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

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ClinicalTrials.gov Identifier: NCT04217148
Recruitment Status : Recruiting
First Posted : January 3, 2020
Last Update Posted : June 12, 2020
Sponsor:
Collaborators:
Beijing Hospital
The Sixth Medical Center of PLA General Hospital
Beijing Aerospace General Hospital
Qilu Hospital of Shandong University
Beijing Tongren Hospital
Information provided by (Responsible Party):
Xiao Hui Zhang, Peking University People's Hospital

Tracking Information
First Submitted Date  ICMJE December 30, 2019
First Posted Date  ICMJE January 3, 2020
Last Update Posted Date June 12, 2020
Actual Study Start Date  ICMJE June 1, 2015
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 10, 2020)
Sustained response [ Time Frame: 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. Interim analysis was scheduled at 50% through recruitment.
Original Primary Outcome Measures  ICMJE
 (submitted: January 2, 2020)
Sustained response [ Time Frame: 6 month ]
Platelet count maintained 50,000 per cubic millimeter with an absence of bleeding symptoms or no requirement for additional ITP-modifying treatment of 6 consecutive months following achievement of initial response. Interim analysis was scheduled at 50% through recruitment.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 10, 2020)
  • complete response (CR) [ Time Frame: day 14 ]
    complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.Interim analysis was scheduled at 50% through recruitment.
  • Response (R) [ Time Frame: 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.Interim analysis was scheduled at 50% through recruitment.
  • Number of patients with bleeding [ Time Frame: 6 month ]
    Number of patients with bleeding complication ( WHO bleeding score). Interim analysis was scheduled at 50% through recruitment.
  • Number of patients with adverse events [ Time Frame: 6 month ]
    Number of patients with adverse events. Interim analysis was scheduled at 50% through recruitment.
  • Time to response [ Time Frame: 6 month ]
    The time from starting treatment to time of achievement of CR or R
  • Duration of response (DOR) [ Time Frame: 6 month ]
    Duration of response at 6-month follow up. Interim analysis was scheduled at 50% through recruitment.
  • Loss of response [ Time Frame: 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)
Original Secondary Outcome Measures  ICMJE
 (submitted: January 2, 2020)
  • complete response (CR) [ Time Frame: 6 month ]
    complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.Interim analysis was scheduled at 50% through recruitment.
  • Response (R) [ Time Frame: 6 month ]
    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.Interim analysis was scheduled at 50% through recruitment.
  • Number of patients [ Time Frame: 6 month ]
    Number of patients with bleeding complication therapy ( bleeding score). Interim analysis was scheduled at 50% through recruitment.
  • Number of patients with adverse events [ Time Frame: 6 month ]
    Number of patients with adverse events. Interim analysis was scheduled at 50% through recruitment.
  • Initial response [ Time Frame: day 10 ]
    An increase in the platelet count of at least 30,000 per cubic millimeter, a platelet count of more than 50,000 per cubic millimeter by day 10 after the initiation of treatment, and cessation of bleeding. Interim analysis was scheduled at 50% through recruitment.
  • Duration of response (DOR) [ Time Frame: 6 month ]
    Duration of response at 6-month follow up. Interim analysis was scheduled at 50% through recruitment.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE The Combination of ATRA and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia
Official Title  ICMJE 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
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).
Detailed Description The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 240 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. Interim analysis was scheduled at 50% through recruitment. In order to report the efficacy and safety of ATRA plus high-dose dexamethasone for the first-line treatment of adults with ITP.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Immune Thrombocytopenia
Intervention  ICMJE
  • 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 Name: All-trans retinoic acid
Study Arms  ICMJE
  • 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
    Interventions:
    • Drug: Dexamethasone
    • Drug: ATRA
  • 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)
    Intervention: Drug: Dexamethasone
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 2, 2020)
250
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2022
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 3 months before the screening visit;
  2. Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) within 3 months before the screening visit;
  3. Received high-dose steroids or IVIG in the 3 weeks prior to the start of the study.
  4. Current HIV infection or hepatitis B virus or hepatitis C virus infections;
  5. 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);
  6. Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period;
  7. 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;
  8. Patients who are deemed unsuitable for the study by the investigator.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Xiaohui Zhang, doctor +8613522338836 zhangxh100@sina.com
Contact: Qiusha Huang, doctor +8613051816058 huangfuqs@163.com
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04217148
Other Study ID Numbers  ICMJE ITP-PKU007
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Xiao Hui Zhang, Peking University People's Hospital
Study Sponsor  ICMJE Peking University People's Hospital
Collaborators  ICMJE
  • Beijing Hospital
  • The Sixth Medical Center of PLA General Hospital
  • Beijing Aerospace General Hospital
  • Qilu Hospital of Shandong University
  • Beijing Tongren Hospital
Investigators  ICMJE
Principal Investigator: Xiaohui Zhang, doctor Peking University People's Hospital, Peking University Insititute of Hematology
PRS Account Peking University People's Hospital
Verification Date June 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP