[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"immune-deficiencies\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:immune-deficiencies":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,47],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":26,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":4,"leadSponsor":43,"locationsCount":46},"100088025","detection-and-characterization-of-infections-and-infection-susceptibility-100088025",false,"NCT00404560","Detection and Characterization of Infections and Infection Susceptibility","Screening Protocol for Detection and Characterization of Infections and Infection Susceptibility","* INCLUSION CRITERIA:\n\nPATIENTS:\n\n* Patients known to have, or suspected of having an infection susceptibility and their healthy blood relatives will be eligible for enrollment.\n* Participants must be over 1 month of age. There will be no limit as to sex, race or disability.\n* Patients must have a primary physician outside of the NIH and may be required to submit a letter from their physician that documents their relevant health history.\n* The participant or the participant's guardian\u002FLAR will be willing and capable of providing informed consent after initial counseling by clinical staff. Separate consent forms for all interventional procedures will be obtained after explanation of the specific procedure.\n* Patients and relatives must agree to have blood and tissue stored for future studies of the immune system and\u002For other medical conditions.\n* Patients and relatives may be concurrently enrolled on other protocols as long as the Principal Investigator is informed.\n* The patient must be enrolled on this protocol to have relatives enrolled.\n\nThe patient and patient relative cohorts will include the following special populations:\n\n* Children: Children are included in this study because immune defects may present in early childhood, and early diagnosis or characterization may benefit subjects. Children who do not meet the age and weight criteria for care at the Clinical Center, may have sample collection only.\n* Decisionally impaired adults: Patients and patient relatives will be able to provide informed consent for themselves or, if they lack the capacity to provide informed consent, the study team will obtain consent from the legally authorized representative. Patients with underlying immune disorders, autoimmune phenomena or severe infections may sometimes present with delirium, encephalopathy, or coma and are therefore unable to provide informed consent. Excluding patients who are unable to provide consent could adversely impact patient access to medical therapy at the NIH as well as adversely impact research recruitment. Excluding patients unable to provide consent would also essentially prohibit us from evaluating patients at higher risk for adverse outcomes and therefore skew our understanding of disease. Similarly, enrolled patient subjects who lose the ability to provide ongoing consent during study participation may continue in the study. The risks and benefits of participation for subjects unable to consent should be identical to those described for less vulnerable patients.\n\nEXCLUSION CRITERIA:\n\n1. A well understood acquired abnormality which leads to infection susceptibility, such as HIV, cytotoxic chemotherapy, or active malignancy may be adequate explanation for the infection diathesis. These may be grounds for exclusion if, in the opinion of the investigators, the presence of such disease process interferes significantly with evaluation (applicable to patients and their blood relatives).\n2. Severe or uncommon infections or syndromes often require highly specialized teams and institutions. Some referred cases will not be able to be handled appropriately at the NIH and may be deemed ineligible, as determined by the Principal Investigator.","ALL","1 Month","100 Years",{"count":20,"type":21},2000,"ESTIMATED","OBSERVATIONAL","This screening study will examine the causes of immune disorders affecting white blood cells, which defend against infections and will try to develop better means of diagnosis and treatment of these immune disorders. This is a 2 visit screening study and patients determined to be of interest for additional study or treatment will be asked to provide consent for enrollment into an appropriate NIH follow up study. This study does not cover the cost of the first visit to NIH for travel or lodgings but does cover the subsequent visit if there is one. A financial assessment may determine if the patient is eligible for financial assistance. This study does not enroll children under the age of 2.\n\nPatients known to have or suspected of having increased susceptibility to infections and their blood relatives may be eligible for this study, at the discretion of the principal investigator. Patients and family members may undergo the following procedures:\n\n* Personal and family medical history.\n* Physical examination and blood and urine tests.\n* Studies of breathing function (pulmonary function testing)\n* Dental examination.\n* Eye examination.\n* Genetic Testing\n* Stored specimens for future analysis\n* Microscopic examination of saliva, wound drainage or tissues removed for medical reasons for cell, hormone or DNA studies.\n\nIn addition, patients will be asked to obtain permission for investigators to obtain their medical records, previous test results, or radiographic studies prior to the first visit. Patients will be asked to undergo imaging studies, such as a chest X-ray, CT scan or MRI scan.\n\n...",[25],"Immune Deficiencies",[27,28,29,30,31,32,33,34,35],"Primary Immune Deficiency","Autoimmune","Genetic Defects","Immune Defects","Infection Susceptibility","Recurrent Infections","Immune Disorders","Immune Defects of Phagocytes","Unusual, Chronic Bacterial, Mycobacterial, and Fungal Infections","RECRUITING","2026-06-13",{"date":39,"type":40},"2026-06-16","ACTUAL",{"date":42,"type":40},"2007-01-02",{"name":44,"class":45},"National Institute of Allergy and Infectious Diseases (NIAID)","NIH",1,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":54,"enrollmentInfo":4,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":56,"conditions":57,"keywords":60,"overallStatus":85,"whyStopped":4,"lastUpdateSubmitDate":86,"lastUpdatePostDateStruct":87,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":89,"locationsCount":46},"100200115","expanded-access-to-t-cell-depleted-haplo-identical-stem-cells-for-patients-receiving-haplo-identical-and-unrelated-cord-blood-transplants-100200115","NCT01881334","Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants","A Compassionate Release Protocol: Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Allogeneic Transplantation Using a Related Haplo-Identical Donor and Unrelated, Umbilical Cord Blood Donor(s) for the Treatment of High Risk Malignancies or Non-Malignant Disorders Requiring Allogeneic Transplantation","Inclusion Criteria:\n\n* Have a consenting related haplo-identical (3\u002F6, 4\u002F6, or 5\u002F6 if DRB1 mismatch) stem cell donor.\n* Have one or two available 4, 5, or 6\u002F6 antigen matching unrelated UCB unit(s) that will deliver a total cell dose \\>3.0 x 10e7 cells\u002Fkg. Patients who do not have a single UCB unit that will deliver the minimum required cell dose, two partially HLA-matched UCB units which together meet the minimum cell dose requirement, can be used for 1 transplant. These units must be HLA-matched minimally at 4 of 6 HLA-A and B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing) loci with the patient, and HLA-matched at 3 of 6 HLA- A, B, DRB1 loci with each other (using same resolution of HLA typing as indicated above). There is no limitation on maximum cell dose.\n* Have a high risk or refractory malignancy, or non-malignant disorder amenable to stem cell transplantation therapy.\n* Meet eligibility requirements for allogeneic transplant per institutional standard practices.\n* Have given written informed consent according to FDA guidelines (or consent of parent\u002Flegal guardian as applicable).\n* Be \\\u003C65 years of age at the time of study enrollment.\n\nExclusion Criteria:\n\n* Have a consenting 8\u002F8 or 10\u002F10 allele matched, consenting, related or unrelated hematopoietic stem cell transplant (HSCT) donor.\n* Have a life expectancy of less than 3 months.\n* Have uncontrolled infections at time of cytoreduction.","65 Years","EXPANDED_ACCESS","The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants who are at a higher risk of not engrafting in a safe amount of time. The purpose of the related stem cells is to give the bone marrow a \"jump start\" towards recovery. Ultimately, the cord blood cells will grow and permanently rescue the bone marrow.",[58,59,25],"Hematologic Malignancies","Inborn Errors of Metabolism Disorders",[61,62,63,64,65,66,67,68,69,70,16,71,72,73,74,75,76,77,78,79,80,81,82,83,84],"Haploidentical Donor","T-cell depleted Stem Cells","Allogeneic Transplant","Umbilical Cord Blood Donor","High Risk Malignancies","Metabolic Disorders","Immune Deficiency","Acute Lymphoblastic Leukemia","Acute Myelogenous Leukemia","Myelodysplastic Syndrome","AML","MDS","CGD","SCID","Adrenoleukodystrophy","Metachromaticleukodystrophy","Krabbe","PMD","Hunter's","Hurler's","Severe Aplastic Anemia","Lymphoma","Sickle Cell Disease","Thalassemia","AVAILABLE","2025-11-17",{"date":88,"type":40},"2025-11-20",{"name":90,"class":91},"Joanne Kurtzberg, MD","OTHER"]