A Phase 2/3 interventional study of Danazol + rhTPO (recombinant human thrombopoietin injection) and Danazol + sodium chloride in Low/Intermediate Risk-1 MDS, sponsored by Peking Union Medical College Hospital. Status unknown at 1 site in China. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-03-27.
Sponsored by Peking Union Medical College Hospital · Phase 2/3, Interventional, and Treatment
Myelodysplastic syndrome (MDS) is a kind of clonal myeloid tumor. The major manifestation is decrease of tri-lineages of blood due to ineffective and abnormal hematopoiesis, some of which can progress to acute myeloid leukemia. According to the international prognosis scoring system (IPSS) of MDS, about 10% low/intermediate risk-1 MDS patients have severe thrombocytopenia (PLT \< 30 × 109/ L). These patients have both decreased platelet count and platelet dysfunction, resulting in a high risk of bleeding. In the new prognostic score, such as IPSS-r, the degree of thrombocytopenia is regarded as a poor prognostic factor. Platelet transfusion is mainly used in the treatment of this kind of patients. The indications of transfusion include bleeding events or severe platelet count reduction (\< 10 × 109 / L). However, platelet transfusion can only lead to short-term platelet elevation, while repeated transfusion increases the possibility of infection and ineffective platelet transfusion. TPO is a newly discovered hematopoietic promoting factor, which can specifically bind to the TPO receptor on the cell and participate in the regulation of proliferation, differentiation, maturation and division of megakaryocyte to form functional platelet. The efficacy and safety of the TPO receptor agonists eltrombopag and romiplostim in the treatment of thrombocytopenia in low/intermediate risk-1 MDS patients have been successfully confirmed in foreign studies. Recombinant human thrombopoietin (rhTPO) is also a kind of a TPO receptor agonists which is highly specific platelet stimulating factor. At present, there is no large report on the application of rhTPO in such patients. The purpose of this study is to explore the short-term and long-term therapeutic effect and safety of rhTPO on low/intermediate risk-1 MDS patients.
697 studies on the registry are indexed under Thrombocytopenia; 153 are open to participants now.
This study's planned enrollment of 75 is above the median of 55 across 472 interventional studies indexed under Thrombocytopenia.
Browse Thrombocytopenia studies →Peking Union Medical College Hospital is the lead sponsor of 1,115 studies on the registry; 463 are open to participants now.
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Exclusion Criteria:
Danazol 0.2g tid po+rhTPO (recombinant human thrombopoietin injection) 300U/kg/d×14d si every month (stop when PLT≥100×10e9/L or increased more than 50×10e9/L), total course 6 months
Drug: Danazol + rhTPO (recombinant human thrombopoietin injection)
Danazol 0.2g tid po+ control (sodium chloride)×14d si every month, total course 6 months
Drug: Danazol + sodium chloride
subcutaneous injection, 300U/kg/d×14d every month，stop if the PLT≥100×10e9/L or increased \>50×10e9/L, total course is 6 months
Danazol 0.2g tid po+ control (sodium chloride)
overall response rate
overall response rate of platelet
Time frame: 1 year
rate of side effects
rate of all kinds of side effects
Time frame: 1 year
WHO bleeding score
to evaluate frequency and severity of bleeding
Time frame: 1 year
change of platelet transfusion
the frequency and amount of platelet transfusion
Time frame: 1 year
onset time for overall response
onset time for complete and partial response
Time frame: through study completion, an average of 1 year
duration of overall response
during time for complete and partial response
Time frame: through study completion, an average of 1 year
incidence of TPO antibody
rate of presence of TPO antibody
Time frame: 1 year
life quality for MDS patients
life quality for MDS patients by QoL-E questionaire
Time frame: 1 year
the increased number of myeloblasts in bone marrow and peripheral blood
the increased number of myeloblasts in bone marrow and peripheral blood
Time frame: 1 year
incidence of progression to high-risk MDS or leukemia
incidence of progression to high-risk MDS or leukemia
Time frame: 1 year
Plan to share: Yes — protocol, consent form, clinical data would be available by personal contact
Supporting information: Study protocol, Sap, Icf, Csr
This study is status unknown, as verified in Mar 2020. You cannot join it, but the record below documents what was studied.
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Peking Union Medical College Hospital