Ignite Creation Date:
2025-12-25 @ 2:04 AM
Ignite Modification Date:
2025-12-26 @ 4:22 AM
Study NCT ID:
NCT04324060
Status:
UNKNOWN
Last Update Posted:
2020-03-27
First Post:
2020-03-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Randomized Trial of rhTPO Versus Placebo for Low/Intermediate-1 Risk MDS With Thrombocytopenia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'D003613', 'term': 'Danazol'}, {'id': 'D013926', 'term': 'Thrombopoietin'}, {'id': 'D012965', 'term': 'Sodium Chloride'}], 'ancestors': [{'id': 'D011245', 'term': 'Pregnadienes'}, {'id': 'D011278', 'term': 'Pregnanes'}, {'id': 'D013256', 'term': 'Steroids'}, {'id': 'D000072473', 'term': 'Fused-Ring Compounds'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D003115', 'term': 'Colony-Stimulating Factors'}, {'id': 'D006023', 'term': 'Glycoproteins'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D016298', 'term': 'Hematopoietic Cell Growth Factors'}, {'id': 'D016207', 'term': 'Cytokines'}, {'id': 'D036341', 'term': 'Intercellular Signaling Peptides and Proteins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D001685', 'term': 'Biological Factors'}, {'id': 'D002712', 'term': 'Chlorides'}, {'id': 'D006851', 'term': 'Hydrochloric Acid'}, {'id': 'D017606', 'term': 'Chlorine Compounds'}, {'id': 'D007287', 'term': 'Inorganic Chemicals'}, {'id': 'D017670', 'term': 'Sodium Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 75}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2020-03-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-03', 'completionDateStruct': {'date': '2022-12-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2020-03-26', 'studyFirstSubmitDate': '2020-03-25', 'studyFirstSubmitQcDate': '2020-03-26', 'lastUpdatePostDateStruct': {'date': '2020-03-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-03-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-03-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'overall response rate', 'timeFrame': '1 year', 'description': 'overall response rate of platelet'}, {'measure': 'rate of side effects', 'timeFrame': '1 year', 'description': 'rate of all kinds of side effects'}], 'secondaryOutcomes': [{'measure': 'WHO bleeding score', 'timeFrame': '1 year', 'description': 'to evaluate frequency and severity of bleeding'}, {'measure': 'change of platelet transfusion', 'timeFrame': '1 year', 'description': 'the frequency and amount of platelet transfusion'}, {'measure': 'onset time for overall response', 'timeFrame': 'through study completion, an average of 1 year', 'description': 'onset time for complete and partial response'}, {'measure': 'duration of overall response', 'timeFrame': 'through study completion, an average of 1 year', 'description': 'during time for complete and partial response'}, {'measure': 'incidence of TPO antibody', 'timeFrame': '1 year', 'description': 'rate of presence of TPO antibody'}, {'measure': 'life quality for MDS patients', 'timeFrame': '1 year', 'description': 'life quality for MDS patients by QoL-E questionaire'}, {'measure': 'the increased number of myeloblasts in bone marrow and peripheral blood', 'timeFrame': '1 year', 'description': 'the increased number of myeloblasts in bone marrow and peripheral blood'}, {'measure': 'incidence of progression to high-risk MDS or leukemia', 'timeFrame': '1 year', 'description': 'incidence of progression to high-risk MDS or leukemia'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Low/Intermediate Risk-1 MDS']}, 'referencesModule': {'references': [{'pmid': '34778037', 'type': 'DERIVED', 'citation': 'Yang Y, Tang Z, Ji J, Yang C, Chen M, Han B. Recombinant Human Thrombopoietin Accelerates the Recovery of Platelet in Patients With Lower-Risk Myelodysplastic Syndrome: A Proof-of-Concept Study. Front Oncol. 2021 Oct 28;11:721764. doi: 10.3389/fonc.2021.721764. eCollection 2021.'}]}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Confirmed MDS, IPSS low / intermediate risk-1\n2. In the 4 weeks before inclusion, the average value of platelets was ≤ 30 × 10e9 / L, or \\< 50 × 10e9 / L with bleeding events\n3. Patients with EPO due to anemia and G-CSF due to severe neutropenia can be included, and the dosage will not change during trial\n4. Baseline liver and kidney function: ALT / ASL within 3 times normal upper limit, TBIL within 2 times normal upper limit, and creatinine within 2 times normal upper limit\n5. ECOG 0-2 points\n6. Able to sign informed consent\n\nExclusion Criteria:\n\n1. Pregnant or lactating\n2. IPSS intermediate risk-2 / high risk MDS\n3. More than 5% of myeloblasts in bone marrow\n4. Myelofibrosis\n5. Previous transplantation or ATG treatment within 6 months\n6. Previous use of IL-11, TPO or other TPO receptor agonists\n7. Active infection or tumor\n8. Thromboembolic or hemorrhagic disease\n9. Serious heart disease, including unstable angina, congestive heart failure, arrhythmia, 1-year history of myocardial infarction\n10. Intracranial hemorrhage within 4 weeks'}, 'identificationModule': {'nctId': 'NCT04324060', 'briefTitle': 'A Randomized Trial of rhTPO Versus Placebo for Low/Intermediate-1 Risk MDS With Thrombocytopenia', 'organization': {'class': 'OTHER', 'fullName': 'Peking Union Medical College Hospital'}, 'officialTitle': 'A Randomized Trial of Recombinant Human Thrombopoietin Versus Placebo for Low/Intermediate-1 Risk Myelodysplastic Syndromes With Thrombocytopenia', 'orgStudyIdInfo': {'id': 'TPO'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'TPO treatment group', 'description': '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', 'interventionNames': ['Drug: Danazol + rhTPO (recombinant human thrombopoietin injection)']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'control', 'description': 'Danazol 0.2g tid po+ control (sodium chloride)×14d si every month, total course 6 months', 'interventionNames': ['Drug: Danazol + sodium chloride']}], 'interventions': [{'name': 'Danazol + rhTPO (recombinant human thrombopoietin injection)', 'type': 'DRUG', 'description': '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', 'armGroupLabels': ['TPO treatment group']}, {'name': 'Danazol + sodium chloride', 'type': 'DRUG', 'description': 'Danazol 0.2g tid po+ control (sodium chloride)', 'armGroupLabels': ['control']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Beijing', 'country': 'China', 'facility': 'Peking union medical college hospital', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}]}, 'ipdSharingStatementModule': {'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF', 'CSR'], 'timeFrame': '10 years', 'ipdSharing': 'YES', 'description': 'protocol, consent form, clinical data would be available by personal contact', 'accessCriteria': 'personal contact including emails'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Peking Union Medical College Hospital', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor', 'investigatorFullName': 'Bing Han', 'investigatorAffiliation': 'Peking Union Medical College Hospital'}}}}