Ignite Creation Date:
2025-12-25 @ 2:40 AM
Ignite Modification Date:
2025-12-26 @ 5:05 PM
Study NCT ID:
NCT06872333
Status:
RECRUITING
Last Update Posted:
2025-03-12
First Post:
2025-03-05
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Allo HSCT for High Risk Hemoglobinopathies
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000074323', 'term': 'Alemtuzumab'}, {'id': 'D014916', 'term': 'Whole-Body Irradiation'}, {'id': 'D007332', 'term': 'Insulin Infusion Systems'}, {'id': 'C512542', 'term': 'thymoglobulin'}, {'id': 'C024352', 'term': 'fludarabine'}, {'id': 'D002066', 'term': 'Busulfan'}, {'id': 'D013852', 'term': 'Thiotepa'}, {'id': 'D003520', 'term': 'Cyclophosphamide'}, {'id': 'D015080', 'term': 'Mesna'}, {'id': 'D020123', 'term': 'Sirolimus'}, {'id': 'D016559', 'term': 'Tacrolimus'}, {'id': 'D009173', 'term': 'Mycophenolic Acid'}], 'ancestors': [{'id': 'D061067', 'term': 'Antibodies, Monoclonal, Humanized'}, {'id': 'D000911', 'term': 'Antibodies, Monoclonal'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}, {'id': 'D011878', 'term': 'Radiotherapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D008919', 'term': 'Investigative Techniques'}, {'id': 'D016503', 'term': 'Drug Delivery Systems'}, {'id': 'D004358', 'term': 'Drug Therapy'}, {'id': 'D007260', 'term': 'Infusion Pumps'}, {'id': 'D004864', 'term': 'Equipment and Supplies'}, {'id': 'D001187', 'term': 'Artificial Organs'}, {'id': 'D013523', 'term': 'Surgical Equipment'}, {'id': 'D002072', 'term': 'Butylene Glycols'}, {'id': 'D006018', 'term': 'Glycols'}, {'id': 'D000438', 'term': 'Alcohols'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D008698', 'term': 'Mesylates'}, {'id': 'D000476', 'term': 'Alkanesulfonates'}, {'id': 'D017738', 'term': 'Alkanesulfonic Acids'}, {'id': 'D000473', 'term': 'Alkanes'}, {'id': 'D006839', 'term': 'Hydrocarbons, Acyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D013451', 'term': 'Sulfonic Acids'}, {'id': 'D013456', 'term': 'Sulfur Acids'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D063088', 'term': 'Phosphoramides'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D013721', 'term': 'Triethylenephosphoramide'}, {'id': 'D001388', 'term': 'Aziridines'}, {'id': 'D001389', 'term': 'Azirines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D010752', 'term': 'Phosphoramide Mustards'}, {'id': 'D009588', 'term': 'Nitrogen Mustard Compounds'}, {'id': 'D009150', 'term': 'Mustard Compounds'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D013438', 'term': 'Sulfhydryl Compounds'}, {'id': 'D018942', 'term': 'Macrolides'}, {'id': 'D007783', 'term': 'Lactones'}, {'id': 'D002208', 'term': 'Caproates'}, {'id': 'D000144', 'term': 'Acids, Acyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D005227', 'term': 'Fatty Acids'}, {'id': 'D008055', 'term': 'Lipids'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'OTHER', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 62}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-11-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2032-06-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-03-06', 'studyFirstSubmitDate': '2025-03-05', 'studyFirstSubmitQcDate': '2025-03-06', 'lastUpdatePostDateStruct': {'date': '2025-03-12', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-03-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2030-06-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of Graft versus Host Disease (GvHD)', 'timeFrame': '1 year'}], 'secondaryOutcomes': [{'measure': 'Overall Survival', 'timeFrame': '1 and 2 years', 'description': 'Overall survival post HCT'}, {'measure': 'Grade 3-4 Acute GvHD', 'timeFrame': '2 years', 'description': 'Grade 3-4 acute Graft versus Host Disease (GvHD) at 2 years post HCT.'}, {'measure': 'Chronic Graft versus Host Disease (GvHD) Free', 'timeFrame': '2 years', 'description': 'Chronic Graft versus Host Disease (GvHD) Free at 2 years post HCT.'}, {'measure': 'Failure Free Survival', 'timeFrame': '2 years', 'description': 'Failure Free Survival 2 years post HCT.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Graft Failure', 'Sickle Cell Disease', 'Hemoglobinopathies']}, 'descriptionModule': {'briefSummary': 'A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '55 Years', 'healthyVolunteers': True, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Sickle Cell Disease (SCD)\n* SCD Patients with a fully matched sibling donor (MSD) irrespective of the frequency or severity of symptoms MSD transplant can be considered. Parents/patient must be counseled as to the risks and benefits and provide their voluntary informed consent\n* Transfusion Dependent Alpha- or Beta- Thalassemia\n* Diamond Blackfan Anemia\n* Other Non-Malignant Hematologic Disorders\n* Karnofsky ≥ 60%, Lansky play score ≥ 60. Patients with lower performance score can be considered based on study team's evaluation.\n* Sexually active persons of childbearing potential or persons with partners of childbearing potential must agree to use a highly effective form of contraception during study treatment and for at least 4 months after the transplant.\n\nExclusion Criteria:\n\n* Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 7 days of the start of treatment\n* HIV Positive\n* Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted\n* Known allergy to any of the study components\n* Psychiatric illness/social situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements\n* Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study"}, 'identificationModule': {'nctId': 'NCT06872333', 'briefTitle': 'Allo HSCT for High Risk Hemoglobinopathies', 'organization': {'class': 'OTHER', 'fullName': 'Masonic Cancer Center, University of Minnesota'}, 'officialTitle': 'Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders', 'orgStudyIdInfo': {'id': '2024LS140'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Arm A', 'description': 'Arm A Matched sib regimen - Age 6 -55 (per physician preference for patients over 6) Campath/TBI', 'interventionNames': ['Drug: Alemtuzumab', 'Radiation: Total Body Irradiation', 'Biological: Cell Infusion', 'Drug: Sirolimus', 'Drug: Mycophenolate Mofetil']}, {'type': 'EXPERIMENTAL', 'label': 'Arm B', 'description': 'Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu', 'interventionNames': ['Biological: Cell Infusion', 'Drug: Thymoglobulin', 'Drug: Fludarabine', 'Drug: Busulfan', 'Drug: Tacrolimus', 'Drug: Mycophenolate Mofetil']}, {'type': 'EXPERIMENTAL', 'label': 'Arm C', 'description': 'Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu', 'interventionNames': ['Biological: Cell Infusion', 'Drug: Thymoglobulin', 'Drug: Fludarabine', 'Drug: Busulfan', 'Drug: Tacrolimus', 'Drug: Mycophenolate Mofetil']}, {'type': 'EXPERIMENTAL', 'label': 'Arm D', 'description': 'Arm D: Haploindentical or mismatched unrelated donors (MMUD) - 0-55 years; ATG/Thiotepa/Cyclophosphamide/MESNA/Flu/TBI', 'interventionNames': ['Radiation: Total Body Irradiation', 'Biological: Cell Infusion', 'Drug: Thymoglobulin', 'Drug: Fludarabine', 'Drug: Thiotepa', 'Drug: Cyclophosphamide', 'Drug: Sirolimus', 'Drug: Mycophenolate Mofetil']}], 'interventions': [{'name': 'Alemtuzumab', 'type': 'DRUG', 'otherNames': ['Campath'], 'description': 'Alemtuzumab (Campath) will be administered IV over 2 hours on day -8 to day -4.', 'armGroupLabels': ['Arm A']}, {'name': 'Total Body Irradiation', 'type': 'RADIATION', 'otherNames': ['TBI'], 'description': '400 cGy in 2 split fractions will be administered per Department of RadiationOncology SOPs.', 'armGroupLabels': ['Arm A', 'Arm D']}, {'name': 'Cell Infusion', 'type': 'BIOLOGICAL', 'description': 'On day 0 the cells will be infused per cell source specific institutional guidelines', 'armGroupLabels': ['Arm A', 'Arm B', 'Arm C', 'Arm D']}, {'name': 'Thymoglobulin', 'type': 'DRUG', 'otherNames': ['Rabbit ATG'], 'description': 'ATG will be administered IV every 24 hours beginning on day -8 for all patients.\n\nDosing will be model-based using Bayesian methodology13,14,15. Total doses and total number of doses (1-4 doses) will be determined based on absolute lymphocyte count and weight.', 'armGroupLabels': ['Arm B', 'Arm C', 'Arm D']}, {'name': 'Fludarabine', 'type': 'DRUG', 'description': 'Fludarabine will be administered IV over 1 hour every 24 hours on day -5 to day - 2. The daily dose of fludarabine will be determined by model-based dosing utilizing Bayesian methodology with a cumulative area under the curve (cAUC) of 20 mg\\*hr/L (range 18-22 mg\\*hr/L).', 'armGroupLabels': ['Arm B', 'Arm C', 'Arm D']}, {'name': 'Busulfan', 'type': 'DRUG', 'description': 'Busulfan dosing and administration and therapeutic drug monitoring (TDM) per institutional guidelines. Initial busulfan dosing will be determined by model-based dosing utilizing Bayesian methods with a cumulative area under the curve (cAUC) of 75 mg\\*hr/L.', 'armGroupLabels': ['Arm B', 'Arm C']}, {'name': 'Thiotepa', 'type': 'DRUG', 'description': 'Thiotepa will be administered at a dose 5 mg/kg IV every 12 hours on day - 7 over 2 hours. Patients will undergo thiotepa skin care per institutional guidelines', 'armGroupLabels': ['Arm D']}, {'name': 'Cyclophosphamide', 'type': 'DRUG', 'otherNames': ['Cyclophosphamide with MESNA'], 'description': 'Cyclophosphamide will be administered at a dose of 14.5 mg/kg over 2 hours IV daily on days -6 and -5. Cyclophosphamide dosing is calculated based on actual body weight (ABW).\n\nFor Arm D - Cyclophosphamide 50 mg/kg IV will be administered over 2 hours on days +3 and\n\n+4. Cyclophosphamide dosing for post-transplant is calculated based on ideal body weight (IBW) unless patient weighs less than IBW, in which case actual body weight (ABW) will be used.', 'armGroupLabels': ['Arm D']}, {'name': 'Sirolimus', 'type': 'DRUG', 'description': 'Patients on Arm A and Arm D will receive sirolimus; beginning on day -3 and continuing until day +180 for patients on Arm A or beginning on day +5 and continuing until 1 year post transplant for patients on Arm D.', 'armGroupLabels': ['Arm A', 'Arm D']}, {'name': 'Tacrolimus', 'type': 'DRUG', 'description': 'Patients on Arm B and Arm C will receive tacrolimus, beginning on day -3 and continuing until day +180. Tacrolimus dosing and monitoring will be per institutional guidelines.', 'armGroupLabels': ['Arm B', 'Arm C']}, {'name': 'Mycophenolate Mofetil', 'type': 'DRUG', 'otherNames': ['(MMF)'], 'description': 'MMF will begin on day -3 (Arm A, B \\& C) or day +5 (Arm D). Patients treated on adult service will receive 15 mg/kg (max 1500 mg/dose) given every 12 hours, rounded to nearest 250 mg. Patients on pediatric service will receive 15 mg/kg (max 1000 mg/dose) given every 8 hours. MMF dosing will be monitored and altered as clinically appropriate based on institutional guidelines. MMF will be stopped at day +30 (Arms A, B \\& C) or day +35 (Arm D) or 7 days after engraftment, whichever day is later, if no acute GVHD.', 'armGroupLabels': ['Arm A', 'Arm B', 'Arm C', 'Arm D']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Ashish Gupta, MBBS, MPH', 'role': 'CONTACT'}], 'facility': 'Masonic Cancer Center', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}], 'centralContacts': [{'name': 'Ashish Gupta, MBBS, MPH', 'role': 'CONTACT', 'email': 'gupta461@umn.edu', 'phone': '612-626-2961'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Masonic Cancer Center, University of Minnesota', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}