[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"autoimmune-lymphoproliferative\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:autoimmune-lymphoproliferative":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,51,80],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":15,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":32,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":40,"startDateStruct":43,"completionDateStruct":45,"leadSponsor":47,"locationsCount":50},"100253594","phase-2-pilot-trial-of-allogeneic-blood-or-marrow-transplantation-for-primary-immunodeficiencies-100253594",false,"NCT02579967","Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies","* INCLUSION CRITERIA - RECIPIENT:\n* Patients age \\>= 4 through 75 years\n* PID deemed to be of sufficient past severity to warrant allo BMT, by meeting the two criteria below:\n\n  1. PID as defined by identified genetic defect or, in the absence of a PID-associated genetic mutation, patients with an immune defect potentially amenable to allo BMT who meet the clinical history criteria below may be eligible upon discussion with the PI\n\n     * Mutations should be confirmed in a CLIA-certified laboratory, if such testing is available.\n     * Patients without a mutation must be deemed eligible and appropriate for allo BMT by the PI. Some patients may meet the clinical history criteria listed below, but will not be eligible if it is thought that their clinical history is due to a condition apart from an immune defect. In addition, patients with a PID of mild severity, such as those with selective IgA deficiency, may meet at least two of the clinical history criteria, but may be deemed inappropriate for allo BMT by the PI if it is felt that the risks of the procedure outweigh the severity of the disease.\n  2. Clinical history of at least two of the following:\n\n     * Life-threatening, organ-threatening, or severely disfiguring infection\n     * Protracted or recurrent infections requiring unusually long or repeated courses of antibiotics\n     * Infection with an opportunistic organism\n     * Chronic elevation in the blood (\\>=2 documented elevations over a period of 6 months or longer) of a latent virus (EBV, CMV, HHV6, HHV8, etc.)\n     * Evidence of immune dysregulation, as manifested by autoimmune disease, atopy, hemophagocytic lymphohistiocytosis\u002Fmacrophage activation syndrome, granulomas, splenomegaly, or lymphadenopathy\n     * Patients with hemophagocytic lymphohistiocytosis or macrophage activation syndrome related to an underlying lymphoma with no other clinical history suggestive of a primary immunodeficiency will not be eligible\n     * Hypogammaglobulinemia, dysglobulinemia, or impaired response to vaccination\n     * Hematologic malignancy or lymphoproliferative disorder\n     * Tissue diagnosis should be confirmed by NCI Department of Pathology, if prior biopsies are available\n     * Virus-associated solid tumor malignancy or pre-cancerous lesion\n     * Tissue diagnosis should be confirmed by NCI Department of Pathology, if prior biopsies are available\n* Availability of at least one 7-8\u002F8 (9-10\u002F10) HLA-matched related (excluding an identical twin) or unrelated donor, or an HLA-haploidentical related donor\n* Consensus among the PI, key AIs, and consultants (as necessary) that correction of the patient s immune system through BMT has the potential to improve the patient s health, quality of life, and\u002For life expectancy, after taking into consideration the patient s existing non-hematopoietic, potentially irreversible organ dysfunction\n* Adequate end-organ function, as measured by:\n\n  * Left ventricular ejection fraction (LVEF) \\>= 40% by 2D echocardiogram (ECHO) or MUGA, or left ventricular shortening fraction \\>= 20% by ECHO for patients receiving RIC or RIC-MMF, or RIC-SHORT, or LVEF \\>= 30% if the patient has radiologic evidence of aortic, renal, or coronary artery vasculitis.\n  * Pulmonary function tests: DL(co) (corrected for hemoglobin) and FEV(1) \\>= 40% of predicted for the RIC, RIC-MMF, and RIC-SHORT arms; or in pediatric patients, if unable to perform pulmonary function tests, there should be no evidence of dyspnea at rest, no requirement for supplemental oxygen, and oxygen saturation \\>92% on room air. Calculations will be based on the values reported in CRIS.\n  * Bilirubin \\\u003C= 3.0 mg\u002FdL (unless due to Gilbert s syndrome or hemolysis) for patients receiving RIC, RIC-MMF, RIC-SHORT; ALT and AST 10 x ULN for patients receiving RIC, RIC-MMF, RIC-SHORT. Patients who are above these bilirubin, ALT, or AST thresholds may be eligible for the RIC, RIC-MMF, or RIC-SHORT arms if evaluated by a hepatologist who deems the liver function test abnormalities to be potentially reversible with bone marrow transplant.\n  * Estimated creatinine clearance of \\>= 40 mL\u002Fmin\u002F1.73 m\\^2, calculated using the Cockcroft-Gault equation for adults and Schwartz formula for pediatric patients, for patients with creatinine levels above the institutional upper limit of normal\n* Karnofsky or Lansky performance status of \\>=60% or ECOG performance status of 2 or less\n* Ability of subject or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document\n* Not pregnant or breastfeeding. 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 BMT. 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.\n* Disease status: Patients with malignancy are to be referred in remission for evaluation, except in cases of virus-associated malignancy who may be referred at any time. Should a patient have progressive disease or a donor becomes unavailable after enrollment, the patient will be referred back to his\u002Fher primary hematologist-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, then the patient may receive standard treatment for the malignant disease under the current study, although this should only occur as a bridge to transplant. If under either of these settings, it becomes apparent that the patient will not be able to proceed to transplant, then he\u002Fshe must come off the study. Patients receiving standard therapy will be told about the therapy, associated risks, potential benefits, alternatives to the proposed therapy, and the availability of receiving the same treatment elsewhere, outside of a research protocol.\n\nEXCLUSION CRITERIA - RECIPIENT:\n\n* 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 BMT.\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (cyclophosphamide, busulfan, pentostatin, sirolimus, MMF, filgrastim or filgrastim biosimilar) used in the study\n* Active psychiatric disorder which may compromise compliance with the transplant protocol, or which does not allow for appropriate informed consent\n* Active central nervous system (CNS) involvement by malignancy, except in cases of virus-associated malignancies with CNS involvement in which case the patient may benefit from the transplant to control the malignancy.\n* MAGT1 mutation and active need to take anti-platelet agents and\u002For therapeutic anti-coagulation that cannot be interrupted during aplasia.\n* HIV positive or other acquired immunodeficiency that, as determined by the PI, interferes with the assessment of PID severity and\u002For the attribution of clinical manifestations of immunodeficiency to a PID.\n* Lack of adequate central venous access potential\n\nInclusion Criteria (Related Donor):\n\n* Ages \\>= 4\n* Related donor deemed suitable and eligible and willing to donate per clinical evalations who are additionally willing to donate blood, urine, and marrow specimens for research. Related donors will be evaluated in accordance with existing Standard Policies and Procedures for determination of eligibility and suitability for clinical donation. Note that participation in this study is offered to all related donors but is not required for clinical donation, so it is possible that not all related donors will enroll on this study.\n\nExclusion Criteria (Related Donor):\n\nNone\n\nINCLUSION CRITERIA - UNRELATED DONOR:\n\n* Ages \\>= 18\n* Unrelated donors will be evaluated in accordance with existing NMDP Standard Policies and Procedures, available at: http:\u002F\u002Fbethematch.org\u002FAbout-Us\u002FGlobal-transplant-network\u002FStandards\u002F, except for the additional requirement of EBV serostatus testing. Note that participation in this study is offered to all unrelated donors but not required for clinical donation, so it is possible that not all unrelated donors will enroll on this study.\n\nEXCLUSION CRITERIA - UNRELATED DONOR:\n\n-Unrelated donors: failure to qualify as a National Marrow Donor Program (NMDP) donor per current NMDP Standards, available at: http:\u002F\u002Fbethematch.org\u002FAbout-Us\u002FGlobal-transplant-network\u002FStandards\u002F.",true,"ALL","4 Years","75 Years",{"count":20,"type":21},354,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","Background:\n\nAllogeneic blood or marrow transplant is when stem cells are taken from one person s blood or bone marrow and given to another person. Researchers think this may help people with immune system problems.\n\nObjective:\n\nTo see if allogeneic blood or bone marrow transplant is safe and effective in treating people with primary immunodeficiencies.\n\nEligibility:\n\nDonors: Healthy people ages 4 or older\n\nRecipients: People ages 4-75 with a primary immunodeficiency that may be treated with allogeneic blood or marrow transplant\n\nDesign:\n\nParticipants will be screened with medical history, physical exam, and blood tests.\n\nParticipants will have urine tests, EKG, and chest x-ray.\n\nDonors will have:\n\nBone marrow harvest: With anesthesia, marrow is taken by a needle in the hipbone.\n\nOR\n\nBlood collection: They will have several drug injections over 5-7 days. Blood is taken by IV in one arm, circulates through a machine to remove stem cells, and returned by IV in the other arm.\n\nPossible vein assessment or pre-anesthesia evaluation\n\nRecipients will have:\n\nLung test, heart tests, radiology scans, CT scans, and dental exam\n\nPossible tissue biopsies or lumbar puncture\n\nBone marrow and a small piece of bone removed by needle in the hipbone.\n\nChemotherapy 1-2 weeks before transplant day\n\nDonor stem cell donation through a catheter put into a vein in the chest or neck\n\nSeveral-week hospital stay. They will take medications and may need blood transfusions and additional procedures.\n\nAfter discharge, recipients will:\n\nRemain near the clinic for about 3 months. They will have weekly visits and may require hospital readmission.\n\nHave multiple follow-up visits to the clinic in the first 6 months, and less frequently for at least 5 years.",[27,28,29,30,31],"Primary T-cell Immunodeficiency Disorders","Common Variable Immunodeficiency","Immune System Diseases","Autoimmune Lymphoproliferative","Lymphoproliferative Disorders",[33,34,35,36,37],"Haploidentical","Autoimmunity","Immune Dysregulation","Congenital","Opportunistic Infection","RECRUITING","2026-06-27",{"date":41,"type":42},"2026-06-30","ACTUAL",{"date":44,"type":42},"2015-11-19",{"date":46,"type":21},"2036-12-31",{"name":48,"class":49},"National Cancer Institute (NCI)","NIH",2,{"id":52,"slug":53,"hasResults":11,"nctId":54,"briefTitle":55,"officialTitle":56,"acronym":4,"eligibilityCriteria":57,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":58,"targetDuration":4,"studyType":22,"phases":60,"briefSummary":61,"conditions":62,"keywords":64,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":69,"lastUpdatePostDateStruct":70,"startDateStruct":72,"completionDateStruct":74,"leadSponsor":76,"locationsCount":79},"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","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.",{"count":59,"type":21},39,[24],"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\"",[63,30,29],"Primary Immune Regulatory Disorder",[65,66,67,68],"Stem Cell Transplant","Emapalumab","Fludarabine","Dexamethasone","2026-05-29",{"date":71,"type":42},"2026-06-02",{"date":73,"type":42},"2023-03-15",{"date":75,"type":21},"2027-03",{"name":77,"class":78},"Joseph Oved","OTHER",13,{"id":81,"slug":82,"hasResults":11,"nctId":83,"briefTitle":84,"officialTitle":85,"acronym":4,"eligibilityCriteria":86,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":87,"enrollmentInfo":88,"targetDuration":4,"studyType":22,"phases":90,"briefSummary":91,"conditions":92,"keywords":93,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":94,"lastUpdatePostDateStruct":95,"startDateStruct":97,"completionDateStruct":99,"leadSponsor":101,"locationsCount":102},"100388586","phase-2-allogeneic-hematopoietic-stem-cell-transplant-for-patients-with-inborn-errors-of-immunity-100388586","NCT04339777","Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity","A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity","* INCLUSION CRITERIAl:\n* Age \\>= 4 years and \\\u003C=69 yo with Weight \\>=12 kilograms\n* Mutation in a known monogenic (IEI) gene performed by a CLIA certified laboratory, who have failed standard medical management, or when no standard medical management is available.\n\nOR\n\nPatients without a known IEI mutation may be eligible if they have a clinical history that is characteristic of an individual with an immune defect including a history of infections requiring prolonged courses of therapy or evidence of immune dysregulation manifested by autoimmune\u002Fautoinflammatory disease, atopy, hemophagocytic lymphohistiocytosis, hypogammaglobulinemia, or impaired response to vaccination. A virally-driven malignancy alone will also constitute basis for inclusion.\n\n* Availability of an 8\u002F8, 7\u002F8, or 6\u002F8 HLA-matched related or unrelated donor (if the mismatch is at DQ this will be considered an 8\u002F8 matched donor), or a haploidentical related donor. Karnofsky or Lansky performance status of \\>= 40%\n* Adequate end-organ function, as measured by:\n\n  --Left ventricular ejection fraction \\> 40%, preferably by 2-D echocardiogram (ECHO) obtained within 60 days prior to enrollment.\n* Creatinine: Adult patients: \\\u003C= 2.0 mg\u002Fdl and creatinine clearance \\>= 30 ml\u002Fmin; Pediatric patients (\\\u003C18 years old): creatinine \\\u003C 1.5 mg\u002FdL and a creatinine clearance, using the Schwartz Formula \\> 30 mL\u002Fmin\u002F1.73m\\^2.\n\n  * Serum conjugated bilirubin \\\u003C 2.5 mg\u002Fdl; serum ALT and AST \\\u003C= 5 times upper\n\nlimit of normal.\n\n--Pulmonary function tests: FEV1 \\> 30% and DLCO \\>30%. Children who are unable to have DLCO assessed due to age, are still eligible if no evidence of dyspnea at rest and no need for supplemental oxygen.\n\n* Ability of subject or parent\u002Fguardian to understand and the willingness to sign a written informed consent document. For subjects \\\u003C18 years old, their legal guardian must give informed consent. Pediatric patients will provide assent.\n* 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 HCT. 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.\n* Willingness to 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. If outpatient in the first 100 days after transplant, patient must commit to having an adult caregiver with them at all times.\n\nEXCLUSION CRITERIA:\n\n* Patients who are receiving any other investigational agents (with the exception of virus-specific therapy e.g. cytotoxic T-cells for the treatment of viral infection\u002Freactivation prior to allo HCT).\n* Patients with known brain metastases should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events.\n* 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.\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (steroids, cyclophosphamide, busulfan, tacrolimus, sirolimus, MMF, G-CSF, alemtuzumab) used in the study\n* Active psychiatric disorder which is deemed by the PI to have significant risk of compromising compliance with the transplant protocol or which does not allow for appropriate informed consent\n* Pregnant women 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 including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.","69 Years",{"count":89,"type":21},66,[24],"Background:\n\nDuring a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies. But transplant has many risks and complications.\n\nObjective:\n\nTo see if stem cell transplant can be successfully performed in people with primary immunodeficiency disease and cure them.\n\nEligibility:\n\nPeople ages 4-69 for whom a primary immunodeficiency (PID) or Primary Immune Regulatory Disorder (PIRD), has caused significant health problems and either standard management has not worked or there are no standard management options, along with their donors\n\nDesign:\n\nDonors will be screened under protocol 01-C-0129. They will donate blood or bone marrow.\n\nParticipants will be screened with:\n\nMedical history\n\nPhysical exam\n\nBlood, urine, and heart tests\n\nCT or PET scans\n\nBefore transplant, participants will have dental and eye exams. They will have a bone marrow biopsy. For this, a needle will be inserted through the skin into the pelvis to remove marrow.\n\nParticipants will be hospitalized before their transplant. They will have a central catheter put into a vein in their chest or neck. They will get medications through the catheter to prevent complications. Participants will get stem cells through the catheter. They will stay in the hospital for at least 4 weeks. They will give blood, urine, bone marrow, and stool samples. They may need blood transfusions. They may need more scans. They will take more medications.\n\nParticipants will have visits on days 30, 60, 100, 180, and 360, and 24 months after the transplant. Then they will have visits once a year for about 5 years",[31,30,29,28,27],[34,33,37,35,36],"2026-05-01",{"date":96,"type":42},"2026-05-04",{"date":98,"type":42},"2020-09-22",{"date":100,"type":21},"2027-11-30",{"name":48,"class":49},1]