Ignite Creation Date:
2025-12-24 @ 3:30 PM
Ignite Modification Date:
2025-12-27 @ 4:50 PM
Study NCT ID:
NCT01652092
Status:
RECRUITING
Last Update Posted:
2025-02-03
First Post:
2012-07-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D016511', 'term': 'Severe Combined Immunodeficiency'}, {'id': 'C538361', 'term': 'Reticular dysgenesis'}, {'id': 'D014923', 'term': 'Wiskott-Aldrich Syndrome'}, {'id': 'D017074', 'term': 'Common Variable Immunodeficiency'}, {'id': 'D006105', 'term': 'Granulomatous Disease, Chronic'}, {'id': 'D053306', 'term': 'Hyper-IgM Immunodeficiency Syndrome'}, {'id': 'D008232', 'term': 'Lymphoproliferative Disorders'}, {'id': 'D051359', 'term': 'Lymphohistiocytosis, Hemophagocytic'}, {'id': 'D002609', 'term': 'Chediak-Higashi Syndrome'}, {'id': 'D006646', 'term': 'Histiocytosis, Langerhans-Cell'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}], 'ancestors': [{'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D049914', 'term': 'DNA Repair-Deficiency Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D008231', 'term': 'Lymphopenia'}, {'id': 'D007970', 'term': 'Leukopenia'}, {'id': 'D000095542', 'term': 'Cytopenia'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D007960', 'term': 'Leukocyte Disorders'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D010585', 'term': 'Phagocyte Bactericidal Dysfunction'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D004406', 'term': 'Dysgammaglobulinemia'}, {'id': 'D001796', 'term': 'Blood Protein Disorders'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D007160', 'term': 'Immunoproliferative Disorders'}, {'id': 'D015616', 'term': 'Histiocytosis, Non-Langerhans-Cell'}, {'id': 'D015614', 'term': 'Histiocytosis'}, {'id': 'D000417', 'term': 'Albinism'}, {'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D017563', 'term': 'Lung Diseases, Interstitial'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000074323', 'term': 'Alemtuzumab'}, {'id': 'D003520', 'term': 'Cyclophosphamide'}, {'id': 'D002066', 'term': 'Busulfan'}, {'id': 'D033581', 'term': 'Stem Cell Transplantation'}, {'id': 'C042382', 'term': 'fludarabine phosphate'}, {'id': 'D008558', 'term': 'Melphalan'}, {'id': 'D015080', 'term': 'Mesna'}, {'id': 'D012964', 'term': 'Sodium'}], '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': 'D010752', 'term': 'Phosphoramide Mustards'}, {'id': 'D009588', 'term': 'Nitrogen Mustard Compounds'}, {'id': 'D009150', 'term': 'Mustard Compounds'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D063088', 'term': 'Phosphoramides'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D002072', 'term': 'Butylene Glycols'}, {'id': 'D006018', 'term': 'Glycols'}, {'id': 'D000438', 'term': 'Alcohols'}, {'id': 'D008698', 'term': 'Mesylates'}, {'id': 'D000476', 'term': 'Alkanesulfonates'}, {'id': 'D017738', 'term': 'Alkanesulfonic Acids'}, {'id': 'D000473', 'term': 'Alkanes'}, {'id': 'D006839', 'term': 'Hydrocarbons, Acyclic'}, {'id': 'D013451', 'term': 'Sulfonic Acids'}, {'id': 'D013456', 'term': 'Sulfur Acids'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'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': 'D010649', 'term': 'Phenylalanine'}, {'id': 'D024322', 'term': 'Amino Acids, Aromatic'}, {'id': 'D000598', 'term': 'Amino Acids, Cyclic'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D013438', 'term': 'Sulfhydryl Compounds'}, {'id': 'D008672', 'term': 'Metals, Alkali'}, {'id': 'D004602', 'term': 'Elements'}, {'id': 'D007287', 'term': 'Inorganic Chemicals'}, {'id': 'D019565', 'term': 'Metals, Light'}, {'id': 'D008670', 'term': 'Metals'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 30}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2012-09-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-01', 'completionDateStruct': {'date': '2026-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-01-31', 'studyFirstSubmitDate': '2012-07-25', 'studyFirstSubmitQcDate': '2012-07-26', 'lastUpdatePostDateStruct': {'date': '2025-02-03', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2012-07-27', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2025-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Neutrophil Engraftment', 'timeFrame': 'Day 42', 'description': 'Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm3 (0.5 x 109/L) or greater.'}], 'secondaryOutcomes': [{'measure': 'Incidence of Graft Failure', 'timeFrame': 'Day 100', 'description': 'Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets.'}, {'measure': 'Incidence of Chimerism', 'timeFrame': 'Day 100, 6 Months, 1 Year', 'description': 'a state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease.'}, {'measure': 'Incidence of Acute Graft-Versus-Host Disease', 'timeFrame': 'Day 100', 'description': 'Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.'}, {'measure': 'Incidence of Chronic Graft-Versus-Host Disease', 'timeFrame': '6 Months and 1 Year', 'description': 'Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.'}, {'measure': 'Incidence of Transplant-Related Mortality', 'timeFrame': '6 Months', 'description': 'In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.'}, {'measure': 'Disease-Free Survival', 'timeFrame': '6 Months', 'description': 'the length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works.'}, {'measure': 'Overall Survival', 'timeFrame': '6 Months', 'description': 'Overall survival will be defined as time from enrollment to date of death or censored at the date of last documented contact for patients still alive.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['immunodeficiency disorder', 'histiocytic disorder'], 'conditions': ['SCID', "Omenn's Syndrome", 'Reticular Dysgenesis', 'Wiskott-Aldrich Syndrome', 'Bare Lymphocyte Syndrome', 'Common Variable Immunodeficiency', 'Chronic Granulomatous Disease', 'CD40 Ligand Deficiency', 'Hyper IgM Syndrome', 'X-linked Lymphoproliferative Disease', 'Hemophagocytic Lymphohistiocytosis', 'Griscelli Syndrome', 'Chediak-Higashi Syndrome', "Langerhan's Cell Histiocytosis"]}, 'descriptionModule': {'briefSummary': 'This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.', 'detailedDescription': 'Based on diagnosis and clinical history, a determination of the most appropriate regimen will be made based on the following prep plans:\n\nArm A: Fully Myeloablative Preparative Regimen, Arm B: Reduced Toxicity Ablative Preparative Regimen, Arm C: Reduced Intensity Conditioning, Arm D: No Preparative Regimen'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '50 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Diagnosis of immunodeficiency or histiocytic disorder including the following:\n\n * Severe combined immunodeficiency (SCID - all variants)\n * Second bone marrow transplant (BMT) for SCID (after graft rejection)\n * Omenn's Syndrome\n * Reticular dysgenesis\n * Wiskott-Aldrich syndrome\n * Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte syndrome)\n * Hyper IgM Syndrome (CD40 Ligand Deficiency)\n * Common variable immunodeficiency (CVID) with severe phenotype\n * Chronic Granulomatous Disease (CGD)\n * Other severe Combined Immune Deficiencies (CID)\n * Hemophagocytic Lymphohistiocytosis (HLH)\n * X-linked Lymphoproliferative Disease (XLP)\n * Chediak-Higashi Syndrome (CHS)\n * Griscelli Syndrome\n * Langerhans Cell Histiocytosis (LCH)\n* Acceptable stem cell sources include:\n\n * HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow\n * HLA identical or up to a 1 antigen mismatched unrelated BM donor\n * Sibling donor cord blood with acceptable HLA match and cell dose as per current institutional standards\n * Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum cell dose of \\>5 x 10\\^7 nucleated cells/kg as per current institutional guidelines\n * Double unrelated umbilical cord blood units that are:\n\n * up to 2 antigen mismatched to the patient\n * up to 2 antigen mismatched to each other\n * minimum cell dose of at least one single unit must be ≥ 3.5 x 10\\^7 nucleated cells/kg\n * combined dose of both units must provide a total cell dose of ≥ 5 x 10\\^7 nucleated cells/kg\n* Age: 0 to 50 years\n* Adequate organ function and performance status.\n\nExclusion Criteria\n\n* pregnant or breastfeeding\n* active, uncontrolled infection and/or HIV positive\n* acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy"}, 'identificationModule': {'nctId': 'NCT01652092', 'briefTitle': 'Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies', 'organization': {'class': 'OTHER', 'fullName': 'Masonic Cancer Center, University of Minnesota'}, 'officialTitle': 'Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies', 'orgStudyIdInfo': {'id': '2012OC055'}, 'secondaryIdInfos': [{'id': 'MT2012-10C', 'type': 'OTHER', 'domain': 'Blood and Marrow Transplantation Program'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'Arm A: Fully Myeloablative regimen', 'description': 'For use in patients with diseases including Wiskott-Aldrich syndrome, MHC Class II deficiency, hypomorphic SCID, etc. Receives Alemtuzumab 0.3 mg/kg intravenously (IV) on days -12 through -10, cyclophosphamide 50 mg/kg IV plus MESNA on days -9 through -6, busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2 and stem cell infusion on day 0.', 'interventionNames': ['Drug: Alemtuzumab 0.3 mg', 'Drug: Cyclophosphamide', 'Drug: Busulfan', 'Biological: Stem Cell Transplantation', 'Drug: MESNA']}, {'type': 'OTHER', 'label': 'Arm B: Reduced Toxicity Ablative Regimen', 'description': 'For use in patients with diseases including SCID, CGD, CHS and other CID. Receives Alemtuzumab 0.3 mg/kg intravenously (IV) on days -12 through -10, busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6, fludarabine phosphate 40 mg/m\\^2 IV on days -5 through -2 and stem cell infusion on day 0.', 'interventionNames': ['Drug: Alemtuzumab 0.3 mg', 'Biological: Stem Cell Transplantation', 'Drug: Fludarabine phosphate 40 mg', 'Drug: Busulfan']}, {'type': 'OTHER', 'label': 'Arm C: Reduced Intensity Conditioning', 'description': 'For use in patients with diseases including HLH. Receives Alemtuzumab 0.2 mg/kg intravenously (IV) on days -14 through -10, fludarabine phosphate 30 mg/m\\^2 IV on days -8 through -4, melphalan 140 mg/m\\^2 IV on day -3 and stem cell infusion on day 0.', 'interventionNames': ['Biological: Stem Cell Transplantation', 'Drug: Melphalan', 'Drug: Alemtuzumab 0.2 mg', 'Drug: Fludarabine phosphate 30 mg']}, {'type': 'OTHER', 'label': 'Arm D: No Preparative Regimen', 'description': 'For use in patients with complete SCID phenotype with no evidence of maternal engraftment or residual immune function who will be receiving their stem cell transplantation from a genotypically matched donor.', 'interventionNames': ['Biological: Stem Cell Transplantation']}], 'interventions': [{'name': 'Alemtuzumab 0.3 mg', 'type': 'DRUG', 'otherNames': ['Campath-1H'], 'description': '0.3 mg/kg intravenously (IV) on days -12 through -10', 'armGroupLabels': ['Arm A: Fully Myeloablative regimen', 'Arm B: Reduced Toxicity Ablative Regimen']}, {'name': 'Cyclophosphamide', 'type': 'DRUG', 'otherNames': ['Cytoxan'], 'description': 'cyclophosphamide 50 mg/kg IV on days -9 through -6', 'armGroupLabels': ['Arm A: Fully Myeloablative regimen']}, {'name': 'Busulfan', 'type': 'DRUG', 'otherNames': ['Myerlan'], 'description': 'busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2', 'armGroupLabels': ['Arm A: Fully Myeloablative regimen']}, {'name': 'Stem Cell Transplantation', 'type': 'BIOLOGICAL', 'description': 'Unrelated donor bone marrow will be collected in the usual manner using established parameters determined by the National Marrow Donor Program. A minimum of 3 x 10\\^8 nucleated cells/kg recipient weight will be collected with a goal of ≥ 5 x 10\\^8 nucleated cells/kg recipient weight.\n\nUmbilical cord blood selection will be per the current University of Minnesota Cord Blood Unit Selection algorithm. One or two units may be used to obtain the minimum cell dose. One of the UCB units selected for transplantation must contain ≥ 3.5 x 10\\^7 nucleated cells/kg recipient weight based on cell numbers at time of cryopreservation, and the total combined cell dose of both units must be \\> 5.0 x 10\\^7 nucleated cells/kg.', 'armGroupLabels': ['Arm A: Fully Myeloablative regimen', 'Arm B: Reduced Toxicity Ablative Regimen', 'Arm C: Reduced Intensity Conditioning', 'Arm D: No Preparative Regimen']}, {'name': 'Fludarabine phosphate 40 mg', 'type': 'DRUG', 'otherNames': ['Fludara'], 'description': '40 mg/m\\^2 IV on days -5 through -2 (for children \\< 6 months and/or \\< 10 kg weight dose at 1.33 mg/kg)', 'armGroupLabels': ['Arm B: Reduced Toxicity Ablative Regimen']}, {'name': 'Melphalan', 'type': 'DRUG', 'otherNames': ['Alkeran'], 'description': '140 mg/m\\^2 IV on day -3', 'armGroupLabels': ['Arm C: Reduced Intensity Conditioning']}, {'name': 'Alemtuzumab 0.2 mg', 'type': 'DRUG', 'otherNames': ['Campath 1-H'], 'description': '0.2 mg/kg intravenously (IV) on days -14 through -10', 'armGroupLabels': ['Arm C: Reduced Intensity Conditioning']}, {'name': 'Busulfan', 'type': 'DRUG', 'otherNames': ['Myerlan'], 'description': 'busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6', 'armGroupLabels': ['Arm B: Reduced Toxicity Ablative Regimen']}, {'name': 'Fludarabine phosphate 30 mg', 'type': 'DRUG', 'otherNames': ['Fludara'], 'description': 'fludarabine 30 mg/m\\^2 IV on days -8 through -4', 'armGroupLabels': ['Arm C: Reduced Intensity Conditioning']}, {'name': 'MESNA', 'type': 'DRUG', 'otherNames': ['mercaptoethane sulfonate Na (Na being the symbol for sodium)', 'Mesnex'], 'description': 'administered as per the standard institutional protocol.', 'armGroupLabels': ['Arm A: Fully Myeloablative regimen']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Christen Ebens, MD', 'role': 'CONTACT', 'email': 'ebens012@umn.edu', 'phone': '612-626-2778'}, {'name': 'Christen Ebens, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Masonic Cancer Center, University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}], 'centralContacts': [{'name': 'Christen Ebens, MD', 'role': 'CONTACT', 'email': 'ebens012@umn.edu', 'phone': '612-626-2778'}], 'overallOfficials': [{'name': 'Christen Ebens, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Masonic Cancer Center, University of Minnesota'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Masonic Cancer Center, University of Minnesota', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}