[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"immunodeficiency\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:immunodeficiency":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,9,0,[8,48,77,103,129,172,198,225,256],{"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":23,"briefSummary":25,"conditions":26,"keywords":30,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":47},"100198569","phase-2-allogeneic-hematopoietic-stem-cell-transplant-for-gata2-mutations-100198569",false,"NCT01861106","Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations","Allogeneic Hematopoietic Stem Cell Transplant for Patients With Mutations in GATA2 or the MonoMAC Syndrome","* ELIGIBILITY CRITERIA:\n\nINCLUSION CRITERIA- Recipient\n\n1. Patient age of 6-70 years.\n2. Mutation in the GATA2 gene, or evidence of loss of expression of one allele of GATA2, by cDNA analysis performed by a CLIA certified laboratory, or the clinical syndrome of MonoMAC.\n3. Clinical history of at least one serious or disfiguring infection and\u002For GATA2 bone marrow immunodeficiency disorder with lose of one or more immune populations in the bone marrow including monocytes, Natural Killer (NK) cells, and B-lymphocytes, with or without additional cytopenias involving the red blood cell, neutrophil, or platelet compartment.\n4. Availability of a 10\u002F10 or 9\u002F10 or 8\u002F10 HLA-matched related or unrelated donor, or a haploidentical related donor.\n5. Patients may have evidence of MDS with one or more peripheral blood cytopenias and greater than 5% blasts but must have less than 10% blasts in the bone marrow in the absence of filgrastim in order to proceed directly to transplant. The majority of patients with MDS will have less than 5% blasts.\n6. Disease status: Patients are to be referred in remission for evaluation. Should a patient have progressive disease with \\>10% blasts on screening\u002Fbaseline bone marrow biopsy, the patient may receive standard treatment under the current study prior to proceeding with transplant. Once the patient has \\\u003C10% blasts, they may proceed to transplant. The patient may also be referred back to their primary hematologist or oncologist for treatment. If this course of action is not in the best interest of the patient according to the clinical judgment of the PI\u002FLAI, then the patient may receive standard treatment for the malignant disease or hematological disorder under the current study. If under either of these settings, it becomes apparent that the participant will not be able to proceed to transplant, then he\u002Fshe must come off study. Recipient-Subjects receiving a standard therapy will be told about the therapy, associated risks, benefits and alternatives of the proposed therapy, and availability of receiving the same treatment elsewhere, outside of a research protocol.\n7. Left ventricular ejection fraction \\> 40%, preferably by 2-D echocardiogram obtained within 90 days prior to initiation of conditioning therapy.\n8. Creatinine: Adult patients: \\\u003C= 2.0 mg\u002Fdl and creatinine clearance \\>= 30 ml\u002Fmin; Pediatric patients (\\\u003C18 years old): creatinine \\\u003C1.5 mg\u002FdL and a creatinine clearance, using the Schwartz Formula, \\> 30 mL\u002Fmin\u002F1.73m\\^2.\n9. Serum conjugated bilirubin \\\u003C 2.5 mg\u002Fdl; serum ALT and AST \\\u003C= 5 times upper limit of normal.\n10. Pulmonary function tests: FEV1 and DLCO \\>30% Note: For children who are unable to cooperate for PFTs, the criterion is: No evidence of dyspnea at rest, no exercise intolerance, and no requirement for supplemental oxygen therapy\n11. Ability of patient or Legally Authorized Representative (LAR) (if the patient is deemed by the treating physician to be cognitively impaired or questionably impaired in such a way that the ability of the patient to give informed consent is questionable) to understand and the willingness to sign a written informed consent document indicating that they are aware of the investigational nature of this study or written informed consent obtained from parent or legal guardian if subject is a minor.\n12. As therapeutic agents used in this trial may be harmful to a fetus, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for at least one-year post-allo HSCT. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in the study, she should inform her treating physician immediately.\n13. All transplant patients remain in the NIH hospital or, if discharged, stay close to the NIH for a minimum of 100 days after transplant or longer, if there are complications. An adult caregiver must be with the patient at all times from discharge to day 100.\n\nEXCLUSION CRITERIA- Recipient\n\n1. Patients who are receiving any other investigational agents with the exception of virus- specific cytotoxic T-cells for the treatment of viral infection\u002Freactivation prior to allo HSCT\n2. HIV-positive patients are ineligible because these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated.\n3. History of allergic reactions attributed to compounds of similar chemical or biological composition to agents (steroids, cyclophosphamide, busulfan) used in the study\n4. Chronic active hepatitis B. Patient may be hepatitis B core antibody positive. For patients with a concomitant positive hepatitis B surface antigen, patients will require a hepatology consultation. The risk-benefit profile of transplant and hepatitis B will be discussed with the patient, and eligibility determined by the PI or Lead Associate Investigator.\n5. History of psychiatric disorder which may compromise compliance with transplant protocol, or which does not allow for appropriate informed consent.\n6. Active infection refractory to antimicrobial therapy.\n7. Active CNS involvement by malignancy (patients with known positive CSF cytology or parenchymal lesions visible by prior CT or MRI).\n8. Pregnant or lactating.\n9. The effects on breast-milk are unknown and may be harmful to the infant; therefore, women should not breast feed during the interval from study entry to one year post-transplant.\n10. Presence of active malignancy in another organ system other than the hematopoietic, except when driven by viruses in which case the immune reconstitution after transplant may control the malignancy. This includes solid tumors not in remission.\n11. No available 10\u002F10 or 9\u002F10 or 8\u002F10 HLA-matched related or unrelated donor, or haploidentical related donor.","ALL","6 Years","70 Years",{"count":20,"type":21},144,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","Background:\n\n\\- GATA2 deficiency is a disease caused by mutations in the GATA2 gene. It can cause different types of leukemia and other diseases. Researchers want to see if a stem cell transplant can be used to treat this condition. A stem cell transplant will give stem cells from a matching donor (related or unrelated) to a recipient. It will allow the donor stem cells to produce healthy bone marrow and blood cells that will attack the recipient s cancer cells.\n\nObjectives:\n\n\\- To see if stem cell transplants are successful at treating GATA2 mutations and related conditions.\n\nEligibility:\n\n\\- Recipients who are between 6 and 70 years of age and have GATA2 deficiency.\n\nDesign:\n\n* All participants will be screened with a physical exam and medical history. Blood samples will be collected. Recipients will have imaging studies and other tests.\n* Recipients will have chemotherapy or radiation to prepare for the transplant. On the day of the transplant, they will receive the donated stem cells.\n* Recipients will stay in the hospital until their condition is stable after transplant.\n* Frequent blood tests and scans will be required for the first 6 months after the transplant, followed by less frequent visits over time.",[27,28,29],"GATA2","Immunodeficiency","MDS",[31,32,28,33,34],"Allogeneic Donors","Peripheral Blood Stem Cell","Myelodysplasia","Haploidentical","RECRUITING","2026-06-23",{"date":38,"type":39},"2026-06-24","ACTUAL",{"date":41,"type":39},"2013-07-24",{"date":43,"type":21},"2028-12-31",{"name":45,"class":46},"National Cancer Institute (NCI)","NIH",1,{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":16,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":58,"phases":4,"briefSummary":59,"conditions":60,"keywords":64,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":71,"startDateStruct":73,"completionDateStruct":4,"leadSponsor":75,"locationsCount":47},"100054498","genetic-analysis-of-immune-disorders-100054498","NCT00001467","Genetic Analysis of Immune Disorders","* INCLUSION \u002F EXCLUSION CRITERIA:\n\nProbands and their blood relatives, of any age, and ethnicity, who are affected, or suspected of being affected with genetic conditions and immune dysregulations under study are eligible to enroll as patients and family member enrollees.","1 Day","101 Years",{"count":57,"type":21},5000,"OBSERVATIONAL","The purposes of this study are to 1) identify the genes responsible for certain immune disorders, 2) learn about the medical problems they cause, and 3) learn how to predict who is likely to develop these disorders and what the risk is of passing them on to children. The immune system is the body s defense system. Some immune deficiencies impair a person s ability to fight infections; others render a person susceptible to allergies, or to autoimmune diseases such as lupus or arthritis, in which the immune cells (white blood cells) attack and destroy the body s own tissues.\n\nPatients with immune disorders known or suspected to have a genetic basis and their family members may enroll in this study. Eligibility will be determined by a review of the patient s medical records and family medical history. Participants will provide a small blood sample for genetic (DNA) and white blood cell analysis. Gene samples (but not white blood cells) may also be obtained by mouth brushing or skin biopsy. For the mouth brushing, a small brush is rubbed against the inside of the cheeks for 1 minute to wipe off some cells. For the skin biopsy, a small circle of skin (about 1\u002F8 inch) is removed under local anesthetic. Pregnant women may be asked to provide a fetal sample (amniotic fluid cells or chorionic villus sample). All samples will be used for immune or genetic studies of the family s immune disorder.\n\nIf test results show a specific genetic variation responsible for the family s immune disorder, a report will be sent to the patient s doctor or genetic counselor, who will discuss the implications for the family. NIH researchers and genetic counselors will also be available to explain results and answer questions. Information will not be available in the case of disorders that cannot yet be linked to a specific genetic abnormality.\n\nInformation from this study will increase knowledge about the immune system and what causes immune deficiencies. Participants may also learn the underlying cause of an immune disorder that affects them or someone in their family information may be useful in guiding treatment and in making decisions regarding family planning.",[61,62,27,28,63],"DOK 8","STAT1","STAT3",[65,66,67,68,69],"Primary Immunodeficiency Disorders","Immunologic Disorders","Mutation","Genetic Analysis","Natural History","2026-06-03",{"date":72,"type":39},"2026-06-04",{"date":74,"type":39},"1995-06-06",{"name":76,"class":46},"National Institute of Allergy and Infectious Diseases (NIAID)",{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":82,"acronym":83,"eligibilityCriteria":84,"healthyVolunteers":85,"sex":16,"minAge":86,"maxAge":4,"enrollmentInfo":87,"targetDuration":4,"studyType":58,"phases":4,"briefSummary":89,"conditions":90,"keywords":91,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":47},"100563157","vaccine-induced-immunity-in-immunocompromised-patients-100563157","NCT06612515","Vaccine-induced Immunity in Immunocompromised Patients","Prospective Cohort-study for the Investigation of the Vac-cine-induced Immune Response After Vaccination Against RESPiratory Viral Infections in Immunocompromised Pa-tients With or Without Haemato-ONcological diseaSEs","RESPONSE","Inclusion Criteria:\n\n* Signed informed consent form\n* Patients with immunosuppression either by treatment or underlying diseases\n* Patients who are vaccinated or willing to be vaccinated against respiratory virus infections in ac-cordance with current recommendations\n* Age of 18 years or older\n\nExclusion Criteria:\n\n* Patients unwilling\u002Fineligible for vaccination under current recommendations",true,"18 Years",{"count":88,"type":21},1000,"Managing respiratory virus infections in immunocompromised patients requires a multidisciplinary approach, including vaccination, though its effectiveness is often suboptimal in these individuals.\n\nIn hematological patients, poor humoral immunogenicity is common, especially when the B cell axis is affected by disease or treatment, while T cell responses may offer better protection. Current immunologic data on these patients is limited, focusing mostly on serologic parameters. To address this, we will conduct an observational study analyzing early and late booster vaccinations, with a focus on virus-specific T cell responses in vaccinated patients.",[28],[92],"vaccine; Immunocompromised; immune response","2026-05-11",{"date":95,"type":39},"2026-05-12",{"date":97,"type":39},"2025-03-25",{"date":99,"type":21},"2029-12-31",{"name":101,"class":102},"University of Cologne","OTHER",{"id":104,"slug":105,"hasResults":11,"nctId":106,"briefTitle":107,"officialTitle":107,"acronym":4,"eligibilityCriteria":108,"healthyVolunteers":11,"sex":16,"minAge":86,"maxAge":109,"enrollmentInfo":110,"targetDuration":4,"studyType":22,"phases":112,"briefSummary":113,"conditions":114,"keywords":116,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":121,"lastUpdatePostDateStruct":122,"startDateStruct":124,"completionDateStruct":126,"leadSponsor":128,"locationsCount":47},"100441406","phase-2-a-phase-ii-study-of-allogeneic-hematopoietic-stem-cell-transplant-for-subjects-with-vexas-vacuoles-e1-enzyme-x-linked-autoinflammatory-somatic-syndrome-100441406","NCT05027945","A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome","* INCLUSION CRITERIA:\n\nNon-disease related\n\n* Age \\>= 18-year-old and \\\u003C= 75-year-old\n* Availability of an 8\u002F8 or 7\u002F8 HLA-matched related or unrelated donor, or a haploidentical related donor\n* Karnofsky performance status of \\>= 40%\n* Adequate end-organ function, defined as follow:\n\n  1. Left ventricular ejection fraction \\> 35%, preferably by 2-D echocardiogram (ECHO) obtained within 60 days prior to treatment initiation.\n  2. Creatinine \\\u003C= 2.0 mg\u002Fdl and creatinine clearance \\>= 30 ml\u002Fmin;\n  3. Serum conjugated bilirubin \\\u003C 3.0 mg\u002Fdl; serum ALT and AST \\\u003C= 5 times upper limit of normal.\n* Pulmonary function tests: FEV1 and DLCO \\>30%\n* Ability of subject to understand and the willingness to sign a written informed consent document.\n* As therapeutic agents used in this trial may be harmful to a fetus, individuals of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) at the study entry and for at least one-year post-allo HCT. Should an individual become pregnant or suspect they are pregnant while she or her partner is participating in the study, she should inform her treating physician immediately.\n* Willingness to remain in the NIH hospital or, if discharged, live within 2 hours drive from the NIH, for a minimum of 100 days after transplant or longer, if there are complications. If outpatient in the first 100 days after transplant, participant must commit to having an adult caregiver with them at all times.\n\nDisease related\n\n* Somatic mutation in UBA1 performed by a CLIA or CAP certified laboratory. NOTE: Participants without a mutation or unknown mutation status may be eligible if they have a clinical history that is characteristic of an individual with VEXAS syndrome including two or more of a-e below.\n* Inflammatory clinical phenotype for VEXAS syndrome with at least one VEXAS disease manifestation below:\n\n  1. constitutional symptoms including fevers, fatigue, and weight loss\n  2. cutaneous symptoms of VEXAS including biopsy proven neutrophilic dermatosis, cutaneous vasculitis, periorbital inflammation\n  3. pulmonary symptoms of VEXAS with pulmonary infiltrates, pleural effusion\n  4. musculoskeletal or cartilaginous involvement including inflammatory arthritis, ear chondritis, and nasal chondritis\n  5. inflammatory disease in other major organ systems including cardiac, gastrointestinal, ocular, etc.\n* Presence of cytopenia defined as at least one of the following:\n\n  i. Absolute neutrophil count \\\u003C=1000\u002F microliter\n\nii. platelet count \\\u003C= 75,000\u002Fmicroliter or platelet transfusion dependence (at least 4 platelet transfusions in the 8 weeks prior to study entry\n\niii. hemoglobin \\\u003C= 10.0g\u002FdL or red cell transfusion-dependence (at least 4 units of PRBCs in the 8 weeks prior to treatment initiation) or meeting criteria for myeloid neoplasm (MN) by updated 2022 WHO criteria or 2022 International Consensus Classification (ICC) of myeloid neoplasms and acute leukemia\n\nOR:\n\n-Participants who have failed standard medical management (requiring \\>= 0.5mg\u002Fkg per day of prednisone for the above listed inflammatory condition or intolerance or refractory to use of corticosteroids and\u002For steroid sparing medications as well as biological response modifiers over the last 6 months), or when no standard medical treatment is available.\n\nEXCLUSION CRITERIA:\n\n* HCT Comorbidity Index \\>= 5. Note: Comorbidities that are specifically addressed in the inclusion criteria will not be included in the calculation of HCT-CI score.\n* Participants with multiple myeloma. Note: participants with low risk smoldering multiple myeloma or monoclonal gammopathy of unknown significance will not be excluded)\n* Participants who are receiving any other investigational agents within the last 30 days before treatment initiation.\n* HIV-positive patients are ineligible because these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy.\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (steroids, cyclophosphamide, busulfan, tacrolimus, MMF, filgrastim or filgrastim biosimilar) used in the study.\n* Pregnant individuals are excluded from this study because the study agents have the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with the study agents, breastfeeding should be discontinued if the mother is treated with the study agents.\n* Uncontrolled intercurrent illness or social situations (as determined by a licensed master social worker) that would limit compliance with study requirements.\n* Presence of active uncontrolled infections that in the opinion of the PI would make it unsafe to proceed with transplantation.\n* Active psychiatric disorder which is deemed by the PI to have significant risk of compromising compliance with the transplant protocol.","75 Years",{"count":111,"type":21},54,[24],"Background:\n\nAllogeneic hematopoietic stem cell transplant involves taking blood stem cells from a donor and giving them to a recipient. The transplants are used to treat certain diseases and cancers. Researchers want to see if the transplant can treat VEXAS Syndrome.\n\nObjective:\n\nTo see if stem cell transplants can be successfully performed in people with VEXAS and even improve the disease.\n\nEligibility:\n\nPeople ages 18-75 who have VEXAS Syndrome that has caused significant health problems and standard treatment either has not worked or is not available.\n\nDesign:\n\nParticipants will be screened with:\n\nPhysical exam\n\nMedical review\n\nBlood and urine tests\n\nHeart and lung function tests\n\nBone marrow biopsy\n\nParticipants will have a chest x-ray. They will have an imaging scan of the head, chest, abdomen, pelvis, and sinus. They will have a bone density scan. They will have a dental exam and eye exam. They will meet with specialists. They will repeat some screening tests.\n\nParticipants will be admitted to the NIH hospital. They have a central venous catheter put into a vein in the chest or neck. They will receive drugs to prepare their bone marrow for the transplant. They may have total body irradiation. They will receive the donor stem cells through the catheter. They will get other drugs to prevent complications and infections. After discharge, they must stay in the DC area for 3 months for weekly study visits.\n\nParticipants will have study visits 30, 60, 100, 180, 210, 240, 300, and 360 days later. After that, they will have yearly visits for 2 years and then be contacted yearly by phone....",[28,115],"Hematopoietic Stem Cell Transplantation",[117,34,118,119,120],"Immune Dysregulation","hematoinflammatory diseases","Myelodysplastic Syndromes","Autoimmune Disorders","2026-05-07",{"date":123,"type":39},"2026-05-08",{"date":125,"type":39},"2023-02-23",{"date":127,"type":21},"2026-07-01",{"name":45,"class":46},{"id":130,"slug":131,"hasResults":11,"nctId":132,"briefTitle":133,"officialTitle":134,"acronym":4,"eligibilityCriteria":135,"healthyVolunteers":11,"sex":16,"minAge":136,"maxAge":137,"enrollmentInfo":138,"targetDuration":4,"studyType":22,"phases":140,"briefSummary":142,"conditions":143,"keywords":157,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":163,"lastUpdatePostDateStruct":164,"startDateStruct":166,"completionDateStruct":168,"leadSponsor":170,"locationsCount":47},"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":139,"type":21},100,[141,24],"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.",[144,145,146,147,148,149,150,151,28,152,153,154,155,156],"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","Primary Immune Regulatory Disorder","Hemoglobinopathies","Bone Marrow Failure","Inborn Errors of Metabolism","HLH",[158,159,160,161,162],"alpha beta T cell depletion","CD45RA","CD45RO","GVHD prevention","Memory T cells","2026-04-13",{"date":165,"type":39},"2026-04-15",{"date":167,"type":39},"2025-03-21",{"date":169,"type":21},"2032-03",{"name":171,"class":102},"Children's Hospital of Philadelphia",{"id":173,"slug":174,"hasResults":11,"nctId":175,"briefTitle":176,"officialTitle":177,"acronym":178,"eligibilityCriteria":179,"healthyVolunteers":11,"sex":16,"minAge":180,"maxAge":4,"enrollmentInfo":181,"targetDuration":4,"studyType":22,"phases":183,"briefSummary":184,"conditions":185,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":187,"lastUpdatePostDateStruct":188,"startDateStruct":190,"completionDateStruct":192,"leadSponsor":194,"locationsCount":197},"100484427","phase-2-optimisation-of-antiviral-therapy-in-immunocompromised-covid-19-patients-a-randomized-factorial-controlled-strategy-trial-100484427","NCT05587894","OPtimisation of Antiviral Therapy in Immunocompromised COVID-19 Patients: a Randomized Factorial Controlled Strategy Trial","OPtimisation of Antiviral Therapy in Immunocompromised COVID-19 Patients: a Randomized Factorial Controlled Strategy Trial: the OPTICOV Study","OPTICOV","Inclusion Criteria:\n\n1. Laboratory confirmed SARS-CoV-2 infection by RT-PCR or positive antigenic test (commercialized assay)\n2. Asymptomatic or mild to moderate COVID-19 (WHO progression scale \\\u003C5. Patients receiving oxygen therapy for reasons other than a pulmonary COVID-19 are eligible).\n3. ≥ 16 years of age (for patients recruited in Italy and in Norway, ≥ 18 years of age);\n4. Immunocompromised as defined by ≥ 1 risk factors for severe COVID-19 as assessed by the FOPH list (criteria 5: diseases\u002Ftreatments leading to immune suppression) or other immunosuppression criteria such as Severe immunosuppression (e.g., HIV infection with CD4 + T cell count \\\u003C350 \u002F µl) Neutropenia (\\\u003C1000 neutrophils \u002F µl) ≥1 week Lymphocytopenia (\\\u003C200 lymphocytes\u002Fµl) On dialysis treatment Hereditary immunodeficiencies Intake of drugs which suppress the immune system (e.g. glucocorticoids for a long time \\[an equivalent dose of prednisone \\>20 mg\u002Fday \\> 3 months\\], monoclonal antibodies, cytostatics, biological products, everolimus, mTOR inhibitors etc.) in the last 12 months Active cancer under cytostatics or targeted therapy known to be immunosuppressive (e.g., platinum salts, cyclophosphamide, anthracyclines, taxanes, 5-fluorouracil, gemcitabine, purine inhibitors, proteasome inhibitors) or associated with hematologic toxicity (neutropenia, lymphopenia), for example sunitinib, imatinib, regorafenib. Aggressive lymphomas (all types) Acute lymphatic leukemia Acute myeloid leukemia Acute promyelocytic leukemia T prolymphocytic leukemia Primary central nervous system lymphoma Stem cell transplantation Light chain amyloidosis Chronic lymphoid leukemia Multiple myeloma Sickle cell disease Bone marrow transplant Organ transplant Being on the waiting list for an organ transplant\n5. Willing and able to comply with study requirements and restrictions as described in the informed consent form (ICF)\n6. Enrolled in or a beneficiary of a Social Security program (State Medical Aid (AME) is not a Social Security program) or holders of health insurance (LAF for participants recruited in Norway).\n7. Participant's or its legal representative's signature of the informed consent form\n\nExclusion Criteria:\n\n1. SARS-CoV-2 PCR ≥30 CT at screening\n2. Hypersensitivity to study drugs (active substance(s) or excipients)\n3. Body weight \\\u003C 40 kg\n4. AST and\u002For ALT \\> 5 times the upper limit\n5. Cirrhosis Child-Pugh score C\n6. Is taking or is anticipated to require any prohibited therapies\\*.\n7. Participation in another interventional clinical study with an investigational compound or device, including COVID-19 therapeutics, where the study intervention is performed in the 28 days preceding the inclusion and the 10 days after the inclusion. Investigators of the different clinical studies should agree on participant's inclusion.\n8. Presence of any condition for which, in the opinion of the investigator, participation would not be in participant's best interest or that could prevent, limit, or confound the protocol-specified assessments\n9. Having received antiviral treatments against SARS-CoV-2 in the 14 days before the inclusion with exception of those having received one or two doses of nirmatrevir\u002Fr in the 24h preceding the inclusion in the study.\n10. Pregnant or breastfeeding female\n\n    * Study SOPs based on recommendations from the Liverpool COVID-19 interactions, French Society for Pharmacology and Therapeutics (https:\u002F\u002Fsfpt-fr.org\u002Frecommandations-et-publications) and French Speaking Transplantation Society will be provided to guide investigators.","16 Years",{"count":182,"type":21},256,[24],"The overall purpose of the trial is to evaluate the efficacy and safety of possible combination antiviral therapy DAA (remdesivir + nirmatrelvir\u002Fr)∞ versus the reference monotherapy (nirmatrelvir\u002Fr alone) and to assess the efficacy and safety of increasing the nirmatrelvir\u002Fr course from 5- to 10 days in immunocompromised patients diagnosed with asymptomatic or mild to moderate COVID-19.",[186,28],"COVID-19","2026-02-19",{"date":189,"type":39},"2026-02-23",{"date":191,"type":39},"2023-04-27",{"date":193,"type":21},"2027-06-30",{"name":195,"class":196},"ANRS, Emerging Infectious Diseases","OTHER_GOV",18,{"id":199,"slug":200,"hasResults":11,"nctId":201,"briefTitle":202,"officialTitle":203,"acronym":204,"eligibilityCriteria":205,"healthyVolunteers":11,"sex":16,"minAge":180,"maxAge":4,"enrollmentInfo":206,"targetDuration":4,"studyType":22,"phases":207,"briefSummary":208,"conditions":209,"keywords":210,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":216,"lastUpdatePostDateStruct":217,"startDateStruct":219,"completionDateStruct":220,"leadSponsor":222,"locationsCount":224},"100624175","phase-2-optimisation-of-antiviral-therapy-in-immunocompromised-covid-19-patients-100624175","NCT07406217","OPtimisation of Antiviral Therapy in Immunocompromised COVID-19 Patients","OPtimisation of Antiviral Therapy in Immunocompromised COVID-19 Patients: a Randomized Factorial Controlled Strategy Trial: the SWISS OPTICOV Study","SWISS OPTICOV","Inclusion Criteria:\n\n1. Laboratory confirmed SARS-CoV-2 infection by real time RT-PCR or positive antigenic test (commercialized assay)\n2. Asymptomatic or mild to moderate COVID-19 (WHO progression scale \\\u003C5. Patients receiving oxygen therapy for reasons other than a pulmonary COVID-19 are eligible).\n3. ≥ 16 years of age;\n4. Immunocompromised as defined by ≥ 1 risk factors for severe COVID-19 as assessed by the Federal Office of Public Health (FOPH) list (criteria 5: diseases\u002Ftreatments leading to immune suppression) or other immunosuppression criteria such as:\n\n   * Severe immunosuppression (e.g., human immunodeficiency virus (HIV) infection with CD4 + T cell count \\\u003C350 \u002F μl)\n   * Neutropenia (\\\u003C1000 neutrophils \u002F μl) ≥1 week\n   * Lymphocytopenia (\\\u003C200 lymphocytes\u002Fμl)\n   * On dialysis treatment\n   * Hereditary immunodeficiencies\n   * Intake of drugs which suppress the immune system (e.g. glucocorticoids for a long time \\[an equivalent dose of prednisone \\>20 mg\u002Fday \\> 3 months\\], monoclonal antibodies, cytostatics, biological products, everolimus, mTOR inhibitors etc.) in the last 12 months\n   * Active cancer under cytostatics or targeted therapy known to be immunosuppressive (e.g., platinum salts, cyclophosphamide, anthracyclines, taxanes, 5-fluorouracil, gemcitabine, purine inhibitors, proteasome inhibitors) or associated with hematologic toxicity (neutropenia, lymphopenia), for example sunitinib, imatinib, regorafenib.\n   * Aggressive lymphomas (all types)\n   * Acute lymphatic leukemia\n   * Acute myeloid leukemia\n   * Acute promyelocytic leukemia\n   * T prolymphocytic leukemia\n   * Primary central nervous system lymphoma\n   * Stem cell transplantation\n   * Light chain amyloidosis\n   * Chronic lymphoid leukemia\n   * Multiple myeloma\n   * Sickle cell disease\n   * Bone marrow transplant\n   * Organ transplant\n   * Being on the waiting list for an organ transplant\n5. Willing and able to comply with study requirements and restrictions as described in the informed consent form (ICF)\n6. Enrolled in or a beneficiary of a Social Security program (State Medical Aid (AME) is not a Social Security program) or holders of health insurance.\n7. Participant's or its legal representative's signature of the informed consent form\n\nExclusion Criteria:\n\n1. SARS-CoV-2 PCR ≥30 CT at screening\n2. Hypersensitivity to study drugs (active substance(s) or excipients)\n3. Body weight \\\u003C 40 kg\n4. AST (Aspartate transaminase) and\u002For alanine transaminase (ALT) \\> 5 times the upper limit\n5. Cirrhosis Child-Pugh score C\n6. Is taking or is anticipated to require any prohibited therapies\\*.\n7. Participation in another interventional clinical study with an investigational compound or device, including COVID-19 therapeutics, where the study intervention is performed in the 28 days preceding the inclusion and the 10 days after the inclusion. Investigators of the different clinical studies should agree on participant's inclusion.\n8. Presence of any condition for which, in the opinion of the investigator, participation would not be in participant's best interest or that could prevent, limit, or confound the protocol-specified assessments\n9. Having received antiviral treatments against SARS-CoV-2 in the 14 days before the inclusion with exception of those having received one or two doses of nirmatrevir\u002Fr in the 24h preceding the inclusion in the study.\n10. Pregnant or breastfeeding female",{"count":182,"type":21},[24],"The overall purpose of the trial is to evaluate the efficacy and safety of possible combination antiviral therapy direct antiviral agents (remdesivir + nirmatrelvir\u002Fr) versus the reference monotherapy (nirmatrelvir\u002Fr alone) and to assess the efficacy and safety of increasing the nirmatrelvir\u002Fr course from 5- to 10 days in immunocompromised patients diagnosed with asymptomatic or mild to moderate Coronavirus Disease 2019 (COVID-19).",[186,28],[186,211,212,213,214,215],"Immunodepression","SARS-CoV-2","Paxlovid","Veklury","Viral load","2026-02-13",{"date":218,"type":39},"2026-02-18",{"date":191,"type":39},{"date":221,"type":21},"2026-06-30",{"name":223,"class":102},"Calmy Alexandra",4,{"id":226,"slug":227,"hasResults":11,"nctId":228,"briefTitle":229,"officialTitle":230,"acronym":231,"eligibilityCriteria":232,"healthyVolunteers":11,"sex":16,"minAge":86,"maxAge":4,"enrollmentInfo":233,"targetDuration":4,"studyType":22,"phases":235,"briefSummary":237,"conditions":238,"keywords":242,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":247,"lastUpdatePostDateStruct":248,"startDateStruct":250,"completionDateStruct":252,"leadSponsor":254,"locationsCount":47},"100564858","phase-4-clozapine-related-immunodeficiency-in-parkinsons-disease-100564858","NCT06634641","Clozapine-related Immunodeficiency in Parkinsons Disease","Assessment of Clozapine-related Immunodeficiency Effect in Parkinsons Disease Patients","CLOZIDPD","Inclusion Criteria:\n\n* Patient ≥ 18 years old with Parkinson's disease according to MDS 2015 criteria\n* Psychotic symptoms requiring treatment with Clozapine\n* Patients with initially a normal leukocyte count (number of white blood cells\n\n  ≥ 3500\u002Fmm3 \\[3.5 x 109\u002Fl\\] and an absolute neutrophil count PNN ≥ 2000\u002Fmm3 \\[2 x 109\u002Fl\\])\n* patients in whom the number of white blood cells (WBC) and the absolute number of neutrophils (PNN) may be determined regularly at the following intervals: once a week during the first 18 weeks of treatment and, thereafter, at least every 4 weeks for the duration of the treatment. This monitoring must be continued throughout the treatment and for 4 weeks who follow the complete cessation of CLOZAPINE\n* Informed and written consent.\n* Affiliation to a social security system\n\nExclusion Criteria:\n\n* Patients with a contraindication to the use of Clozapine according to the summary of product characteristics (SPC)\n* Hypersensitivity to the active substance or to any of the excipients.\n* Patients who cannot receive regular blood tests.\n* History of granulopenia or toxic or idiosyncratic agranulocytosis (unless it results from previous chemotherapy).\n* History of agranulocytosis induced by CLOZAPINE\n* Treatment with CLOZAPINE should not be started at the same time as substances known to have a high potential for inducing agranulocytosis; The concomitant administration of depot antipsychotics is not recommended.\n* Functional bone marrow failure.\n* Uncontrolled epilepsy.\n* Alcoholic or induced psychosis, drug intoxication, comatose states.\n* Circulatory collapse and \u002F or CNS depression regardless of the aetiology.\n* Severe renal or cardiac disorders (eg: myocarditis).\n* Active liver disease with nausea, anorexia or jaundice; progressive liver disease, liver failure.\n* Paralytic ileus.\n* Patient with another potential cause of immunosuppression\n* Immunosuppressive or immune modulatory treatment active or stopped for less than 5 years\n* Anti-epileptic treatment active or stopped for less than 5 years\n* Chemotherapy active or stopped for less than 5 years\n* Solid or hematologic cancer active or treated for less than 5 years\n* Human immunodeficiency virus infection\n* Already known constitutional immune deficiency\n* Nephrotic syndrome\n* Protein-losing enteropathy\n* A history of radiotherapy\n* Long-term use of corticosteroids\n* Patient with potentially major cognitive disorders defined by a MoCA score less than or equal to 23\n* Pregnant or breastfeeding women\n* Patient under guardianship\u002Fcuratorship or deprived of liberty",{"count":234,"type":21},24,[236],"PHASE4","Clozapine is a second generation antipsychotic drug used in psychiatry to treat schizophrenia, affective disorders or certain symptoms of dementia. In neurology, clozapine is frequently used and recommended to manage symptoms of psychosis associated with Parkinson's disease (PD). The risk of neutropenia or agranulocytosis associated with clozapine estimated at 1.3% is well known to doctors around the world with a peak at one month and a decrease in risk after more than a year of treatment. This risk has led to the policy of \"no blood, no drugs\" and monitoring of the complete blood count (CBC) weekly for 18 weeks and then monthly for the duration of treatment.\n\nSome studies suggest an increased risk of infections related to immunodeficiency induced by clozapine itself. This clozapine-induced immunodeficiency would be comparable to that encountered in patients with common variable immunodeficiency or under immunosuppressive treatment. In addition, this immunosuppressive effect linked to clozapine would not be dose dependent but time dependent. However, the only studies currently performed have been in psychiatric patients treated for schizophrenia.\n\nIt seems important to specifically explore clozapine-related immunodeficiency in PD patients treated with clozapine for PD-related psychosis. In this study, the investigators propose to evaluate the variations in serum immunoglobulin levels and lymphocyte subpopulations (B, T, NK) in parkinsonian patients treated with Clozapine at 6 months and 1 year after initiation of treatment.",[239,240,28,241],"Clozapine","Parkinson's Disease (PD)","Psychosis",[243,244,245,246],"clozapine","parkinson's disease","immunodeficiency","psychosis","2025-11-17",{"date":249,"type":39},"2025-11-19",{"date":251,"type":39},"2024-10-01",{"date":253,"type":21},"2027-09",{"name":255,"class":102},"Centre Hospitalier Universitaire, Amiens",{"id":257,"slug":258,"hasResults":11,"nctId":259,"briefTitle":260,"officialTitle":260,"acronym":261,"eligibilityCriteria":262,"healthyVolunteers":85,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":263,"targetDuration":4,"studyType":58,"phases":4,"briefSummary":265,"conditions":266,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":271,"lastUpdatePostDateStruct":272,"startDateStruct":274,"completionDateStruct":276,"leadSponsor":278,"locationsCount":47},"100535217","systems-level-analyses-of-immune-dysregulation-100535217","NCT06248957","SYSTEMS-LEVEL ANALYSES OF IMMUNE DYSREGULATION","SAID","Inclusion Criteria:\n\nPatients of all ages seeking care or being referred for suspected immune dysregulation or with a known immune-mediated disease and failing to respond to standard therapy\n\nExclusion Criteria:\n\nHealthy control individuals will be excluded on the basis of having a diagnosis of an immune mediated disorder, immunomodulatory treatment or current infection or cancer.",{"count":264,"type":21},500,"The aim of the SAID study is to create a national resource in Sweden to enable comprehensive immunological analyses of an extremely complex and clinically challenging group of individuals with variable forms of immune system dysregulation. We hope to establish a biobank of primarily blood and fecal samples from children and adults, with confirmed or suspected immune dysregulation, as well as age- and sex- matched healthy controls, for comparisons of immune cell\u002Fmediator alongside various clinical presentations of these immunological diseases as well as microbiome samples as possible a possible modifier of clinical presentations. The project will also include the establishment of a national database with deep immunological data, treatment and clinical outcomes for these patients, accessible to participating researchers and clinicians.",[28,267,268,269,270],"Autoimmune Diseases","Autoinflammatory Syndrome","Allergy","Dysregulated Host Response","2024-01-31",{"date":273,"type":39},"2024-02-08",{"date":275,"type":39},"2024-01-01",{"date":277,"type":21},"2031-12-31",{"name":279,"class":102},"Karolinska Institutet"]