Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA (NCT07400341) | Clinical Trial Compass
RecruitingPhase 2
Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA
China66 participantsStarted 2026-01-31
Plain-language summary
This study is for adults aged 18-65 with myelodysplastic syndrome (MDS) or severe aplastic anemia (AA) who are scheduled to receive a donor stem cell transplant (allogeneic hematopoietic stem cell transplant). After the transplant, it is critical for the body to start making its own blood cells again. A common and serious problem is a delay in the recovery of platelets (the cells that help stop bleeding), which increases the risk of bleeding, infection, and death.
This study aims to see if a new treatment can help platelets recover faster and more safely after transplant. We are comparing two drugs:
Romiplostim: A long-acting injection given just once a week. rhTPO (Recombinant Human Thrombopoietin): A standard injection given every day. Both drugs are designed to help the body make more platelets. The main question is whether the once-weekly romiplostim works as well or better than the daily rhTPO, and if it is safe.
About 66 patients will participate. By random chance (like flipping a coin), each participant will be assigned to receive either romiplostim or rhTPO. The treatment will start a few days after the transplant and continue until platelets recover to a safe level or for up to 8 weeks. Doctors will closely monitor all participants for 100 days to track platelet recovery, need for transfusions, side effects, and overall health.
Who can participate
Age range18 Years – 65 Years
SexALL
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Inclusion criteria
✓. Age 18-65 years (inclusive).
✓. Diagnosis of Myelodysplastic Syndrome (MDS) per WHO criteria, or Severe/Very Severe Aplastic Anemia (SAA/VSAA) per Camitta criteria, and deemed eligible for allogeneic hematopoietic stem cell transplantation (allo-HSCT).
✓. Planned to receive allo-HSCT from a matched sibling, haploidentical, or unrelated donor (including cord blood).
✓. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
✓. Persistent platelet count \<20×10⁹/L with platelet transfusion dependence between post-transplant days +4 and +10. Transfusion dependence is defined as platelet count not doubling within 24-48 hours after transfusion or ongoing need for prophylactic transfusion.
✓. Adequate cardiac, hepatic, and renal function as required for transplantation, per investigator assessment.
✓. Voluntary participation with written informed consent obtained prior to any study-specific procedures.
Exclusion criteria
✕. Active, uncontrolled bacterial, fungal, or viral infection at the time of enrollment.
What they're measuring
1
Rate of Platelet Engraftment by Day +60 (Platelets ≥50×10^9/L without transfusion for 7 consecutive days)
Timeframe: From the start of transplantation until Day 60 post-transplant (or until the engraftment criterion is met).
2
Incidence of ≥ Grade 3 Adverse Events within 100 Days Post-Transplant (including GVHD and thrombotic events)
Timeframe: From the start of transplantation until Day 100 post-transplant.
Trial details
NCT IDNCT07400341
SponsorThe First Affiliated Hospital of Soochow University
Sponsor typeOTHER
Study typeINTERVENTIONAL
Primary completion2028-01-31
Contact for this trial
Depei Wu Professor, Director of Hematology Department, MD
✕. History of arterial thrombosis, or venous thromboembolism within the past 6 months (unless cured or stable for over 6 months).
✕. Active transplant-associated thrombotic microangiopathy (TA-TMA).
✕. Pre-transplant bone marrow biopsy showing fibrosis grade ≥ MF-2 (according to WHO criteria).
✕. Known hypersensitivity to Romiplostim, recombinant human thrombopoietin (rhTPO), or any of their excipients.
✕. Pregnant or lactating women.
✕. Women of childbearing potential or men with partners of childbearing potential who are unwilling to use highly effective contraception during the study period and for at least 3 months after the last dose of study drug.
✕. Any other condition that, in the opinion of the investigator, would make the patient unsuitable for participation in the study.