Ignite Creation Date:
2025-12-24 @ 9:16 PM
Ignite Modification Date:
2026-01-02 @ 2:46 AM
Study NCT ID:
NCT05126004
Status:
NOT_YET_RECRUITING
Last Update Posted:
2021-11-18
First Post:
2021-07-30
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
AZA Combined With NAC for PIT After HSCT
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013921', 'term': 'Thrombocytopenia'}], 'ancestors': [{'id': 'D001791', 'term': 'Blood Platelet Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D000095542', 'term': 'Cytopenia'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D001374', 'term': 'Azacitidine'}, {'id': 'D000111', 'term': 'Acetylcysteine'}], 'ancestors': [{'id': 'D001372', 'term': 'Aza Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D003562', 'term': 'Cytidine'}, {'id': 'D011741', 'term': 'Pyrimidine Nucleosides'}, {'id': 'D011743', 'term': 'Pyrimidines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D009705', 'term': 'Nucleosides'}, {'id': 'D009706', 'term': 'Nucleic Acids, Nucleotides, and Nucleosides'}, {'id': 'D012263', 'term': 'Ribonucleosides'}, {'id': 'D003545', 'term': 'Cysteine'}, {'id': 'D000603', 'term': 'Amino Acids, Sulfur'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-11-01', 'size': 62529, 'label': 'Informed Consent Form', 'hasIcf': True, 'hasSap': False, 'filename': 'ICF_000.pdf', 'typeAbbrev': 'ICF', 'uploadDate': '2021-11-08T03:05', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Arm1: Control Arm2:AZA+NAC'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2021-12-01', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-04', 'completionDateStruct': {'date': '2026-06-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2021-11-08', 'studyFirstSubmitDate': '2021-07-30', 'studyFirstSubmitQcDate': '2021-11-08', 'lastUpdatePostDateStruct': {'date': '2021-11-18', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-11-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-06-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'infection', 'timeFrame': 'From date of randomization until the date of diagnose of infection, assessed up to 100 days', 'description': 'clinical diagnose of infection or evidence of next generation of sequencing（NGS）of body fluid or CT or culture of samples collected from patients'}, {'measure': 'Severe adverse events', 'timeFrame': 'From date of randomization until the date of occurrence of any severe adverse event, assessed up to 100 days', 'description': 'any events that prevent the clinical trial continue besides hepatic injury renal function impairment, et al.'}], 'primaryOutcomes': [{'measure': 'platelet reconstruction', 'timeFrame': 'From date of randomization until the date of platelet reconstruction, assessed up to 100 days', 'description': 'platelet count above 50\\*10\\^9/L independent of transfusion'}], 'secondaryOutcomes': [{'measure': 'overall survival', 'timeFrame': 'From date of randomization until the date of death from any cause, assessed up to 1 year', 'description': 'the time from the date of day 1 post HSCT to the date of death due to any cause'}, {'measure': 'overall response rate', 'timeFrame': 'From date of randomization until the date of platelet count between 30*10^9/L and 50*10^9/L, assessed up to 100 days', 'description': 'platelet count evaluating above 30\\*10\\^9/L but below 50\\*10\\^9/L independent of platelet transfusion'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Azacitidine', 'N-Acetyl-L-cysteine', 'Prolonged Isolated Thrombocytopenia', 'Hematopoietic Stem Cell Transplantation'], 'conditions': ['Thrombocytopenia, Isolated', 'Stem Cell Transplant Complications']}, 'referencesModule': {'references': [{'pmid': '3881142', 'type': 'BACKGROUND', 'citation': 'First LR, Smith BR, Lipton J, Nathan DG, Parkman R, Rappeport JM. Isolated thrombocytopenia after allogeneic bone marrow transplantation: existence of transient and chronic thrombocytopenic syndromes. Blood. 1985 Feb;65(2):368-74.'}, {'pmid': '11302549', 'type': 'BACKGROUND', 'citation': 'Bruno B, Gooley T, Sullivan KM, Davis C, Bensinger WI, Storb R, Nash RA. Secondary failure of platelet recovery after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2001;7(3):154-62. doi: 10.1053/bbmt.2001.v7.pm11302549.'}, {'pmid': '28642072', 'type': 'BACKGROUND', 'citation': 'Wang H, Huang M, Zhao Y, Qi JQ, Chen C, Tang YQ, Qiu HY, Fu CC, Tang XW, Wu DP, Ruan CG, Han Y. Recombinant Human Thrombopoietin Promotes Platelet Engraftment and Improves Prognosis of Patients with Myelodysplastic Syndromes and Aplastic Anemia after Allogeneic Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2017 Oct;23(10):1678-1684. doi: 10.1016/j.bbmt.2017.06.010. Epub 2017 Jun 19.'}, {'pmid': '28484721', 'type': 'BACKGROUND', 'citation': 'Zhao X, Zhao X, Huo M, Fan Q, Pei X, Wang Y, Zhang X, Xu L, Huang X, Liu K, Chang Y. Donor-Specific Anti-Human Leukocyte Antigen Antibodies Predict Prolonged Isolated Thrombocytopenia and Inferior Outcomes of Haploidentical Hematopoietic Stem Cell Transplantation. J Immunol Res. 2017;2017:1043836. doi: 10.1155/2017/1043836. Epub 2017 Apr 16.'}, {'pmid': '20921943', 'type': 'BACKGROUND', 'citation': 'Ramirez P, Brunstein CG, Miller B, Defor T, Weisdorf D. Delayed platelet recovery after allogeneic transplantation: a predictor of increased treatment-related mortality and poorer survival. Bone Marrow Transplant. 2011 Jul;46(7):981-6. doi: 10.1038/bmt.2010.218. Epub 2010 Oct 4.'}, {'pmid': '33646303', 'type': 'BACKGROUND', 'citation': 'Tang Y, Chen J, Liu Q, Chu T, Pan T, Liang J, He XF, Chen F, Yang T, Ma X, Wu X, Hu S, Cao X, Hu X, Hu J, Liu Y, Qi J, Shen Y, Ruan C, Han Y, Wu D. Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial. Blood Adv. 2021 Mar 9;5(5):1250-1258. doi: 10.1182/bloodadvances.2020002790.'}, {'pmid': '20682444', 'type': 'BACKGROUND', 'citation': 'Mohyeldin A, Garzon-Muvdi T, Quinones-Hinojosa A. Oxygen in stem cell biology: a critical component of the stem cell niche. Cell Stem Cell. 2010 Aug 6;7(2):150-61. doi: 10.1016/j.stem.2010.07.007.'}, {'pmid': '23846224', 'type': 'BACKGROUND', 'citation': 'Chen S, Su Y, Wang J. ROS-mediated platelet generation: a microenvironment-dependent manner for megakaryocyte proliferation, differentiation, and maturation. Cell Death Dis. 2013 Jul 11;4(7):e722. doi: 10.1038/cddis.2013.253.'}, {'pmid': '29396859', 'type': 'BACKGROUND', 'citation': 'Kong Y, Shi MM, Zhang YY, Cao XN, Wang Y, Zhang XH, Xu LP, Huang XJ. N-acetyl-L-cysteine improves bone marrow endothelial progenitor cells in prolonged isolated thrombocytopenia patients post allogeneic hematopoietic stem cell transplantation. Am J Hematol. 2018 Jul;93(7):931-942. doi: 10.1002/ajh.25056. Epub 2018 Feb 24.'}, {'pmid': '26181032', 'type': 'BACKGROUND', 'citation': 'Han Y, Tang Y, Chen J, Liang J, Ye C, Ruan C, Wu D. Low-Dose Decitabine for Patients With Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation: A Pilot Therapeutic Study. JAMA Oncol. 2015 May;1(2):249-51. doi: 10.1001/jamaoncol.2014.316. No abstract available.'}, {'pmid': '25566808', 'type': 'BACKGROUND', 'citation': 'Zhou H, Hou Y, Liu X, Qiu J, Feng Q, Wang Y, Zhang X, Min Y, Shao L, Liu X, Li G, Li L, Yang L, Xu S, Ni H, Peng J, Hou M. Low-dose decitabine promotes megakaryocyte maturation and platelet production in healthy controls and immune thrombocytopenia. Thromb Haemost. 2015 May;113(5):1021-34. doi: 10.1160/TH14-04-0342. Epub 2015 Jan 8.'}]}, 'descriptionModule': {'briefSummary': 'Prolonged isolated thrombocytopenia (PIT) that is refractory to conventional treatments has remained a critical complication after allogeneic hematopoietic cell transplantation since decades years ago. Recombinant human thrombopoietin (rhTPO) is the main therapy in clinical practice, but remains low efficiency for PIT. Demethylating drugs have shown thier potential in high-risk myelodysplastic syndromes (MDS) and acte myeloid leukemia (AML). In addition, decitabine has demonstrated its efficacy of over 70% for response rate in treatment for PIT in early clinical trials with elusive mechanism. Preliminary experiments revealed that PIT was associated with abnormality of oxidation microenvironment, and N-Acetyl-L-cysteine (NAC) was the most commonly used antioxidant. Therefore, the investigators have been wondering whether Azacitidine in combination with NAC could improve PIT post HSCT and explore the possible mechanism of it.', 'detailedDescription': 'Prolonged isolated thrombocytopenia (PIT) that is refractory to conventional treatments has remained a critical complication after allogeneic hematopoietic cell transplantation since decades years ago. Recombinant human thrombopoietin (rhTPO) is the main therapy in clinical practice, but remains low efficiency for PIT. Demethylating drugs have shown thier potential in high-risk myelodysplastic syndromes (MDS) and acte myeloid leukemia (AML). In addition, decitabine has demonstrated its efficacy of over 70% for response rate in treatment for PIT in early clinical trials with elusive mechanism. Preliminary experiments revealed that PIT was associated with abnormality of oxidation microenvironment, and N-Acetyl-L-cysteine (NAC) was the most commonly used antioxidant. Furthermore, AZA had shown its potential in immune regulation. Therefore, the investigators have been wondering whether Azacitidine in combination with NAC could improve PIT post HSCT and explore the possible mechanism of it.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Platelet count ≤ 30 × 10\\^9/L persistently at day 60 post-HSCT or later;\n* Neutrophil and hemoglobin were well recovered;\n* Full donor chimerism was achieved;\n\nExclusion Criteria:\n\n* Patients with malignancy relapse;\n* Active infections;\n* Grade Ⅲ-Ⅳ acute graft-versus-host disease or severe chronic graft-versus-host disease according to National Institute of Health criteria;\n* Severe organ damage;\n* Thrombosis requiring treatment;'}, 'identificationModule': {'nctId': 'NCT05126004', 'briefTitle': 'AZA Combined With NAC for PIT After HSCT', 'organization': {'class': 'OTHER', 'fullName': 'The First Affiliated Hospital of Soochow University'}, 'officialTitle': 'Azacitidine (AZA) Combined With N-Acetyl-L-cysteine (NAC) for Prolonged Isolated Thrombocytopenia (PIT) After Hematopoietic Stem Cell Transplantation (HSCT)', 'orgStudyIdInfo': {'id': 'SOOCHOW-HY-2021-2'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'NO_INTERVENTION', 'label': 'Control group', 'description': 'receive supportive care'}, {'type': 'EXPERIMENTAL', 'label': 'AZA+NAC group', 'description': 'AZA 50mg Subcutaneous daily d1-d5 + NAC 600mg oral bid d1-28， 28 days for one cycle', 'interventionNames': ['Drug: Azacitidine', 'Drug: N Acetyl L Cysteine']}], 'interventions': [{'name': 'Azacitidine', 'type': 'DRUG', 'otherNames': ['AZA'], 'description': '28 days for one cycle, evaluation post 3 cycles of treatment. Continue if response exists, otherwise, quit the trial and seek for other therapies.', 'armGroupLabels': ['AZA+NAC group']}, {'name': 'N Acetyl L Cysteine', 'type': 'DRUG', 'otherNames': ['NAC'], 'description': '28 days for one cycle, evaluation post 3 cycles of treatment. Continue if response exists, otherwise, quit the trial and seek for other therapies.', 'armGroupLabels': ['AZA+NAC group']}]}, 'contactsLocationsModule': {'locations': [{'zip': '215000', 'city': 'Suzhou', 'state': 'Jiangsu', 'country': 'China', 'contacts': [{'name': 'Yue Han, PhD, MD', 'role': 'CONTACT', 'email': 'hanyue@suda.edu.cn', 'phone': '8615606133002'}, {'name': 'Meng Zhou, MD', 'role': 'CONTACT', 'email': 'zhoumeng@suda.edu.cn', 'phone': '8615606133002'}], 'facility': 'The first affiliated hospital of Soochow University', 'geoPoint': {'lat': 31.30408, 'lon': 120.59538}}], 'centralContacts': [{'name': 'Yue Han, PhD,MD', 'role': 'CONTACT', 'email': 'hanyue@suda.edu.cn', 'phone': '0512-67781856'}, {'name': 'Depei Wu, PhD,MD', 'role': 'CONTACT'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'The First Affiliated Hospital of Soochow University', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}