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

Sponsor
Peking University People's Hospital (Other)
Overall Status
Completed
CT.gov ID
NCT04217148
Collaborator
Beijing Hospital (Other), The Sixth Medical Center of PLA General Hospital (Other), Beijing Aerospace General Hospital (Other), Qilu Hospital of Shandong University (Other), Beijing Tongren Hospital (Other)
132
Enrollment
1
Location
2
Arms
5.9
Actual Duration (Months)
22.2
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or DiseaseIntervention/TreatmentPhase
Phase 2

Detailed Description

The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 132 adults with ITP in China. Patients were randomized to ATRA+ 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
Actual Enrollment :
132 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
The Combination of Oral All-trans Retinoic Acid and High-dose Dexamethasone vs High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia: A Multicenter, Randomized, Open-label Trial
Actual Study Start Date :
Jan 1, 2020
Actual Primary Completion Date :
Jun 30, 2020
Actual Study Completion Date :
Jun 30, 2020

Arms and Interventions

ArmIntervention/Treatment
Experimental: ATRA and HD-DXM

Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10) and ATRA 10mg bid po, 12 consecutive weeks

Drug: Dexamethasone
Dexamethasone, iv, 40 mg/d, for 4 days (The 4-day course of dexamethasone was repeated in the case of lack of response by day 10)
Other Names:
  • HD-DXM
  • High-dose Dexamethasone
  • Drug: ATRA
    ATRA, po,10mg bid, for 12 weeks
    Other Names:
  • All-trans retinoic acid
  • Active Comparator: HD-DXM

    Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10)

    Drug: Dexamethasone
    Dexamethasone, iv, 40 mg/d, for 4 days (The 4-day course of dexamethasone was repeated in the case of lack of response by day 10)
    Other Names:
  • HD-DXM
  • High-dose Dexamethasone
  • Outcome Measures

    Primary Outcome Measures

    1. Sustained response [6 month]

      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. Number of patients with bleeding [6 month]

      Number of patients with bleeding complication ( WHO bleeding score).

    4. Number of patients with adverse events [6 month]

      Number of patients with adverse events.

    5. Time to response [6 month]

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

    6. Duration of response (DOR) [6 month]

      Duration of response at 6-month follow up.

    7. Loss of response [6 month]

      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)

    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

    SiteCityStateCountryPostal Code
    1Peking University Insititute of Hematology, Peking University People's HospitalBeijingBeijingChina100044

    Sponsors and Collaborators

    • Peking University People's Hospital
    • Beijing Hospital
    • The Sixth Medical Center of PLA General Hospital
    • Beijing Aerospace General Hospital
    • Qilu Hospital of Shandong University
    • Beijing Tongren Hospital

    Investigators

    • Principal Investigator: Xiaohui Zhang, doctor, Peking University People's Hospital, Peking University Insititute of Hematology

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Xiao Hui Zhang, Vice president of Peking Univeristy Institute of Hematology, Peking University People's Hospital
    ClinicalTrials.gov Identifier:
    NCT04217148
    Other Study ID Numbers:
    • ITP-PKU007
    First Posted:
    Jan 3, 2020
    Last Update Posted:
    Sep 28, 2021
    Last Verified:
    Sep 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    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 Sep 28, 2021