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

Sponsor
Peking University People's Hospital (Other)
Overall Status
Recruiting
CT.gov ID
NCT04747080
Collaborator
Beijing Hospital (Other), Beijing Friendship Hospital (Other), China-Japan Friendship Hospital (Other), The First Affiliated Hospital of Zhengzhou University (Other), First Affiliated Hospital of Chongqing Medical University (Other), Shanxi Bethune hospital, Shanxi, China (Other)
120
1
2
28
4.3

Study Details

Study Description

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).

Condition or Disease Intervention/Treatment Phase
Phase 2

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.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
120 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
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
Anticipated Study Start Date :
Mar 1, 2021
Anticipated Primary Completion Date :
Mar 1, 2023
Anticipated Study Completion Date :
Jul 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: TAC and HD-DXM

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 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.

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).
Other Names:
  • HD-DXM
  • High-dose Dexamethasone
  • Drug: 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.
    Other Names:
  • TAC
  • Active Comparator: HD-DXM

    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) .

    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).
    Other Names:
  • HD-DXM
  • High-dose Dexamethasone
  • Outcome Measures

    Primary Outcome Measures

    1. Sustained(Durable) response [6 months]

      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.

    Secondary Outcome Measures

    1. Complete response (CR) [Day 14]

      Complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.

    2. Response (R) [Day 14]

      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.

    3. Time to response [6 months]

      The time from starting treatment to time of achievement of CR or R.

    4. Duration of response (DOR) [6 months]

      Duration of response at 6-month follow up.

    5. Loss of response [6 months]

      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. Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale [From the start of study treatment (Day 1) up to the end of week 24.]

      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.

    7. Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) [Time Frame: From the start of study treatment (Day 1) up to the end of week 24.]

      In all participants ,use ITP-PAQ to assess the Health Related Quality of Life(HRQoL) before and after treatment.

    8. Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F) [Time Frame: From the start of study treatment (Day 1) up to the end of week 24.]

      In all participants ,use FACIT-F to assess the Health Related Quality of Life(HRQoL) before and after treatment.

    9. Number of Participants with side effects of the drugs [Time Frame: From the start of study treatment (Day 1) up to the end of week 24.]

      Side effects of the drugs included fever, headache, serum disease, infection, hypotension, rashes, infection liver injury, hypokalaemia, etc.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 70 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Peking University People's Hospital Beijing China 100000

    Sponsors and Collaborators

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

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Xiao Hui Zhang, Vice President of Peking University Institute of Hematology, Peking University People's Hospital
    ClinicalTrials.gov Identifier:
    NCT04747080
    Other Study ID Numbers:
    • ITP-PKU022
    First Posted:
    Feb 10, 2021
    Last Update Posted:
    Feb 10, 2021
    Last Verified:
    Feb 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    Plan to Share IPD:
    Undecided
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Feb 10, 2021