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NCT Number: NCT05438875

The Combination of ATRA and Eltrombopag as the Treatment of Steroid-resistant/Relapse ITP Based on MSC-C5b-9

A Prospective, Randomized, Open-Label, Multicenter Clinical Trial study to compare the efficacy and safety of ATRA plus eltrombopag compared to eltrombopag monotherapy in the treatment of steroid-resistant/relapsed immune thrombocytopenia (ITP).

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Beijing Tongren hospital, Beijing, Beijing Municipality, China

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About this study

The investigators are undertaking a parallel group, multicenter, randomized controlled trial of patients with ITP in China. Patients were tested for MSCs, and they were divided into MSC-C5b-9+ group and MSC-C5b-9- group according to the test results, and the two groups were randomized to ATRA + eltrombopag and eltrombopag monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 1. Isolated thrombocytopenia (platelet count <30 × 109/L); 2. age > 18 years; 3. normal white blood cells and red blood cells on bone marrow examination; 4. increased number of megakaryocytes (bone marrow examination was performed in all patients except for myelofibrosis or other conditions that can cause thrombocytopenia disease); 5. the spleen was normal in size; 6. Eastern Cooperative Oncology Group status score (ECOG score) ≤ 2; 7. ineffective or relapsed after at least 1 course of full-dose full-course hormone therapy; 8. Failure of prior ITP therapy (eg, hormones, splenectomy, and cyclosporine) and at least 4 weeks from enrollment.

Exclusion criteria

  • 1. Secondary ITP such as drug-related thrombocytopenia; 2. thrombocytopenia due to viral infection (HIV, hepatitis B virus, or hepatitis C virus); 3. severe cardiac, renal, hepatic, or respiratory insufficiency; 4. severe immunodeficiency; 5. pregnancy or lactation; 6. myelodysplasia or Myelofibrosis; 7. history of malignancy; 8. ongoing immunosuppressive therapy for other diseases; 9. patients previously treated with eltrombopag were excluded from this study.

Treatment and study plan

All-trans retinoic acid

Drug

ATRA 10 mg, 2 times a day, orally.The treatment course is 12 weeks.

Other names: retinoic acid

eltrombopag

Drug

The initial dose of eltrombopag is 50 mg/time, once a day, and the dose is increased when the platelet count is lower than 5×109/L, the maximum is 75 mg/d, and the dose is higher than 200×109/L. When the drug is temporarily discontinued, the drug is re-administered according to the platelet count.The treatment course is 12 weeks.

Other names: TPO-RA

Primary outcomes

  1. Sustained Response Rate (SR) at 18 months

    Time frame: 18 months

    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 18-month follow-up

Secondary outcomes

  1. Complete response rate (CR)

    Time frame: 18 months

    The complete response (CR) was defined as platelet count more than 100×10^9/L and absence of bleeding.

  2. Response rate (R)

    Time frame: 18 months

    The Response rate (R) was defined as platelet count more than 30×10^9/L and more than 2 times higher than baseline, without bleeding

  3. Inefficiency (NR)

    Time frame: 18 months

    Platelet count <30×10^9/L, or less than 2-fold increase from baseline or associated with bleeding

  4. Recurrence rate (relapse)

    Time frame: 18 months

    After the treatment is effective, the platelet count drops below 30×10^9/L or drops to less than 2 times the basal value, or bleeding symptoms occur

  5. Early response

    Time frame: 1 week

    Platelet count ≥30×10^9/L and at least doubling baseline at 1 wk.

  6. Initial response

    Time frame: 1 month

    Initial response as platelet count more than 30×10^9/L and at least 2-fold increase of the baseline count and absence of bleeding.

  7. Time to response (TTR)

    Time frame: 18 months

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

  8. Time to relapse (duration of efficacy)

    Time frame: 18 months

    The time from achievement of CR or R to time of relapse

Sponsors and collaborators

Lead sponsor

Peking University People's Hospital

Other

Collaborators

  • Beijing Friendship Hospital
  • Beijing Hospital
  • Beijing Tongren Hospital
  • Navy General Hospital, Beijing

Registry information

Official study title

The Combination of ATRA and Eltrombopag as the Treatment Strategy for Glucocorticoid-Resistant/Relapsed ITP Based on the Stratification of the New Biomarker MSC-C5b-9: A Prospective, Randomized, Open-Label, Multicenter Clinical Trial

Important dates

Study start
2022
Primary completion
2024
Study completion
2024
First posted
Jun 30, 2022
Registry last updated
Sep 9, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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