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NCT Number: NCT07653581

rhTPO + CsA vs CsA in the Treatment of LR-MDS With Thrombocytopenia

This study aims to compare the efficacy and safety of recombinant human thrombopoietin (rhTPO) + cyclosporine (CsA) and CsA monotherapy in the treatment of lower-risk myelodysplastic neoplasms with thrombocytopenia.

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Key information

Conditions

Lower-risk Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Age ≥ 18 years old;
  • Confirmed diagnosis of lower-risk MDS;
  • Platelet ≤ 30×109/L, or < 50×109/L and accompanied by bleeding events;
  • Baseline liver and kidney functions <2 ULN;
  • Signed the informed consent form;
  • ECOG 0-2;

Exclusion criteria

  • History of leukemia or stem cell transplantation, treatment-related MDS or malignant tumors;
  • With splenomegaly or myelofibrosis;
  • Infections or bleeding that cannot be controlled by standard treatment;
  • Any concurrent malignant tumors within the past 5 years, or basal cell carcinoma of the skin at the local site;
  • History of thromboembolic events, heart attack or stroke and current use of anticoagulants;
  • Previously received cyclosporine (CsA) or ATG within 6 months;
  • Previously used IL-11, rhTPO or TPO-RA;
  • Pregnant or lactating women;
  • Participated in other clinical trials within 3 months.

Treatment and study plan

rhTPO

Drug

rhTPO 300U/kg/d, subcutaneous injection, once a month for 14 consecutive days

Cyclosporine (CsA)

Drug

CsA 3-5mg/kg/d

Primary outcomes

  1. HI-P

    Time frame: 1-month, 2-month, 3-month, 6-month

    hematological response - platelet

  2. ORR

    Time frame: 1-month, 2-month, 3-month, 6-month

    ORR=CRR+PRR+HI

Secondary outcomes

  1. Number of bleeding incidents and severity assessed by the World Health Organization Bleeding Scale

    Time frame: 3-month, 6-month

    minimum value = 0, maximum value = 4; higher scores indicate worse outcome

  2. Platelet transfusion independence

    Time frame: 3-month, 6-month

    according to the number and interval of platelet transfusions

  3. Time to CR/OR

    Time frame: through study period, an average of 6 months

    Time from baseline to first response

  4. Duration of HI-P

    Time frame: through study period, an average of 6-month

    Time to patients achieving HI-P to losing response

  5. Quality of life score measured by the Short Form-36 Health Survey

    Time frame: 3-month

    minimum value = 0, maximum value = 100; higher scores indicate better quality of life

  6. Clonal evolution

    Time frame: 12-month

    The incidence of elevation of blast cells in bone marrow and peripheral blood within one year, and the incidence of progressing to higher-risk MDS or AML within one year

  7. OS

    Time frame: 12-month

    Overall survival (OS), defined as the time from randomization/treatment start to death from any cause.

Study contacts

Contact information is provided by the study sponsor or research team.

Bing Han, Doctor

CONTACT

[email protected]

+86 13601059938

Sponsors and collaborators

Lead sponsor

Peking Union Medical College Hospital

Other

Registry information

Official study title

rhTPO + CsA Versus CsA Alone in the Treatment of Lower-risk MDS With Thrombocytopenia: a Single-center Randomized Controlled Trial

Important dates

Study start
2026
Primary completion
2028
Study completion
2029
First posted
Jun 17, 2026
Registry last updated
Jun 17, 2026

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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