Ignite Creation Date:
2025-12-24 @ 10:08 PM
Ignite Modification Date:
2025-12-31 @ 2:41 AM
Study NCT ID:
NCT04712435
Status:
UNKNOWN
Last Update Posted:
2021-03-09
First Post:
2021-01-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Efficacy of N-acetylcysteine Versus Placebo as Prophylaxis of Sinusoidal Obstruction Syndrome in Patients Undergoing Hematopoietic Stem Cell Transplantation
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006504', 'term': 'Hepatic Veno-Occlusive Disease'}], 'ancestors': [{'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000111', 'term': 'Acetylcysteine'}], 'ancestors': [{'id': 'D003545', 'term': 'Cysteine'}, {'id': 'D000603', 'term': 'Amino Acids, Sulfur'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR'], 'maskingDescription': 'Matching placebo'}, 'primaryPurpose': 'PREVENTION', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'This is a single-center, randomized, placebo-controlled study to evaluate the efficacy and safety of N-acetylcysteine'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 80}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2021-04', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-01', 'completionDateStruct': {'date': '2023-03-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2021-03-08', 'studyFirstSubmitDate': '2021-01-13', 'studyFirstSubmitQcDate': '2021-01-13', 'lastUpdatePostDateStruct': {'date': '2021-03-09', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-01-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-03-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Presence or absence of SOS', 'timeFrame': 'Day + 30 post HSCT', 'description': 'To evaluate if prophylactic of N-acetylcysteine has an impact on the incidence of Sinusoidal obstruction syndrome (SOS)'}], 'secondaryOutcomes': [{'measure': 'Severity of SOS', 'timeFrame': 'within 30 days of bone marrow transplant', 'description': 'Death as noted in medical record'}, {'measure': 'Sinusoidal obstruction syndrome (SOS) -Free Survival at Day 30 Post-Hematopoietic Stem Cell Transplant (HSCT)', 'timeFrame': '30 Days Post-Transplant', 'description': 'Comparison of efficacy of N-acetylcysteine vs Placebo for the prevention of SOS'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Stem cell transplant', 'Hematopoietic stem cell transplant (HSCT)', 'Sinusoidal obstruction syndrome (SOS)', 'Veno-occlusive disease (VOD)', 'N-acetylcysteine'], 'conditions': ['Sinusoidal Obstruction Syndrome (SOS)', 'Hematopoietic Stem Cell Transplant (HSCT)']}, 'referencesModule': {'references': [{'pmid': '24102514', 'type': 'BACKGROUND', 'citation': 'Dignan FL, Wynn RF, Hadzic N, Karani J, Quaglia A, Pagliuca A, Veys P, Potter MN; Haemato-oncology Task Force of British Committee for Standards in Haematology; British Society for Blood and Marrow Transplantation. BCSH/BSBMT guideline: diagnosis and management of veno-occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation. Br J Haematol. 2013 Nov;163(4):444-57. doi: 10.1111/bjh.12558. Epub 2013 Sep 17.'}, {'pmid': '25798682', 'type': 'BACKGROUND', 'citation': 'Mohty M, Malard F, Abecassis M, Aerts E, Alaskar AS, Aljurf M, Arat M, Bader P, Baron F, Bazarbachi A, Blaise D, Ciceri F, Corbacioglu S, Dalle JH, Duarte RF, Fukuda T, Huynh A, Masszi T, Michallet M, Nagler A, NiChonghaile M, Pagluica T, Peters C, Petersen FB, Richardson PG, Ruutu T, Savani BN, Wallhult E, Yakoub-Agha I, Carreras E. Sinusoidal obstruction syndrome/veno-occlusive disease: current situation and perspectives-a position statement from the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant. 2015 Jun;50(6):781-9. doi: 10.1038/bmt.2015.52. Epub 2015 Mar 23.'}, {'pmid': '26431626', 'type': 'BACKGROUND', 'citation': 'Dalle JH, Giralt SA. Hepatic Veno-Occlusive Disease after Hematopoietic Stem Cell Transplantation: Risk Factors and Stratification, Prophylaxis, and Treatment. Biol Blood Marrow Transplant. 2016 Mar;22(3):400-9. doi: 10.1016/j.bbmt.2015.09.024. Epub 2015 Oct 23.'}, {'pmid': '28401107', 'type': 'BACKGROUND', 'citation': 'Ozdemir ZC, Turhan AB, Eren M, Bor O. Is N-acetylcysteine infusion an effective treatment option in L-asparaginase associated hepatotoxicity? Blood Res. 2017 Mar;52(1):69-71. doi: 10.5045/br.2017.52.1.69. Epub 2017 Mar 27. No abstract available.'}, {'pmid': '18176610', 'type': 'BACKGROUND', 'citation': 'Barkholt L, Remberger M, Hassan Z, Fransson K, Omazic B, Svahn BM, Karlsson H, Brune M, Hassan M, Mattsson J, Ringden O. A prospective randomized study using N-acetyl-L-cysteine for early liver toxicity after allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant. 2008 May;41(9):785-90. doi: 10.1038/sj.bmt.1705969. Epub 2008 Jan 7.'}, {'pmid': '10800069', 'type': 'BACKGROUND', 'citation': 'Ringden O, Remberger M, Lehmann S, Hentschke P, Mattsson J, Klaesson S, Aschan J. N-acetylcysteine for hepatic veno-occlusive disease after allogeneic stem cell transplantation. Bone Marrow Transplant. 2000 May;25(9):993-6. doi: 10.1038/sj.bmt.1702387.'}]}, 'descriptionModule': {'briefSummary': 'Sinusoidal obstruction syndrome (SOS) or hepatic veno-occlusive disease (VOD) is a serious complication that occurs, most often occurring in patients undergoing hematopoietic stem cell transplantation (HSCT), especially in its first thirty days. The morbidity and mortality in this syndrome are considerable, since severe SOS is associated with a mortality of more than 90% in the first hundred days of HSCT.\n\nSome risk factors are modifiable, especially those related to transplantation, but when non-alterable factors are present, preventive measures are needed that can reduce the incidence and / or severity of SOS.', 'detailedDescription': 'This is a single-center, randomized, placebo-controlled study to evaluate the efficacy and safety of N-acetylcysteine as prophylaxis of sinusoidal obstruction syndrome in patients undergoing hematopoietic stem cell transplantation Up to date, no approved medications are available for prophylactic treatment of sinusoidal obstruction syndrome in patients undergoing hematopoietic stem cell transplantation. N-acetylcysteine is a drug that has been long used as a mucolytic. And placebo responses contribute to help assess the appropriateness of randomized clinical trials in determining the size of drug effect.\n\nThe study will enroll patients undergoing hematopoietic stem cell transplantation to receive sequentially one of two treatment: N-acetylcysteine or Placebo.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Men or women\n* Age ≥ 18 years\n* A proven diagnosis of one of the hematological malignancies: Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Chronic Myeloid Leukemia, Chronic Lymphoid Leukemia, Multiple Myeloma, Non-Hodgkin's Lymphoma, Hodgkin's Lymphoma\n* Patients must be scheduled to undergo of Allogeneic hematopoietic transplantation or HLA (Human Leukocyte Antigen) haploidentical related or Unrelated or HLA-matched related or autologous stem cell transplant\n* Patients must be able to understand and sign a written informed consent\n\nExclusion Criteria:\n\n* Patient has a serious active disease or co-morbid medical condition, as judged by the investigator, which would interfere with the conduct of this study\n* Patient, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study\n* Known hypersensitivity to N-acetylcysteine\n* Contraindications to perform any procedure provided for in this study\n* Patients who have already undergone a previous transplant"}, 'identificationModule': {'nctId': 'NCT04712435', 'acronym': 'ALISON', 'briefTitle': 'Efficacy of N-acetylcysteine Versus Placebo as Prophylaxis of Sinusoidal Obstruction Syndrome in Patients Undergoing Hematopoietic Stem Cell Transplantation', 'organization': {'class': 'OTHER', 'fullName': 'Instituto Brasileiro de Controle do Cancer'}, 'officialTitle': 'Prospective, Randomized Study Using N-Acetylcysteine as Prophylaxis of Sinusoidal Obstruction Syndrome in Patients Undergoing Hematopoietic Stem Cell Transplantation', 'orgStudyIdInfo': {'id': '33788420.4.0000.0072'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental: N-acetylcysteine', 'description': 'Sachets containing N-acetylcysteine 200 mg Powder for Oral Solution administered every 8 hours (Daily dose 600 mg/day). is administered on the first day of conditioning and will continue until Day +30 post HSCT or hospital discharge, whichever is sooner', 'interventionNames': ['Drug: N-acetylcysteine']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo Comparator: Placebo', 'description': '200mg granulated solution of matching placebo administered every 8 hours (Daily dose 600 mg/day), is administered on the first day of conditioning and will continue until Day +30 post HSCT or hospital discharge, whichever is sooner', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'N-acetylcysteine', 'type': 'DRUG', 'description': 'Eligible patients will be randomised to receive N-acetylcysteine versus matching placebo', 'armGroupLabels': ['Experimental: N-acetylcysteine']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Eligible patients will be randomised to receive N-acetylcysteine versus matching placebo', 'armGroupLabels': ['Placebo Comparator: Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '03102-002', 'city': 'São Paulo', 'state': 'São Paulo', 'country': 'Brazil', 'contacts': [{'name': 'Jamilla N Cavalcante, MD', 'role': 'CONTACT', 'email': 'millacavalcante@hotmail.com', 'phone': '+55113474-4428'}, {'name': 'Alayne D Yamada, PhD', 'role': 'CONTACT', 'email': 'alayne.pesquisa@ibcc-mooca.org.br', 'phone': '+55113474-4242'}, {'name': 'Jamilla N Cavalcante, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Maria Cristina M Macedo, PhD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Larissa Y Okada, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Lauro Augusto C Leite, MD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Jamilla Neves Cavalcante', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}], 'centralContacts': [{'name': 'Jamilla N Cavalcante, MD', 'role': 'CONTACT', 'email': 'millacavalcante@hotmail.com', 'phone': '+55113474-4428'}, {'name': 'Alayne D Yamada, PhD', 'role': 'CONTACT', 'email': 'alayne.pesquisa@ibcc-mooca.org.br', 'phone': '+55113474-4242'}], 'overallOfficials': [{'name': 'Jamilla N Cavalcante, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'IBCC Oncologia'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Instituto Brasileiro de Controle do Cancer', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Principal Investigator', 'investigatorFullName': 'Jamilla Neves Cavalcante', 'investigatorAffiliation': 'Instituto Brasileiro de Controle do Cancer'}}}}