[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"primary-immune-regulatory-disorder\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:primary-immune-regulatory-disorder":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,46,99],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":23,"conditions":24,"keywords":28,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":40,"leadSponsor":42,"locationsCount":45},"100499770","phase-2-a-study-of-treatment-of-inflammation-before-stem-cell-transplant-in-people-with-a-primary-immune-regulatory-disorder-pird-andor-an-autoinflammatory-condition-100499770",false,"NCT05787574","A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and\u002For an Autoinflammatory Condition","Phase 2 Study Bridging Pre-Transplant Inflammatory Dampening for Primary Immune Regulatory Disorders (BRIDGE Trial)","Inclusion criteria:\n\n1. Patients receiving first allo-HCT for the following immunologic conditions:\n\n   * Primary Immune Regulatory Disorder with or without a genetic lesion as defined by the Primary Immune Deficiency Treatment Consortium (PIDTC)12 - Patients with autoinflammatory disorders evidenced by cytokine or inflammation assays with at least 1.5x ULN of measured cytokines (defined in section 4) and\u002For an elevated ferritin or ESR \\> 2 ULN\n   * For inclusion on the emapalumab group, the lesion must be related to the IFNγ pathway (or mediators thereof) with an elevated CXCL9 \\>1.5 ULN OR sIL2R \\>1.5 ULN (or already controlled on immune modulation, provided that CXCL9 or sIL2R levels were elevated prior to initiation of immune modulation).\n   * Inclusion on the Fludarabine\u002Fdexamethasone group requires inflammation (as defined above) other than an IFNy pathway defect\n2. Able to tolerate cytoreduction (based on adequate organ function as described below)\n3. Patients of any age can enroll so long as they meet all other eligibility criteria\n4. Adequate organ function is required, defined as follows:\n\n   * Hepatic: Serum bilirubin ≤ 2 mg\u002FdL, unless benign congenital hyperbilirubinemia. Patients with hyperbilirubinemia related to paroxysmal nocturnal hemoglobinuria or other hemolytic disorders related to their PIRD diagnosis are eligible.\n   * Hepatic: AST, ALT, and alkaline phosphatase \\\u003C 2.5 times the upper limit of normal unless thought to be disease-related. Investigator will need to perform clinically indicated evaluations to assess if disease related or intrinsic liver disease. Additional testing may be done if clinically indicated, after the pre-transplant immune prophase and prior to start of conditioning as this will provide additional data to confirm disease related versus intrinsic liver dysfunction.\n   * Renal: serum creatinine \\\u003C1.5x normal for age. If serum creatinine is outside the normal range, then CrCl \\> 50 mL\u002Fmin\u002F1.73m\\^2 (calculated or estimated) or GFR (mL\u002Fmin\u002F1.72m\\^2) \\>30% of predicted normal for age.\n   * Cardiac: LVEF ≥ 50% by MUGA or resting echocardiogram.\n   * Pulmonary: Pulmonary function testing (FEV1 and corrected DLCO) ≥ 50% predicted (pediatric patients unable to complete PFTs will need oxygen saturation as recorded by pulse oximetry of ≥92% on room air).\n5. Adequate performance status:\n\n   * Age ≥ 16 years: ECOG ≤ 1 or Karnofsky ≥ 70%\n   * Age \\\u003C 16 years: Lansky 70%\n6. Each patient must be willing to participate as a research subject and must sign an informed consent form or legal guardian with assent as appropriate.\n\nDonor Inclusion Criteria:\n\n1. Related Donors:\n\n   * 8\u002F8 or 7\u002F8 HLA matched at A, B, C, and DRB1 loci, as tested by DNA analysis.\n   * Haploidentical donors at A, B, C and DRB1 loci, as tested by DNA analysis\n2. Unrelated Donors:\n\n   * 8\u002F8 or 7\u002F8 matched at A, B, C, and DRB1 loci, as tested by DNA analysis.\n3. Able to provide informed consent for the donation process per institutional standards.\n4. Meet standard criteria for donor collection (e.g. National Marrow Donor Program Guidelines or collecting center guidelines as approved by treating physician).\n\nExclusion Criteria:\n\n1. Uncontrolled infection at the time of enrollment.\n2. Patients who have undergone previous allo-HCT.\n3. Patient seropositivity for HIV I\u002FII and\u002For HTLV I\u002FII.\n4. Females who are pregnant or breastfeeding.\n5. Patients unwilling to use contraception during the study period.\n6. Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.","ALL",{"count":18,"type":19},39,"ESTIMATED","INTERVENTIONAL",[22],"PHASE2","The researchers are doing this study to find out whether emapalumab or a combination of fludarabine and dexamethasone are effective in preparing people with a primary immune regulatory disorder (PIRD) and\u002For an autoinflammatory condition to receive a stem cell transplant. The researchers will look at how well the study treatments reduce inflammation and aid in the engraftment process (the process of donated stem cells traveling to the bone marrow, where they begin to make new immune cells.\n\n\"Funding Source - FDA OOPD\"",[25,26,27],"Primary Immune Regulatory Disorder","Autoimmune Lymphoproliferative","Immune System Diseases",[29,30,31,32],"Stem Cell Transplant","Emapalumab","Fludarabine","Dexamethasone","RECRUITING","2026-05-29",{"date":36,"type":37},"2026-06-02","ACTUAL",{"date":39,"type":37},"2023-03-15",{"date":41,"type":19},"2027-03",{"name":43,"class":44},"Joseph Oved","OTHER",13,{"id":47,"slug":48,"hasResults":11,"nctId":49,"briefTitle":50,"officialTitle":50,"acronym":51,"eligibilityCriteria":52,"healthyVolunteers":11,"sex":16,"minAge":53,"maxAge":54,"enrollmentInfo":55,"targetDuration":4,"studyType":20,"phases":57,"briefSummary":58,"conditions":59,"keywords":74,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":89,"lastUpdatePostDateStruct":90,"startDateStruct":92,"completionDateStruct":94,"leadSponsor":96,"locationsCount":98},"100614826","phase-2-hematopoietic-stem-cell-transplantation-hsct-for-common-variable-immunodeficiency-cvid-and-other-autoimmune-manifestations-of-primary-immune-regulatory-disorders-pird-100614826","NCT07284641","Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)","CVID\u002FPIRD","Inclusion Criteria:\n\n1. Patient, parent, or legal guardian must have given written informed consent. For pediatric subjects who are developmentally able, assent or affirmation will be obtained.\n2. Male or female, 5 through 40 years old, inclusive, at the time of informed consent.\n3. Patients must have evidence of common variable immunodeficiency (CVID) or other autoimmune manifestation of a primary immune regulatory disorder (PIRD). Genetic screening is required by a targeting gene panel to determine presence of genetic variations that may lead to inborn errors of immunity.\n\n   Examples of such diseases include, but are not limited to:\n   * Common variable immunodeficiency (CVID)\n   * Combined Immunodeficiency (CID)\n   * Immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX syndrome), IPEX like syndromes\n   * Combined immunodeficiency with defects in T-cell-mediated immunity, including Omenn syndrome and DiGeorge Syndrome\n   * Chronic Granulomatous Disease (CGD)\n   * Signal Transducer and Activator of Transcription (STAT 1) Gain of Function (STAT1 GOF)\n   * Signal Transducer and Activator of Transcription (STAT 3) Gain of Function (STAT3 GOF)\n   * Hypomorphic Recombination-Activating Genes (RAG) 1 and RAG 2\n   * CD40 or CD40L deficiency\n   * Mendelian Susceptibility to Mycobacterial Disease\n   * GATA-binding factor 2 (GATA2) Associated Immunodeficiency\n   * Mouth and Genital Ulcers with Inflamed Cartilage Syndrome (MAGIC)\n4. Must have previously failed, due to lack of response or intolerance, mycophenolate mofetil and a B cell-depleting antibody, such as Rituximab\n5. Glomerular Filtration Rate (GFR) ≥50 mL\u002Fmin\u002F1.73 m2\n6. Aspartate Aminotransferase (AST) ≤4x upper limit of normal\n7. Alanine Aminotransferase (ALT) ≤4x upper limit of normal\n8. Direct bilirubin ≤ 2.5 mg\u002FdL\n9. Human Immunodeficiency Virus (HIV) negative by serology and PCR\n10. Human T-cell Lymphotropic Virus (HTLV) negative by serology\n11. Cardiac ejection fraction ≥ 40% or shortening fraction ≥26%\n12. Forced Vital Capacity (FVC) and Forced Expiratory Volume in 1 second (FEV1) ≥40% predicted for age\n13. Peripheral Capillary Oxygen Saturation (SpO2) of \\>92% at rest on room air\n14. Subjects must be a minimum of 8 weeks post-solid organ transplant prior to start of conditioning, if applicable\n15. Negative pregnancy test for females \\>10 years old or who have reached menarche, unless surgically sterilized.\n16. All females of childbearing potential and sexually active males must agree to use a FDA approved method of birth control for up to 12 months after stem cell transplant or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause birth defects.\n17. Subject and\u002For parent guardian informed of the potential risks of infertility following stem cell transplant and advised to discuss sperm banking or oocyte harvesting.\n18. Transplant endorsement from clinical immunologist\n\nExclusion Criteria:\n\n1. Allergy to Dimethylsulfoxide (DMSO) or any other ingredient used in the manufacturing of the stem cell product\n2. Uncontrolled systemic infection, as determined by the appropriate confirmatory testing e.g. blood cultures, Polymerase chain reaction (PCR) testing, etc.\n3. Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of stem cell transplant\n4. Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the subject's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.","5 Years","40 Years",{"count":56,"type":19},25,[22],"This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradiation (TBI) in those diagnosed with Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD).",[60,25,61,62,63,64,65,66,67,68,69,70,71,72,73],"Common Variable Immunodeficiency (CVID)","Immune Dysregulation","DiGeorge Syndrome","STAT 1 Gain of Function","STAT 3 Gain of Function","Hypomorphic RAG1 Deficiency","CD40 Ligand Deficiency","Mendelian Susceptibility to Mycobacterial Disease","GATA2 Associated Immunodeficiency","CD40 Deficiency","Hypomorphic RAG2 Deficiency","Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome","Omenn Syndrome","Chronic Granulomatous Disease",[75,76,77,78,79,80,81,82,83,84,62,85,73,63,64,86,87,67,68,88],"HSCT","CVID","PIRD","primary immune regulatory disorder","common variable immunodeficiency","IPEX like syndromes","Immune dysregulation","polyendocrinopathy","enteropathy","X-linked (IPEX syndrome)","Combined Immunodeficiency","Hypomorphic RAG 1 and RAG 2","CD40 or CD40L deficiency","Inflamed Cartilage Syndrome (MAGIC)","2026-05-13",{"date":91,"type":37},"2026-05-15",{"date":93,"type":37},"2026-05-04",{"date":95,"type":19},"2031-02-01",{"name":97,"class":44},"Paul Szabolcs",1,{"id":100,"slug":101,"hasResults":11,"nctId":102,"briefTitle":103,"officialTitle":104,"acronym":4,"eligibilityCriteria":105,"healthyVolunteers":11,"sex":16,"minAge":106,"maxAge":107,"enrollmentInfo":108,"targetDuration":4,"studyType":20,"phases":110,"briefSummary":112,"conditions":113,"keywords":127,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":133,"lastUpdatePostDateStruct":134,"startDateStruct":136,"completionDateStruct":138,"leadSponsor":140,"locationsCount":98},"100580604","phase-1-phase-12-cd45ra-depleted-stem-cell-addback-to-prevent-viral-or-fungal-infections-post-tcrabcd19-depleted-hsct-100580604","NCT06839456","Phase 1\u002F2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab\u002FCD19 Depleted HSCT","Phase 1\u002F2 Study: CD45RA Depleted Peripheral Stem Cell Addback to Prevent Viral and Fungal Infections Following Alternative Donor TCRab\u002FCD19 Depleted Hematopoietic Stem Cell Transplant","Inclusion Criteria:\n\n1. Disease for which allogeneic HSCT may be curative.\n2. Remission status of hematologic malignancies and additional disease-specific eligibility determinations will be according to standards of practice within the CHOP Cellular Immunotherapy and Transplant Program (CTTS).\n3. Patients must be 25 years of age and less\n4. Evaluation for organ and infectious status as per our CTTS standard operating procedure.\n5. Signed consent by parent\u002Fguardian or able to give consent if 18 years of age and older.\n6. Participants of childbearing potential must have a negative pregnancy test as per institutional SOP.\n\nExclusion Criteria:\n\n1. Patients who have performance score less than 60.\n2. No suitable donor available for mobilized peripheral stem cells.\n3. Patients with Hodgkin lymphoma or non-Burkitt, non-lymphoblastic lymphoma.\n4. Planned receipt of alemtuzumab during conditioning.\n5. Patients with an available 10\u002F10 HLA matched sibling donor.\n6. Patients who do not meet institutional disease, organ or infectious criteria.\n\nDonor selection and eligibility:\n\n1. Unrelated donor meets National Marrow Donor Program criteria for donation.\n2. Related donor (at least haploidentical) willing and able to donate mobilized peripheral stem cells.\n3. HLA testing\u002Fmatching\n\n   * HLA testing to be done by molecular methods for A, B, C, DRB1, DQB1\n   * Related donor: Must be ≥ 5\u002F10 match\n   * Unrelated donor: 10\u002F10 or 9\u002F10 match\n   * KIR typing for haploidentical donor for hematologic malignancies\n   * Donor specific HLA antibodies (DSA) should be assessed for all subjects receiving an HLA mismatched graft (≤ 9\u002F10).\n4. Donor must be willing to undergo granulocyte colony stimulating factor (GCSF) mobilization and peripheral blood stem cell collection\n5. Donors must be willing to sign consent to participate in this study.","1 Month","25 Years",{"count":109,"type":19},100,[111,22],"PHASE1","The major morbidities of allogeneic hematopoietic stem cell transplant (HSCT) using donors that are not human leukocyte antigen (HLA) matched siblings are graft vs host disease (GVHD) and life- threatening infections. T cell receptor alpha beta (TCRαβ) T lymphocyte depletion and CD19+ B lymphocyte depletion of alternative donor hematopoietic stem cell (HSC) grafts is effective in preventing GVHD, but immune reconstitution may be delayed, increasing the risk of infections. The central hypothesis of this study is that an addback of CD45RO memory T lymphocytes, derived from a fraction of the original donor peripheral stem cell product depleted of CD45RA naïve T lymphocytes, will accelerate immune reconstitution and help decrease the risk of infections in TCRab\u002FCD19 depleted PSCT.",[114,115,116,117,118,119,120,121,122,25,123,124,125,126],"Leukemia","High Risk Acute Lymphoblastic Leukemia","High Risk Acute Myeloid Leukemia","Relapse Leukemia","MDS (Myelodysplastic Syndrome)","Relapsed Non-Hodgkin Lymphoma","Acquired Aplastic Anemia","Inherited BMF Syndrome","Immunodeficiency","Hemoglobinopathies","Bone Marrow Failure","Inborn Errors of Metabolism","HLH",[128,129,130,131,132],"alpha beta T cell depletion","CD45RA","CD45RO","GVHD prevention","Memory T cells","2026-04-13",{"date":135,"type":37},"2026-04-15",{"date":137,"type":37},"2025-03-21",{"date":139,"type":19},"2032-03",{"name":141,"class":44},"Children's Hospital of Philadelphia"]