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

Primary Purpose

Immune Thrombocytopenia

Status

Completed

Phase

Phase 2

Locations

China

Study Type

Interventional

Intervention

Dexamethasone

ATRA

About this trial

This is an interventional treatment trial for Immune Thrombocytopenia

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Confirmed newly-diagnosed, treatment-naive ITP;
Platelet counts <30×109/L ;
Platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
Willing and able to sign written informed consent.

Exclusion Criteria:

Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ;
Current HIV infection or hepatitis B virus or hepatitis C virus infections;
Active infection;
Maligancy;
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);
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
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;
Patients who are deemed unsuitable for the study by the investigator.

Sites / Locations

Peking University Insititute of Hematology, Peking University People's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

ATRA and HD-DXM

HD-DXM

Arm Description

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

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)

Outcomes

Primary Outcome Measures

Sustained 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.

Secondary Outcome Measures

complete response (CR)

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

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.

Number of patients with bleeding

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

Number of patients with adverse events

Number of patients with adverse events.

Time to response

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

Duration of response (DOR)

Duration of response at 6-month follow up.

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)

Full Information

NCT ID

NCT04217148

First Posted

December 30, 2019

Last Updated

September 22, 2021

Sponsor

Peking University People's Hospital

Collaborators

Beijing Hospital, Navy General Hospital, Beijing, Beijing Aerospace General Hospital, Qilu Hospital of Shandong University, Beijing Tongren Hospital

1. Study Identification

Unique Protocol Identification Number

NCT04217148

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2021

Overall Recruitment Status

Completed

Study Start Date

January 1, 2020 (Actual)

Primary Completion Date

June 30, 2020 (Actual)

Study Completion Date

June 30, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Peking University People's Hospital

Collaborators

Beijing Hospital, Navy General Hospital, Beijing, Beijing Aerospace General Hospital, Qilu Hospital of Shandong University, Beijing Tongren Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

5. 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).

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Immune Thrombocytopenia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

132 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ATRA and HD-DXM

Arm Type

Experimental

Arm Description

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

Arm Title

HD-DXM

Arm Type

Active Comparator

Arm Description

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 Type

Drug

Intervention Name(s)

Dexamethasone

Other Intervention Name(s)

HD-DXM, High-dose Dexamethasone

Intervention Description

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)

Intervention Type

Drug

Intervention Name(s)

ATRA

Other Intervention Name(s)

All-trans retinoic acid

Intervention Description

ATRA, po,10mg bid, for 12 weeks

Primary Outcome Measure Information:

Title

Sustained response

Description

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.

Time Frame

6 month

Secondary Outcome Measure Information:

Title

complete response (CR)

Description

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

Time Frame

day 14

Title

Response (R)

Description

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.

Time Frame

day 14

Title

Number of patients with bleeding

Description

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

Time Frame

6 month

Title

Number of patients with adverse events

Description

Number of patients with adverse events.

Time Frame

6 month

Title

Time to response

Description

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

Time Frame

6 month

Title

Duration of response (DOR)

Description

Duration of response at 6-month follow up.

Time Frame

6 month

Title

Loss of response

Description

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)

Time Frame

6 month

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

70 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Confirmed newly-diagnosed, treatment-naive ITP; Platelet counts <30×109/L ; Platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above); Willing and able to sign written informed consent. Exclusion Criteria: Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit; Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ; Current HIV infection or hepatitis B virus or hepatitis C virus infections; Active infection; Maligancy; 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); 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 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; Patients who are deemed unsuitable for the study by the investigator.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Xiaohui Zhang, doctor

Organizational Affiliation

Peking University People's Hospital, Peking University Insititute of Hematology

Official's Role

Principal Investigator

Facility Information:

Facility Name

Peking University Insititute of Hematology, Peking University People's Hospital

City

Beijing

State/Province

Beijing

ZIP/Postal Code

100044

Country

China

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Qualified researchers can request access to data, including deidentified individual participant data, and the study protocol from this clinical trial. These data will be available beginning 3 months and ending 36 months following publication. All requests must include a description of the research proposal and be sent to the corresponding author (zhangxh@bjmu.edu.cn). Data requestors will need to sign a data access agreement to obtain access.

IPD Sharing Time Frame

These data will be available beginning 3 months and ending 36 months following publication.

IPD Sharing Access Criteria

All requests must include a description of the research proposal and be sent to the corresponding author (zhangxh@bjmu.edu.cn). Data requestors will need to sign a data access agreement to obtain access.

Citations:

PubMed Identifier

34560012

Citation

Huang QS, Liu Y, Wang JB, Peng J, Hou M, Liu H, Feng R, Wang JW, Xu LP, Wang Y, Huang XJ, Zhang XH. All-trans retinoic acid plus high-dose dexamethasone as first-line treatment for patients with newly diagnosed immune thrombocytopenia: a multicentre, open-label, randomised, controlled, phase 2 trial. Lancet Haematol. 2021 Oct;8(10):e688-e699. doi: 10.1016/S2352-3026(21)00240-4.

Results Reference

derived

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

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