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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

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 disease Intervention/treatment Phase
Immune Thrombocytopenia Drug: Dexamethasone Drug: ATRA Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 250 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 : June 1, 2015
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : December 2022


Arm Intervention/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 Name: 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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. Number of patients with adverse events [ Time Frame: 6 month ]
    Number of patients with adverse events. Interim analysis was scheduled at 50% through recruitment.

  5. Time to response [ Time Frame: 6 month ]
    The time from starting treatment to time of achievement of CR or R

  6. Duration of response (DOR) [ Time Frame: 6 month ]
    Duration of response at 6-month follow up. Interim analysis was scheduled at 50% through recruitment.

  7. 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)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04217148


Contacts
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Contact: Xiaohui Zhang, doctor +8613522338836 zhangxh100@sina.com
Contact: Qiusha Huang, doctor +8613051816058 huangfuqs@163.com

Locations
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China, Beijing
Peking University Insititute of Hematology, Peking University People's Hospital Recruiting
Beijing, Beijing, China, 100044
Contact: Xiao-hui Zhang, Professor       zhangxh100@sina.com   
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
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Principal Investigator: Xiaohui Zhang, doctor Peking University People's Hospital, Peking University Insititute of Hematology
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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: January 3, 2020    Key Record Dates
Last Update Posted: June 12, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Dexamethasone
Dexamethasone acetate
Tretinoin
BB 1101
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Protease Inhibitors