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

NCT04217148 | Completed Phase 2

• 1 other identifier: ITP-PKU007
• Study Type: interventional
• Target: 132
• Locations: 1 country
• Sites: 1

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).

Conditions

Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• 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.

  6 month

Secondary Outcomes (7)

• 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

Study Arms (2)

ATRA and HD-DXM

EXPERIMENTAL

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; Drug: ATRA

HD-DXM

ACTIVE COMPARATOR

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

Interventions

Dexamethasone DRUG

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)

Also known as: HD-DXM, High-dose Dexamethasone

ATRA and HD-DXM; HD-DXM

ATRA DRUG

ATRA, po,10mg bid, for 12 weeks

Also known as: All-trans retinoic acid

ATRA and HD-DXM

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Peking University People's Hospital: lead
• Beijing Hospital: collaborator
• Navy General Hospital, Beijing: collaborator
• Beijing Aerospace General Hospital: collaborator
• Qilu Hospital of Shandong University: collaborator
• Beijing Tongren Hospital: collaborator

Study Sites (1)

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

Beijing, Beijing Municipality, 100044, China

Location

Related Publications (1)

• 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.

  PMID: 34560012 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Dexamethasone; Tretinoin

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated; Vitamin A; Retinoids; Carotenoids; Polyenes; Alkenes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Cyclohexenes; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Terpenes; Diterpenes; Pigments, Biological; Biological Factors

Study Officials

• Xiaohui Zhang, doctor

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking Univeristy Institute of Hematology

Study Record Dates

• First Submitted: December 30, 2019
• First Posted: January 3, 2020
• Study Start: January 1, 2020
• Primary Completion: June 30, 2020
• Study Completion: June 30, 2020
• Last Updated: September 28, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: These data will be available beginning 3 months and ending 36 months following publication.
• 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.

Locations

CN (1)