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

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

Sponsors

Lead Sponsor: Peking University People's Hospital

Collaborator: Beijing Hospital
The Sixth Medical Center of PLA General Hospital
Beijing Aerospace General Hospital
Qilu Hospital of Shandong University
Beijing Tongren Hospital

Source 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).

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.

Overall Status Recruiting
Start Date June 1, 2015
Completion Date December 2022
Primary Completion Date December 2022
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Sustained response 6 month
Secondary Outcome
Measure Time Frame
complete response (CR) day 14
Response (R) day 14
Number of patients with bleeding 6 month
Number of patients with adverse events 6 month
Time to response 6 month
Duration of response (DOR) 6 month
Loss of response 6 month
Enrollment 250
Condition
Intervention

Intervention Type: Drug

Intervention Name: Dexamethasone

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: ATRA

Description: ATRA, po,10mg bid, for 12 weeks

Arm Group Label: ATRA and HD-DXM

Other Name: All-trans retinoic acid

Eligibility

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.

Gender: All

Minimum Age: 18 Years

Maximum Age: 70 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Xiaohui Zhang, doctor Principal Investigator Peking University People's Hospital, Peking University Insititute of Hematology
Overall Contact

Last Name: Xiaohui Zhang, doctor

Phone: +8613522338836

Email: [email protected]

Location
Facility: Status: Contact: Peking University Insititute of Hematology, Peking University People's Hospital Xiao-hui Zhang, Professor [email protected]
Location Countries

China

Verification Date

October 2020

Responsible Party

Type: Principal Investigator

Investigator Affiliation: Peking University People's Hospital

Investigator Full Name: Xiao Hui Zhang

Investigator Title: Vice president of Peking Univeristy Institute of Hematology

Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: ATRA and HD-DXM

Type: Experimental

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

Label: HD-DXM

Type: Active Comparator

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)

Patient Data Undecided
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov