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

NCT07653581 | Not Yet Recruiting Phase 3

• 1 other identifier: K10660
• Study Type: interventional
• Target: 78
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Lower-risk Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (2)

• HI-P

  hematological response - platelet

  1-month, 2-month, 3-month, 6-month

• ORR

  ORR=CRR+PRR+HI

  1-month, 2-month, 3-month, 6-month

Secondary Outcomes (7)

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

  3-month, 6-month

• Platelet transfusion independence

  3-month, 6-month

• Time to CR/OR

  through study period, an average of 6 months

• Duration of HI-P

  through study period, an average of 6-month

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

  3-month

Study Arms (2)

rhTPO+CsA

EXPERIMENTAL

rhTPO 300U/kg/d, CsA 3-5mg/kg/d

Drug: rhTPO; Drug: Cyclosporine (CsA)

CsA

ACTIVE COMPARATOR

CsA 3-5mg/kg/d

Drug: Cyclosporine (CsA)

Interventions

rhTPO DRUG

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

rhTPO+CsA

Cyclosporine (CsA) DRUG

CsA 3-5mg/kg/d

CsA; rhTPO+CsA

Eligibility Criteria

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

You may qualify if:

• 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;

You may not qualify if:

• 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.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

MeSH Terms

Interventions

Cyclosporine

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Central Study Contacts

Bing Han, Doctor

CONTACT

+86 13601059938; hanbing_li@sina.com.cn

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: June 7, 2026
• First Posted: June 17, 2026
• Study Start: June 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029
• Last Updated: June 17, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share