Ignite Creation Date:
2025-12-24 @ 11:17 PM
Ignite Modification Date:
2026-01-02 @ 10:10 AM
Study NCT ID:
NCT04217356
Status:
RECRUITING
Last Update Posted:
2025-12-18
First Post:
2020-01-02
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of Stem Cell Transplant vs. Non-Transplant Therapies in High-Risk Myelofibrosis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D055728', 'term': 'Primary Myelofibrosis'}, {'id': 'D019046', 'term': 'Bone Marrow Neoplasms'}], 'ancestors': [{'id': 'D009196', 'term': 'Myeloproliferative Disorders'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D019337', 'term': 'Hematologic Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D033581', 'term': 'Stem Cell Transplantation'}, {'id': 'C540383', 'term': 'ruxolitinib'}, {'id': 'D006918', 'term': 'Hydroxyurea'}], 'ancestors': [{'id': 'D017690', 'term': 'Cell Transplantation'}, {'id': 'D064987', 'term': 'Cell- and Tissue-Based Therapy'}, {'id': 'D001691', 'term': 'Biological Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D014180', 'term': 'Transplantation'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}, {'id': 'D014508', 'term': 'Urea'}, {'id': 'D000577', 'term': 'Amides'}, {'id': 'D009930', 'term': 'Organic Chemicals'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'Blood samples for future research related to hematological (blood) cancers and their treatments.'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 90}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2020-08-05', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2026-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-11', 'studyFirstSubmitDate': '2020-01-02', 'studyFirstSubmitQcDate': '2020-01-02', 'lastUpdatePostDateStruct': {'date': '2025-12-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2020-01-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-12-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of patients allocated to hematopoietic stem cell transplantation (HCT)', 'timeFrame': '5 years'}, {'measure': 'Number of patients allocated to best available non-transplant therapies (BAT)', 'timeFrame': '5 years'}, {'measure': 'Overall survival rate of patients who receive hematopoietic stem cell transplantation (HCT)', 'timeFrame': '5 years', 'description': 'Time from study allocation to death or last follow up.'}, {'measure': 'Overall survival rate of patients who receive best available non-transplant therapies (BAT)', 'timeFrame': '5 years', 'description': 'Time from study allocation to death or last follow up.'}], 'secondaryOutcomes': [{'measure': 'Median change in Patient Global Impression of Change (PGIC) score', 'timeFrame': '0 and 36 months', 'description': 'Range from -3 to 3. Positive number equals increase in quality of life.'}, {'measure': 'Median change in MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)', 'timeFrame': '0 and 36 months', 'description': 'Range from 0 to 10. Increase equals worsening of symptoms.'}, {'measure': 'Median change in FACT-BMT Questionnaire', 'timeFrame': '0 and 36 months', 'description': 'Range from 1 to 4. Increase equals increase in quality of life.'}, {'measure': 'Disease-free survival of patients who receive hematopoietic stem cell transplantation (HCT)', 'timeFrame': '5 years', 'description': 'Time from allocation to study arm to death/acute myeloid leukemia transformation or last follow up.'}, {'measure': 'Disease-free survival of patients who receive best available non-transplant therapies (BAT)', 'timeFrame': '5 years', 'description': 'Time from allocation to study arm to death/acute myeloid leukemia transformation or last follow up.'}, {'measure': 'Number of patients who receive hematopoietic stem cell transplantation (HCT) in remission (complete and partial)', 'timeFrame': '3 years'}, {'measure': 'Number of patients who receive best available non-transplant therapies (BAT) in remission (complete and partial)', 'timeFrame': '3 years'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['High-Risk Myelofibrosis', 'Stem Cell', 'Transplantation', 'Myelofibrosis', 'Bone marrow cancer'], 'conditions': ['Myelofibrosis', 'High-Risk Cancer', 'Bone Marrow Cancer']}, 'descriptionModule': {'briefSummary': 'The purpose of this research study is to see how effective hematopoietic stem cell transplantation (HCT) is compared to best available non-transplant therapies (BAT) in patients with high risk myelofibrosis. This will be done by asking participants to choose the treatment that they prefer to receive (HCT or BAT) and then comparing the outcomes of the participants in both treatment groups.', 'detailedDescription': "There is currently little information regarding which treatments are best for patients with myelofibrosis. On one hand, hematopoietic stem cell transplantation (HCT) is potentially curative treatment but is associated with significant risk of complications related to graft failure (the new donor cells does not grow properly after the transplant), side effects such as graft versus host disease (the patient's cells attack the new donor cells), and risk of infections. Non-transplant therapies such as ruxolitinib provide effective symptom control for few months to few years, but are not curative in nature. As such, this study will compare the effectiveness of HCT versus best available non-transplant therapies (BAT) in patients with high risk myelofibrosis.\n\nThis is an observational study, meaning that participants will be followed to assess the effects of their treatment, but no intervention (treatments) will be given as a part of this study."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients with high-risk myelofibrosis including pre-fibrotic primary myelofibrosis (pre-fibrotic primary myelofibrosis), overt primary myelofibrosis, post-polycythemia myelofibrosis or post-essential thrombocythemia myelofibrosis.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nRecruitment Part:\n\n* Documented diagnosis of pre-fibrotic primary myelofibrosis (pre-fibrotic PMF), overt PMF, post-polycythemia MF (PPV-MF) or post-essential thrombocythemia MF (PET-MF) confirmed by bone marrow biopsy\n* Have been tested or have results available for phenotypic driver mutations (JAK2/CALR/MPL) and high molecular risk (HMR) mutations using a broad myeloid malignancies targeted gene panel.\n* Eastern Cooperative Oncology Group (ECOG) performance status 0-2\n* Able to provide informed consent\n* Adequate organ function\n* Donor search initiated or patient is agreeable to donor search\n* Meet the definition/criteria for high-risk myelofibrosis\n\nStudy Arm Allocation:\n\n* Grade of fibrosis on bone marrow biopsy available according to World Health Organization (WHO) criteria\n* Results available for phenotypic driver mutations (JAK2/CALR/MPL) and targeted sequencing results using a broad myeloid malignancy panel with a minimal requirement to include results on High molecular risk (HMR) mutations such as ASXL1/EZH2/IDH1/IDH2/SRSF2/U2AF1/TP53\n* ECOG performance status 0-2\n* Adequate organ function\n* Information on donor search and donor type available\n\nExclusion Criteria:\n\nRecruitment Part:\n\n* Blasts in peripheral blood or bone marrow ≥10%\n* For patients already on ruxolitinib at study entry, and meet the criteria of ruxolitinib failure\n* Previous history of transformation to blast phase or acute myeloid leukemia\n* Received allogeneic stem cell transplant for myeloproliferative neoplasm\n* Presence of an active uncontrolled infection\n* Myocardial infarction in the preceding 3 months\n* Active hepatitis A, B or C\n* Known human immunodeficiency virus (HIV) positive\n* History of active malignancy in the previous 2 years, except basal cell carcinoma or squamous cell carcinoma of skin or stage 0 cervical cancer\n* Any psychiatric illness or social circumstances or significant co-morbid conditions that will prevent patient from proceeding to allogeneic hematopoietic cell transplantation.\n* Pregnant or breastfeeding women\n\nStudy Arm Allocation:\n\n* Blasts in peripheral blood or bone marrow ≥10%\n* Meet the criteria of ruxolitinib failure\n* Presence of an active uncontrolled infection\n* Myocardial infarction in the preceding 3 months\n* Active hepatitis A, B or C\n* Known HIV positive\n* History of active malignancy in the previous 2 years, except basal cell carcinoma or squamous cell carcinoma of skin or stage 0 cervical cancer\n* Pregnant or breastfeeding women\n* Any psychiatric illness or social circumstances or significant co-morbid conditions that will prevent patient from proceeding to allogeneic hematopoietic cell transplantation.\n* Time between registration and allocation of study arm \\>24 weeks'}, 'identificationModule': {'nctId': 'NCT04217356', 'acronym': 'ALLO-BAT', 'briefTitle': 'Study of Stem Cell Transplant vs. Non-Transplant Therapies in High-Risk Myelofibrosis', 'organization': {'class': 'OTHER', 'fullName': 'University Health Network, Toronto'}, 'officialTitle': 'A Patient Preferences-Controlled Study of Allogeneic Hematopoietic Cell Transplantation Versus Best Available Non-Transplant Therapies in Patients With High-Risk Myelofibrosis (ALLO-BAT Study)', 'orgStudyIdInfo': {'id': '19-6362'}, 'secondaryIdInfos': [{'id': 'ALLO-BAT', 'type': 'OTHER', 'domain': 'Princess Margaret Cancer Centre'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Hematopoietic stem cell transplant (HCT)', 'description': 'Standard of care hematopoietic stem cell transplant with a matched donor.', 'interventionNames': ['Biological: Hematopoietic stem cell transplant']}, {'label': 'Best available non-transplant therapies (BAT)', 'description': 'Standard of care treatment with a janus kinase (JAK) inhibitor drug called ruxolitinib or treatment with an antimetabolite drug called hydroxyurea.', 'interventionNames': ['Drug: Ruxolitinib', 'Drug: Hydroxyurea']}], 'interventions': [{'name': 'Hematopoietic stem cell transplant', 'type': 'BIOLOGICAL', 'description': 'Intravenous infusion of hematopoietic stem cells from a donor.', 'armGroupLabels': ['Hematopoietic stem cell transplant (HCT)']}, {'name': 'Ruxolitinib', 'type': 'DRUG', 'otherNames': ['JAKAVI'], 'description': 'Ruxolitinib is type of drug called a janus kinase (JAK) inhibitor. Ruxolitinib is taken orally (by mouth).', 'armGroupLabels': ['Best available non-transplant therapies (BAT)']}, {'name': 'Hydroxyurea', 'type': 'DRUG', 'description': 'Hydroxyurea is a type of drug called an antimetabolite. Hydroxyurea is taken orally (by mouth).', 'armGroupLabels': ['Best available non-transplant therapies (BAT)']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'T2N4N2', 'city': 'Calgary', 'state': 'Alberta', 'status': 'RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Sonia Cerquozzi, M.D.', 'role': 'CONTACT', 'phone': '403-944-5948'}, {'name': 'Sonia Cerquozzi, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Tom Baker Cancer Centre', 'geoPoint': {'lat': 51.05011, 'lon': -114.08529}}, {'zip': 'T6G2G3', 'city': 'Edmonton', 'state': 'Alberta', 'status': 'NOT_YET_RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Elena Liew, M.D.', 'role': 'CONTACT', 'phone': '780-417-1584'}, {'name': 'Elena Liew, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Cross Cancer Institute', 'geoPoint': {'lat': 53.55014, 'lon': -113.46871}}, {'zip': 'V6E1M7', 'city': 'Vancouver', 'state': 'British Columbia', 'status': 'NOT_YET_RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Lynda Foltz, M.D.', 'role': 'CONTACT', 'phone': '604-682-2344', 'phoneExt': '64986'}, {'name': 'Lynda Foltz, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "St. Paul's Hospital", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': 'B3H2Y9', 'city': 'Halifax', 'state': 'Nova Scotia', 'status': 'NOT_YET_RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Mahmoud Elsawy, M.D.', 'role': 'CONTACT', 'phone': '902-473-7006'}, {'name': 'Mahmoud Elsawy, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Nova Scotia Health Authority', 'geoPoint': {'lat': 44.64269, 'lon': -63.57688}}, {'zip': 'M5G2M9', 'city': 'Toronto', 'state': 'Ontario', 'status': 'RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Vikas Gupta, M.D.', 'role': 'CONTACT', 'phone': '416-946-2885'}, {'name': 'Vikas Gupta, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Princess Margaret Cancer Centre', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}], 'centralContacts': [{'name': 'Vikas Gupta, M.D.', 'role': 'CONTACT', 'email': 'vikas.gupta@uhn.ca', 'phone': '416-946-2885'}], 'overallOfficials': [{'name': 'Vikas Gupta, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Princess Margaret Cancer Centre'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University Health Network, Toronto', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}