Day 1) up to the end of week 24.
Secondary	Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F)	In all participants ,use FACIT-F to assess the Health Related Quality of Life(HRQoL) before and after treatment.	Time Frame: From the start of study treatment (Day 1) up to the end of week 24.
Secondary	Number of Participants with side effects of the drugs	Side effects of the drugs included fever, headache, serum disease, infection, hypotension, rashes, infection liver injury, hypokalaemia, etc.	Time Frame: From the start of study treatment (Day 1) up to the end of week 24.