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

NCT07653581 — Phase 3, Not yet recruiting, 78 participants, Peking Union Medical College Hospital

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

Registry	NCT07653581
Status	Not yet recruiting
Phase	Phase 3
Study type	Interventional; randomized, parallel, masking: none, treatment
Enrollment	78 (Estimated)
Sponsor	Peking Union Medical College Hospital (Other)
Conditions	Lower-risk Myelodysplastic Syndromes
Interventions	rhTPO, Cyclosporine (CsA) (Drug)
Dates	start 2026-06-01 (estimated), primary completion 2028-12-01 (estimated), completion 2029-12-01 (estimated), first posted 2026-06-17, last updated 2026-06-17

-Summary

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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+Arms and interventions

Arm	Type	Interventions	Description
rhTPO+CsA	Experimental	Drug: rhTPO; Drug: Cyclosporine (CsA)	rhTPO 300U/kg/d, CsA 3-5mg/kg/d
CsA	Active comparator	Drug: Cyclosporine (CsA)	CsA 3-5mg/kg/d

rhTPO (Drug): rhTPO 300U/kg/d, subcutaneous injection, once a month for 14 consecutive days
Cyclosporine (CsA) (Drug): CsA 3-5mg/kg/d

+Outcome measures

Primary outcomes

HI-P [1-month, 2-month, 3-month, 6-month]: hematological response - platelet
ORR [1-month, 2-month, 3-month, 6-month]: ORR=CRR+PRR+HI

Secondary outcomes

Number of bleeding incidents and severity assessed by the World Health Organization Bleeding Scale [3-month, 6-month]: minimum value = 0, maximum value = 4; higher scores indicate worse outcome
Platelet transfusion independence [3-month, 6-month]: according to the number and interval of platelet transfusions
Time to CR/OR [through study period, an average of 6 months]: Time from baseline to first response
Duration of HI-P [through study period, an average of 6-month]: Time to patients achieving HI-P to losing response
Quality of life score measured by the Short Form-36 Health Survey [3-month]: minimum value = 0, maximum value = 100; higher scores indicate better quality of life
Clonal evolution [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
OS [12-month]: Overall survival (OS), defined as the time from randomization/treatment start to death from any cause.

+Eligibility

Sex: All, ages: 18 Years to any

Inclusion Criteria:

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

Exclusion Criteria:

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