Ignite Creation Date:
2025-12-24 @ 8:02 PM
Ignite Modification Date:
2026-01-04 @ 3:43 AM
Study NCT ID:
NCT02282904
Status:
TERMINATED
Last Update Posted:
2020-05-12
First Post:
2014-11-04
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006105', 'term': 'Granulomatous Disease, Chronic'}], 'ancestors': [{'id': 'D010585', 'term': 'Phagocyte Bactericidal Dysfunction'}, {'id': 'D007960', 'term': 'Leukocyte Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D020123', 'term': 'Sirolimus'}, {'id': 'D003520', 'term': 'Cyclophosphamide'}, {'id': 'C024352', 'term': 'fludarabine'}, {'id': 'C042382', 'term': 'fludarabine phosphate'}, {'id': 'D002066', 'term': 'Busulfan'}], 'ancestors': [{'id': 'D018942', 'term': 'Macrolides'}, {'id': 'D007783', 'term': 'Lactones'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'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': '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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ekang@niaid.nih.gov', 'phone': '3014027567', 'title': 'Elizabeth Kang', 'organization': 'NIAID'}, 'certainAgreement': {'piSponsorEmployee': True, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Adverse Events were collected over 5 years (2015 to 2019)', 'eventGroups': [{'id': 'EG000', 'title': 'CGD Recipient', 'description': 'CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described\n\nSirolimus: For pediatric patients: Begin sirolimus 1 mg/m2 PO q4h for 3 doses, then 1 mg/m2 once a day (QD). For adult patients, begin sirolimus 5 mg PO q4h for 3 doses, then 5 mg once a day (QD).\n\nDoses may be adjusted to maintain trough levels between 8-14 ng/ml. Recipients will take sirolimus from Day +5 to at least Day 100 (minimum).\n\nDonor peripheral blood stem cells.: Infuse donor graft.\n\nCyclophosphamide post transplant: 50 mg/kg/d IV infused over 90 minutes. Day +3 and +4\n\nTotal body 200cGy: Day -1\n\nCyclophosphamide: 14.5 mg/kg IV over one hour Day -6 and -5\n\nFludarabine: 30 mg/m2 over 30 minutes Day -6 through Day -2\n\nBusulfan: Busulfan 3.2 mg/kg IV once daily over 2-3 hours Day -4,-3,-2', 'otherNumAtRisk': 7, 'deathsNumAtRisk': 7, 'otherNumAffected': 7, 'seriousNumAtRisk': 7, 'deathsNumAffected': 2, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Mild GvHD', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 7, 'numAffected': 7}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Severe Acute GvHD', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hemorrhagic Cystitis', 'notes': 'Development of irritation and bleeding from the bladder due to infection and/or chemotherapy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Post transplant lymphoproliferative disease', 'notes': 'Development of a clonal outgrowth of cells due to suppression of the immune system', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CGD Recipient', 'description': 'CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described\n\nSirolimus: For pediatric patients: Begin sirolimus 1 mg/m2 PO q4h for 3 doses, then 1 mg/m2 once a day (QD). For adult patients, begin sirolimus 5 mg PO q4h for 3 doses, then 5 mg once a day (QD).\n\nDoses may be adjusted to maintain trough levels between 8-14 ng/ml. Recipients will take sirolimus from Day +5 to at least Day 100 (minimum).\n\nDonor peripheral blood stem cells.: Infuse donor graft.\n\nCyclophosphamide post transplant: 50 mg/kg/d IV infused over 90 minutes. Day +3 and +4\n\nTotal body 200cGy: Day -1\n\nCyclophosphamide: 14.5 mg/kg IV over one hour Day -6 and -5\n\nFludarabine: 30 mg/m2 over 30 minutes Day -6 through Day -2\n\nBusulfan: Busulfan 3.2 mg/kg IV once daily over 2-3 hours Day -4,-3,-2'}], 'classes': [{'title': 'Greater than 20% donor chimerism', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Resolution of inflammation or infection', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '5 years', 'description': 'Patient will have donor chimerism of greater than 20% and resolution of infection or autoimmunity at end of follow up', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'To Determine the Safety of This Allogeneic HSCT Approach in Patients With CGD Including Transplant Related Toxicity, the Incidence of Acute and Chronic Graft-versus-host Disease, Immune Reconstitution, Overalland Disease-free Survival.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CGD Recipient', 'description': 'CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described\n\nSirolimus: For pediatric patients: Begin sirolimus 1 mg/m2 PO q4h for 3 doses, then 1 mg/m2 once a day (QD). For adult patients, begin sirolimus 5 mg PO q4h for 3 doses, then 5 mg once a day (QD).\n\nDoses may be adjusted to maintain trough levels between 8-14 ng/ml. Recipients will take sirolimus from Day +5 to at least Day 100 (minimum).\n\nDonor peripheral blood stem cells.: Infuse donor graft.\n\nCyclophosphamide post transplant: 50 mg/kg/d IV infused over 90 minutes. Day +3 and +4\n\nTotal body 200cGy: Day -1\n\nCyclophosphamide: 14.5 mg/kg IV over one hour Day -6 and -5\n\nFludarabine: 30 mg/m2 over 30 minutes Day -6 through Day -2\n\nBusulfan: Busulfan 3.2 mg/kg IV once daily over 2-3 hours Day -4,-3,-2'}], 'classes': [{'title': 'Number of patients with stable chimerism at 1 year', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Patients with normal DHR at 1 year', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Patients with Acute (Grade 3 or higher) GvHD', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Patients with Chronic GvHD', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Overall Survival', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Disease Free Survival', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year post transplant', 'description': '1\\. Stable chimerism as indicated by 30-50% myeloid engraftment and 50% lymphoid engraftment as assessed by 1 year post transplant. 2. Immune reconstitution levels with DHR as a marker of normal neutrophil function by 1 year post transplant. 3. GvHD grades of less than 3.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'CGD Recipient', 'description': 'CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described\n\nSirolimus: For pediatric patients: Begin sirolimus 1 mg/m2 PO q4h for 3 doses, then 1 mg/m2 once a day (QD). For adult patients, begin sirolimus 5 mg PO q4h for 3 doses, then 5 mg once a day (QD).\n\nDoses may be adjusted to maintain trough levels between 8-14 ng/ml. Recipients will take sirolimus from Day +5 to at least Day 100 (minimum).\n\nDonor peripheral blood stem cells.: Infuse donor graft.\n\nCyclophosphamide post transplant: 50 mg/kg/d IV infused over 90 minutes. Day +3 and +4\n\nTotal body 200cGy: Day -1\n\nCyclophosphamide: 14.5 mg/kg IV over one hour Day -6 and -5\n\nFludarabine: 30 mg/m2 over 30 minutes Day -6 through Day -2\n\nBusulfan: Busulfan 3.2 mg/kg IV once daily over 2-3 hours Day -4,-3,-2'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': 'Patient accrual was stopped early, and the protocol closed after completing followup', 'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'CGD Recipient', 'description': 'CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described\n\nSirolimus: For pediatric patients: Begin sirolimus 1 mg/m2 PO q4h for 3 doses, then 1 mg/m2 once a day (QD). For adult patients, begin sirolimus 5 mg PO q4h for 3 doses, then 5 mg once a day (QD).\n\nDoses may be adjusted to maintain trough levels between 8-14 ng/ml. Recipients will take sirolimus from Day +5 to at least Day 100 (minimum).\n\nDonor peripheral blood stem cells.: Infuse donor graft.\n\nCyclophosphamide post transplant: 50 mg/kg/d IV infused over 90 minutes. Day +3 and +4\n\nTotal body 200cGy: Day -1\n\nCyclophosphamide: 14.5 mg/kg IV over one hour Day -6 and -5\n\nFludarabine: 30 mg/m2 over 30 minutes Day -6 through Day -2\n\nBusulfan: Busulfan 3.2 mg/kg IV once daily over 2-3 hours Day -4,-3,-2'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '19.42', 'spread': '4.8', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}, {'title': 'India', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Infection present', 'classes': [{'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Autoimmunity present', 'classes': [{'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-04-04', 'size': 457167, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2020-03-10T23:35', 'hasProtocol': True}, {'date': '2018-06-18', 'size': 253904, 'label': 'Informed Consent Form', 'hasIcf': True, 'hasSap': False, 'filename': 'ICF_001.pdf', 'typeAbbrev': 'ICF', 'uploadDate': '2020-03-10T23:40', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 7}}, 'statusModule': {'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2014-10-23'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-12-10', 'completionDateStruct': {'date': '2019-12-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-04-30', 'studyFirstSubmitDate': '2014-11-04', 'resultsFirstSubmitDate': '2020-04-13', 'studyFirstSubmitQcDate': '2014-11-04', 'lastUpdatePostDateStruct': {'date': '2020-05-12', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-04-21', 'studyFirstPostDateStruct': {'date': '2014-11-05', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-05-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-04-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD', 'timeFrame': '5 years', 'description': 'Patient will have donor chimerism of greater than 20% and resolution of infection or autoimmunity at end of follow up'}], 'secondaryOutcomes': [{'measure': 'To Determine the Safety of This Allogeneic HSCT Approach in Patients With CGD Including Transplant Related Toxicity, the Incidence of Acute and Chronic Graft-versus-host Disease, Immune Reconstitution, Overalland Disease-free Survival.', 'timeFrame': '1 year post transplant', 'description': '1\\. Stable chimerism as indicated by 30-50% myeloid engraftment and 50% lymphoid engraftment as assessed by 1 year post transplant. 2. Immune reconstitution levels with DHR as a marker of normal neutrophil function by 1 year post transplant. 3. GvHD grades of less than 3.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Chronic Granulomatous Disease', 'Halo-Identical Protocol', 'Transplant'], 'conditions': ['Chronic Granulomatous Disease']}, 'referencesModule': {'references': [{'pmid': '18489989', 'type': 'BACKGROUND', 'citation': "Luznik L, O'Donnell PV, Symons HJ, Chen AR, Leffell MS, Zahurak M, Gooley TA, Piantadosi S, Kaup M, Ambinder RF, Huff CA, Matsui W, Bolanos-Meade J, Borrello I, Powell JD, Harrington E, Warnock S, Flowers M, Brodsky RA, Sandmaier BM, Storb RF, Jones RJ, Fuchs EJ. HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant. 2008 Jun;14(6):641-50. doi: 10.1016/j.bbmt.2008.03.005."}, {'pmid': '21921045', 'type': 'BACKGROUND', 'citation': 'Reisner Y, Hagin D, Martelli MF. Haploidentical hematopoietic transplantation: current status and future perspectives. Blood. 2011 Dec 1;118(23):6006-17. doi: 10.1182/blood-2011-07-338822. Epub 2011 Sep 14.'}, {'pmid': '22053277', 'type': 'BACKGROUND', 'citation': 'Munchel A, Kesserwan C, Symons HJ, Luznik L, Kasamon YL, Jones RJ, Fuchs EJ. Nonmyeloablative, HLA-haploidentical bone marrow transplantation with high dose, post-transplantation cyclophosphamide. Pediatr Rep. 2011 Jun 22;3 Suppl 2(Suppl 2):e15. doi: 10.4081/pr.2011.s2.e15.'}]}, 'descriptionModule': {'briefSummary': 'Background:\n\n\\- Chronic Granulomatous Disease (CGD) causes immune system problems. Treatment is usually a bone marrow transplant from a fully matched donor. Researchers want to try using partially matched donors for patients who do not have a fully matched donor available. The researchers will also use the drug cyclophosphamide to try to improve the outcomes when using a partially matched donor.\n\nObjective:\n\n\\- To learn the effectiveness of using cyclophosphamide with a transplant from a partially matched donor in treating CGD.\n\nEligibility:\n\n\\- Recipients: age 2-65 with CGD with an ongoing infection that has not been cured by standard treatment and no fully matched donor available in an appropriate timeframe.\n\nDesign:\n\n* Recipients will:\n\n * be admitted to the hospital 2 weeks before transplant.\n * be screened with blood and urine tests, breathing and heart health tests, X-rays, and/or magnetic resonance imaging. They may have a bone marrow aspiration and biopsy.\n* meet with a social worker and dentist.\n* get chemotherapy, radiation, and other medicines.\n* get an intravenous (IV) catheter in their chest.\n* have the transplant.\n* get more medicines and standard supportive care.\n* have blood drawn frequently.\n* have to stay in the Washington, D.C. area for 3 months post-transplant.\n* be followed closely for the first 6 months, and then less frequently for at least 5 years.', 'detailedDescription': 'Allogeneic transplant using HLA matched donors, both related and unrelated, has proven curative for patients with various immunodeficiencies, including those with ongoing infections. However donor availability remains a limiting factor in the application of this treatment modality. The use of haploidentical donors has in the past been fraught with a greater rate of complications related to both higher rates of GvHD and delayed immunorecovery. Newer transplant regimens appear to have diminished these risks and improved outcomes. We propose using a subablative conditioning regimen followed by post-transplant cyclophosphamide for patients with CGD who do not have an HLA matched donor but whose circumstances necessitate the use of a potentially curative, albeit high-risk treatment modality.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': '* INCLUSION CRITERIA:\n* Must have sufficient complications from underlying disease to warrant undergoing transplantation\n* Ages 2 years - 65 years\n* No appropriate HLA matched donor (available donor has greater than 1 mismatch or the single mismatch is not at DQ for unrelated donors (including cord blood products), or no available 6 out of 6 HLA matched related donor), or patients who may have an unrelated donor, but whose clinical status is such that the time required to obtain an unrelated donor would be life threatening.\n* HLA haploidentical family donor graft available.\n* Ability to comprehend and willingness to sign the informed consent or have a parent/guardian consent if the donor is a minor; assent being obtained from minors as appropriate\n* Must be HIV negative\n* Must not be pregnant (confirmed by a negative serum beta-human chorionic gonadotropin (Beta-hCG) for women of child-bearing potential) or breastfeeding\n* Must be able to stay within one hour s travel of the NIH for the first 3 months after transplantation and have a family member or other designated companion to stay with during the post-transplant period.\n* Must provide a durable power of attorney for health care decisions to an appropriate adult relative or guardian in accordance to NIH Form-200 NIH Durable Power of Attorney for Health Care Decision Making.\n* Where appropriate, subjects must agree to use contraception for 3 months post-transplant\n\nEXCLUSION CRITERIA:\n\n* Major anticipated illness or organ failure incompatible with survival from Allo-transplant\n* Inadequate collection from prospective donors.'}, 'identificationModule': {'nctId': 'NCT02282904', 'briefTitle': 'Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide', 'organization': {'class': 'NIH', 'fullName': 'National Institutes of Health Clinical Center (CC)'}, 'officialTitle': 'Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide', 'orgStudyIdInfo': {'id': '150007'}, 'secondaryIdInfos': [{'id': '15-I-0007', 'type': 'OTHER', 'domain': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'CGD Recipient', 'description': 'CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described', 'interventionNames': ['Drug: Sirolimus', 'Biological: Donor peripheral blood stem cells.', 'Drug: Cyclophosphamide post transplant', 'Radiation: Total body 200cGy', 'Drug: Cyclophosphamide', 'Drug: Fludarabine', 'Drug: Busulfan']}], 'interventions': [{'name': 'Sirolimus', 'type': 'DRUG', 'otherNames': ['Rapamycin'], 'description': 'For pediatric patients: Begin sirolimus 1 mg/m2 PO q4h for 3 doses, then 1 mg/m2 once a day (QD). For adult patients, begin sirolimus 5 mg PO q4h for 3 doses, then 5 mg once a day (QD).\n\nDoses may be adjusted to maintain trough levels between 8-14 ng/ml. Recipients will take sirolimus from Day +5 to at least Day 100 (minimum).', 'armGroupLabels': ['CGD Recipient']}, {'name': 'Donor peripheral blood stem cells.', 'type': 'BIOLOGICAL', 'description': 'Infuse donor graft.', 'armGroupLabels': ['CGD Recipient']}, {'name': 'Cyclophosphamide post transplant', 'type': 'DRUG', 'otherNames': ['Cytoxan post transplant'], 'description': '50 mg/kg/d IV infused over 90 minutes. Day +3 and +4', 'armGroupLabels': ['CGD Recipient']}, {'name': 'Total body 200cGy', 'type': 'RADIATION', 'description': 'Day -1', 'armGroupLabels': ['CGD Recipient']}, {'name': 'Cyclophosphamide', 'type': 'DRUG', 'otherNames': ['Cytoxan'], 'description': '14.5 mg/kg IV over one hour Day -6 and -5', 'armGroupLabels': ['CGD Recipient']}, {'name': 'Fludarabine', 'type': 'DRUG', 'otherNames': ['Fludara'], 'description': '30 mg/m2 over 30 minutes Day -6 through Day -2', 'armGroupLabels': ['CGD Recipient']}, {'name': 'Busulfan', 'type': 'DRUG', 'otherNames': ['Busulfex'], 'description': 'Busulfan 3.2 mg/kg IV once daily over 2-3 hours Day -4,-3,-2', 'armGroupLabels': ['CGD Recipient']}]}, 'contactsLocationsModule': {'locations': [{'zip': '20892', 'city': 'Bethesda', 'state': 'Maryland', 'country': 'United States', 'facility': 'National Institutes of Health Clinical Center, 9000 Rockville Pike', 'geoPoint': {'lat': 38.98067, 'lon': -77.10026}}], 'overallOfficials': [{'name': 'Elizabeth M Kang, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'National Institute of Allergy and Infectious Diseases (NIAID)'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'National Institute of Allergy and Infectious Diseases (NIAID)', 'class': 'NIH'}, 'responsibleParty': {'type': 'SPONSOR'}}}}