[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"immune-system-diseases\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:immune-system-diseases":32},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,15,0,[8,49,81,107,138,167,195,220,241,279,323,352,380,414,457],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100054307","phase-2-metabolic-modulation-to-enhance-insulin-sensitivity-and-mitochondrial-function-in-type-1-diabetes-metmod-t1d-100054307",false,"NCT07699380","METabolic MODulation to Enhance Insulin Sensitivity and Mitochondrial Function in Type 1 Diabetes (MetMod-T1D)","MetMod-T1D","Inclusion Criteria:\n\n1. Adults ≥18 years to \\\u003C70 years of age with established T1D (duration ≥1 year)\n2. Currently on insulin therapy (multiple daily injections or insulin pump)\n3. HbA1c \\\u003C9.5%\n4. BMI 18.5-40 kg\u002Fm2\n5. On stable dose of RASB or statin, if indicated\n6. Willing and able to comply with all study procedures\n\nExclusion Criteria:\n\n1. History of pancreatic disease (including pancreatitis) or pancreatic surgery\n2. History of cardiovascular disease or stroke within the past 6 months\n3. History of heart failure per New York Heart Association criteria\n4. History of severe edema or salt restriction requirement\n5. Biliary disease or pathologies that may alter enterohepatic circulation of bile acids\n6. Estimated glomerular filtration rate (eGFR) \\\u003C60 mL\u002Fmin\u002F1.73m²\n7. Liver disease (ALT\u002FAST \\>3x upper limit of normal \\[ULN\\])\n8. Pregnancy, breastfeeding, or planning pregnancy during the study period\n9. Known hypersensitivity to study drug components\n10. Abnormal baseline ECG\n11. Use of off label medications that affect insulin sensitivity within the past 1 month (e.g., metformin, GLP-1RA, SGLT2i, pioglitazone)\n12. Chronic use of anticoagulants\n13. Use of bile acid sequestering agents, inhibitors of bile acid transporters, bile acid derivatives, aluminum-based antacids, probenecid, pan-HDAC inhibitors, phase 2 metabolizing enzymes (e.g., uridine diphosphate glucuronosyl transferases), phase 1 metabolizing enzymes other than cytochrome P450 enzymes (CYPs), and OATP1B3\n14. Use of substrates of CYP1A2, CYP2C8, CYP2B6, CYP3A4, Organic Anion transporter 1, P-glycoprotein, and Breast Cancer Resistance Protein\n15. History of severe hypoglycemia requiring assistance within the past 3 months\n16. History of diabetic ketoacidosis (DKA) within the past 3 months\n17. Personal or family history of breast cancer or ovarian cancer\n18. Current participation in another clinical trial\n19. Any condition(s) found by the study team and confirmed with the Investigator that make it unsafe to participate","ALL","18 Years","69 Years",{"count":20,"type":21},60,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","The study is a randomized, double-blind, parallel-group clinical trial to examine the effects of 24 weeks of oral AMX0035 (sodium phenylbutyrate + taurursodiol) versus placebo in 60 adults with Type 1 Diabetes (T1D) (n=30 per arm). Enrollment will be distributed equally between the University of Washington and Amsterdam University Medical Center\u002FDiabetes Center Amsterdam. Participants will be recruited through diabetes research registries, local T1D clinics, and community outreach.",[27,28,29,30,31,32,33,34,35],"Type 1 Diabetes (T1D)","Metabolic Diseases","Glucose Metabolism Disorders","Endocrine System Diseases","Autoimmune Diseases","Immune System Diseases","Diabetes Melletus, Type 1","Nutritional and Metabolic Diseases","Combination Therapy","RECRUITING","2026-07-09",{"date":39,"type":40},"2026-07-13","ACTUAL",{"date":42,"type":21},"2026-06",{"date":44,"type":21},"2029-12",{"name":46,"class":47},"University of Washington","OTHER",2,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":4,"eligibilityCriteria":55,"healthyVolunteers":56,"sex":16,"minAge":57,"maxAge":58,"enrollmentInfo":59,"targetDuration":4,"studyType":22,"phases":61,"briefSummary":62,"conditions":63,"keywords":66,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":71,"lastUpdatePostDateStruct":72,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":48},"100356570","phase-2-allogeneic-hematopoietic-cell-transplantation-for-peripheral-t-cell-lymphoma-100356570","NCT03922724","Allogeneic Hematopoietic Cell Transplantation for Peripheral T Cell Lymphoma","Phase II Trial of Allogeneic Hematopoietic Cell Transplantation for Peripheral T Cell Lymphoma","* INCLUSION CRITERIA-RECIPIENT:\n* Age \\>=12 years\n* Diagnosis of PTCL, confirmed by NCI pathology review, that is relapsed or refractory to prior therapy, and\u002For PTCL where upfront allo HCT in first remission is reasonable (PIT score of intermediate-low risk or higher or supported by clinical practice guidelines)\n\n  --ALK-positive ALCL patients will only be eligible if relapsed or refractory\n* At least one potential 7-8\u002F8 HLA-matched related (excluding an identical twin) or unrelated donor (at HLA-A, -B, -C, and DR), or an HLA-haploidentical related donor, based on initial low resolution unrelated donor search and\u002For at least one biologically-related family member who has at least a 25% chance of being at minimum an HLAhaploidentical match and is potentially suitable to donate based on reported family history. HLA typing of potential donors and\u002For mutation testing does not need to be completed for eligibility.\n* Adequate end-organ function, as measured by:\n\n  * For RIC: Left ventricular ejection fraction (LVEF) \\>= 40% by 2D echocardiogram (ECHO) or MUGA, left ventricular shortening fraction \\>= 20% by ECHO, or LVEF \\>= 30% if the patient has radiologic evidence of aortic, renal, or coronary artery vasculitis. For IOC: LVEF \\>= 30% by 2D ECHO or MUGA.\n  * Pulmonary function tests: DLco (corrected for hemoglobin) and FEV1 \\>= 40% of predicted for the RIC arm, and \\>= 30% predicted for the IOC arm; 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.\n  * Bilirubin \\\u003C= 3.0 mg\u002FdL (unless due to Gilbert's syndrome or hemolysis) for patients receiving RIC and bilirubin \\\u003C= 5.0 mg\u002FdL for patients receiving IOC (unless due to Gilbert s syndrome or hemolysis); ALT and AST \\\u003C= 10 x ULN for patients receiving RIC or IOC. Patients who are above these bilirubin, ALT, or AST thresholds may be eligible for the RIC or IOC arm if evaluated by a hepatologist who deems the liver function test abnormalities to be potentially disease related, either because of direct involvement by PTCL, due to an associated process such as hemophagocytic lymphohistiocytosis, or as sequelae of prior chemotherapy that is thought to improve with time.\n  * Estimated creatinine clearance of \\>= 50 mL\u002Fmin\u002F1.73 m\\^2, calculated using eGFR in the clinical lab for adults and the Schwartz formula for pediatrics.\n* Karnofsky (adults) or Lansky (children) performance status of \\>= 50% or ECOG performance status of 2 or less for the RIC arm and Karnofsky (adults) or Lansky (children) \\>= 30% or ECOG performance status of 3 or less for the IOC arm\n* Ability of subject or parent\u002Fguardian to understand and the willingness to sign a written informed consent document\n* Not pregnant or breastfeeding.\n* As therapeutic agents used in this trial may be harmful to a fetus, individuals of childbearing potential and individuals who can father children 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.\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 HCT.\n* Prohibitive allergy to a study drug or to compounds of similar chemical or biologic composition of the agents (e-ATG, steroids, cyclophosphamide, busulfan, pentostatin, sirolimus, MMF, filgrastim or biosimilar drug) used in the study\n* Lack of central venous access potential\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\nINCLUSION CRITERIA-RELATED DONOR:\n\n-Related donor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood and\u002For peripheral blood stem cells for research. Related donors will be evaluated in accordance with existing Standard Policies and Procedures for determination of eligibility and suitability for clinical donation.\n\nEXCLUSION CRITERIA-RELATED DONOR:\n\nNone\n\nINCLUSION CRITERIA (UNRELATED DONOR):\n\n-Unrelated donors will be evaluated in accordance with existing NMDP Standard Policies and Procedures, available at: http:\u002F\u002Fbethematch.org\u002FAbout-Us\u002FGlobal-transplantnetwork\u002F Standards\u002F, except for the additional requirement of EBV serostatus testing for clinical purposes of donor selection. 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. Unrelated donors only enroll if they contribute research specimens, which is optional.\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\u002FGlobaltransplant- network\u002FStandards\u002F. Exceptions to donor eligibility (e.g. foreign travel, tattoos) do not automatically exclude the donor and will be reviewed by the PI.",true,"12 Years","120 Years",{"count":60,"type":21},330,[24],"Background:\n\nLymphoma is a type of blood cancer. Blood cell transplant can cure some people with lymphoma. Researchers want to see if they can limit the complications transplant can cause.\n\nObjective:\n\nTo test if a stem cell transplant can cure or control lymphoma. Also to test if new ways of getting a recipient ready for a transplant may result in fewer problems and side effects.\n\nEligibility:\n\nRecipients: People ages 12 and older with peripheral T cell lymphoma that does not respond to standard treatments\n\nDonors: Healthy people ages 18 and older whose relative has lymphoma\n\nDesign:\n\nParticipants will be screened with:\n\nPhysical exam\n\nBlood and urine tests\n\nBone marrow biopsy: A needle inserted into the participant s hip bone will remove marrow.\n\nDonors will also be screened with:\n\nX-rays\n\nRecipients will also be screened with:\n\nLying in scanners that take pictures of the body\n\nTumor sample\n\nDonors may donate blood. They will take daily shots for 5 7 days. They will have apheresis: A machine will take blood from one arm and take out their stem cells. The blood will be returned into the other arm.\n\nRecipients will be hospitalized at least 2 weeks before transplant. They will get a catheter: A plastic tube will be inserted into a vein in the neck or upper chest. They will get antibody therapy or chemotherapy.\n\nRecipients will get the transplant through their catheter.\n\nRecipients will stay in the hospital several weeks after transplant. They will get blood transfusions. They will take drugs including chemotherapy for about 2 months.\n\nRecipients will have visits 6, 12, 18, 24 months after transplant, then once a year for 5 years.",[64,65,32],"Peripheral T-cell Lymphomas","Lymphoproliferative Disorders",[67,68,69,70],"Autoimmunity","Immune Dysregulation","Congenital","Opportunistic Infection","2026-07-01",{"date":73,"type":40},"2026-07-02",{"date":75,"type":40},"2019-04-18",{"date":77,"type":21},"2030-10-31",{"name":79,"class":80},"National Cancer Institute (NCI)","NIH",{"id":82,"slug":83,"hasResults":11,"nctId":84,"briefTitle":85,"officialTitle":85,"acronym":4,"eligibilityCriteria":86,"healthyVolunteers":56,"sex":16,"minAge":87,"maxAge":88,"enrollmentInfo":89,"targetDuration":4,"studyType":22,"phases":91,"briefSummary":92,"conditions":93,"keywords":97,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":48},"100253594","phase-2-pilot-trial-of-allogeneic-blood-or-marrow-transplantation-for-primary-immunodeficiencies-100253594","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.","4 Years","75 Years",{"count":90,"type":21},354,[24],"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.",[94,95,32,96,65],"Primary T-cell Immunodeficiency Disorders","Common Variable Immunodeficiency","Autoimmune Lymphoproliferative",[98,67,68,69,70],"Haploidentical","2026-06-27",{"date":101,"type":40},"2026-06-30",{"date":103,"type":40},"2015-11-19",{"date":105,"type":21},"2036-12-31",{"name":79,"class":80},{"id":108,"slug":109,"hasResults":11,"nctId":110,"briefTitle":111,"officialTitle":111,"acronym":4,"eligibilityCriteria":112,"healthyVolunteers":56,"sex":16,"minAge":113,"maxAge":58,"enrollmentInfo":114,"targetDuration":4,"studyType":116,"phases":4,"briefSummary":117,"conditions":118,"keywords":123,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":132,"startDateStruct":133,"completionDateStruct":4,"leadSponsor":135,"locationsCount":137},"100058416","natural-history-management-and-genetics-of-the-hyperimmunoglobulin-e-recurrent-infection-syndrome-hies-100058416","NCT00006150","Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES)","* INCLUSION CRITERIA:\n\nPatients may be included in this study who:\n\n* Were referred to the NIH with a diagnosis or a suspicion of Hyper IgE syndrome.\n* Are patients referred for other immune syndromes that demonstrate some of the characteristics of HIES.\n\n  * \\>=1 month for affected subjects\n  * Aged \\>=2 years for unaffected subjects\n* For unaffected subjects, are able to understand and have the willingness to sign a written informed consent document.\n\nUnaffected biological relatives of HIES patients are also eligible to enroll in a separate relative cohort.\n\nEXCLUSION CRITERIA:\n\nCoronary CTA will not be performed on any patient younger than 30 years or with contraindication to IV contrast media. This includes patients with 1) creatinine value of \\>1.3 mg\u002FdL, 2) history of multiple myeloma, 3) Use of metformin-containing products less than 24 hours prior to contrast media, and 4) history of significant allergic reaction to CT contrast agents despite the use of premedication.\n\nSubjects with a medical, psychiatric, or social condition which, in the opinion of the investigator, would place undue burden on the subject, NIH resources, or increase risk of participation, may be excluded.","1 Month",{"count":115,"type":21},600,"OBSERVATIONAL","The Hyper IgE Syndromes (HIES) are primary immunodeficiencies resulting in eczema and recurrent skin and lung infections. Autosomal dominant Hyper IgE syndrome (AD-HIIES; Job's syndrome) is caused by STAT3 mutations, and is a multi-system disorder with skeletal, vascular, and connective tissue manifestations. Understanding how STAT3 mutations cause these diverse clinical manifestations is critical to our complete understanding of bone metabolism, bronchiectasis, dental maturation, and atherosclerosis. Bi-allelic mutations in DOCK8 cause a combined immunodeficiency previously described as autosomal-recessive Hyper IgE syndrome. These individuals suffer from extensive viral infections as well as have a high incidence of malignancy and mortality. The pathogenesis of this disease and long-term natural history is being investigated. Therefore, we seek to enroll patients and families with a confirmed or suspected diagnosis of HIES syndrome for extensive phenotypic and genotypic study as well as disease management. Patients will be carefully examined by a multidisciplinary team and followed longitudinally. Through these studies we hope to better characterize the clinical presentation of STAT3-mutated HIES, DOCK8 deficiency and other causes of the hyper IgE phenotype, and to be able to identify further genetic etiologies, as well as understand the pathogenesis of HIES. We seek to enroll 300 patients and 300 relatives....",[119,120,32,121,122],"Infections","Pneumonia","STAT3 Transcription Factor","Job Syndrome",[124,125,126,127,128,129,130,131],"DOCK8 Deficiency","PGM3 Deficiency","STAT3 Mutation","Job's Syndrome","Immunodeficiency","Natural History","Hyperimmunologobulin E Syndrome","HIE Syndrome",{"date":101,"type":40},{"date":134,"type":40},"2000-08-10",{"name":136,"class":80},"National Institute of Allergy and Infectious Diseases (NIAID)",1,{"id":139,"slug":140,"hasResults":11,"nctId":141,"briefTitle":142,"officialTitle":143,"acronym":4,"eligibilityCriteria":144,"healthyVolunteers":11,"sex":16,"minAge":145,"maxAge":146,"enrollmentInfo":147,"targetDuration":4,"studyType":116,"phases":4,"briefSummary":149,"conditions":150,"keywords":155,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":161,"lastUpdatePostDateStruct":162,"startDateStruct":164,"completionDateStruct":4,"leadSponsor":166,"locationsCount":137},"100054447","activation-and-function-of-eosinophils-in-conditions-with-blood-or-tissue-eosinophilia-100054447","NCT00001406","Activation and Function of Eosinophils in Conditions With Blood or Tissue Eosinophilia","Eosinophil Activation and Function in Parasitic Infections and Other Conditions With Increased Tissue or Peripheral Blood Eosinophilia in Humans","* INCLUSION CRITERIA:\n\nTo be eligible to participate in this study, an individual must meet all of the following criteria:\n\n1. Stated willingness to comply with all study procedures and availability for the duration of the study\n2. Male or female, aged 1-100\n3. Ability of subject (or Legally Authorized Representative (LAR)) to understand and sign a written informed consent document\n\n   Eosinophilic Patients only:\n4. Documented peripheral blood count \\>1500\u002Fmm3, tissue eosinophilia (abnormal accumulation of eosinophils in the skin or other body tissues) or suspected eosinophilic end organ involvement\n5. Primary (non-NIH) physician for routine medical care\n\n   Relatives only:\n6. Extended family member of an eosinophilic participant on this protocol\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n1. Any condition(s) or diagnosis, physical and\u002For psychological, that the investigator feels precludes the patient from participation in the study.\n\n   Relatives only:\n2. Females must not be pregnant","1 Year","100 Years",{"count":148,"type":21},800,"This study will investigate how, why and under what conditions eosinophils (a type of white blood cell) become activated and will examine their function in immune reactions. Eosinophil counts often rise in response to allergies, asthma, and parasitic worm infections. They can also go up in uncommon autoimmune conditions and, rarely, in association with tumors. Elevated levels of these cells is called eosinophilia. Usually, eosinophilia causes no apparent symptoms, but in rare cases there may be local swelling and itching, allergic lung problems, heart disease or nerve damage caused by the release of toxic substances in these cells into body tissues.\n\nPatients 1 to 100 years of age with eosinophil counts greater than 750\u002Fml or an abnormal accumulation of eosinophils in the skin or body tissues may be eligible for this study. All participants will have a thorough medical history, physical examination and blood tests. Depending on the person's age and symptoms, other diagnostic tests may be done, including specialized studies of the eye, lungs, skin, bone marrow, nerves or heart. This is not a treatment study, and no experimental treatments will be offered. Patients who require treatment will receive standard medical care.\n\nCertain other procedures may be requested solely for research purposes. All participants will be asked to donate extra blood for laboratory studies investigating how immune cells and other immune substances in the blood act to stimulate a rise in eosinophils. In addition, some participants may undergo one or more of the following:\n\n* Annual Follow-up evaluations - Physical examinations and blood tests to evaluate changes in the patient's condition and eosinophil counts over time.\n* Bone marrow biopsy and aspiration will be recommended during the initial evaluation, and in certain patients at other times when it is important to look directly at the newly developing cells in the bone marrow. For this procedure an area of skin and bone is anesthetized with xylocaine (an anesthetic similar to that used by dentists), and a very sharp needle is used to sample the bone marrow for evaluation. Bone marrow biopsy and aspiration can have side effects of pain and\u002For bleeding into the skin and soft tissues at the site of the procedure. Rarely the area at the biopsy site can become infected, and is treated with antibiotics.\n* Genetic testing: Some of the blood drawn from you as part of this study will be used for genetic tests. Genetic tests can help researchers study how health or illness is passed on to you by your parents or from you to your children. Any genetic information collected or discovered about you or your family will be confidential.\n* Leukapheresis (only patients 18 years and older) to collect large numbers of certain cells - In this procedure, whole blood is collected through a needle placed in an arm vein. The blood circulates through a machine that separates it into its components. The white cells are then removed and the rest of the blood is returned to the body, either through the same needle used to draw the blood or through a second needle placed in the other arm....",[32,151,152,153,154],"Eosinophilia","Helminthiasis","Hypersensitivity","Parasitic Disease",[156,157,158,159,160,129],"Eosinophils","Helminth Parasites","Hypereosinophilia","Allergy","Asthma","2026-06-03",{"date":163,"type":40},"2026-06-04",{"date":165,"type":40},"1997-04-21",{"name":136,"class":80},{"id":168,"slug":169,"hasResults":11,"nctId":170,"briefTitle":171,"officialTitle":172,"acronym":4,"eligibilityCriteria":173,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":174,"targetDuration":4,"studyType":22,"phases":176,"briefSummary":177,"conditions":178,"keywords":180,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":185,"lastUpdatePostDateStruct":186,"startDateStruct":188,"completionDateStruct":190,"leadSponsor":192,"locationsCount":194},"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":175,"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\"",[179,96,32],"Primary Immune Regulatory Disorder",[181,182,183,184],"Stem Cell Transplant","Emapalumab","Fludarabine","Dexamethasone","2026-05-29",{"date":187,"type":40},"2026-06-02",{"date":189,"type":40},"2023-03-15",{"date":191,"type":21},"2027-03",{"name":193,"class":47},"Joseph Oved",13,{"id":196,"slug":197,"hasResults":11,"nctId":198,"briefTitle":199,"officialTitle":200,"acronym":201,"eligibilityCriteria":202,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":203,"targetDuration":4,"studyType":22,"phases":205,"briefSummary":206,"conditions":207,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":185,"lastUpdatePostDateStruct":212,"startDateStruct":213,"completionDateStruct":215,"leadSponsor":217,"locationsCount":219},"100413408","phase-2-a-study-of-the-safety-and-activity-of-sparsentan-for-the-treatment-of-patients-with-immunoglobulin-a-nephropathy-100413408","NCT04663204","A Study of the Safety and Activity of Sparsentan for the Treatment of Patients With Immunoglobulin A Nephropathy","A Multi-centre, Open-label, Exploratory Trial of the Safety and Activity of Sparsentan for the Treatment of Incident (Cohort A) and Recurrent (Cohort B) Patients With Immunoglobulin A Nephropathy","SPARTAN","For Cohort A (Patients with Incident IgAN)\n\nInclusion Criteria:\n\n* The patient is willing and able to provide signed informed consent.\n* The patient can understand written and spoken English.\n* The patient is male or female, aged ≥18 years.\n* The patient has been diagnosed with biopsy-proven IgAN within the last 6 months (calculated from the date of kidney biopsy, upon which the IgAN-positive diagnosis was made, to the signing of the informed consent form).\n* The patient has a urine total protein value ≥0.5 g\u002Fday at screening.\n* The patient has an eGFR value ≥30 mL\u002Fmin\u002F1.73 m2 at screening.\n* The patient has not previously been treated with ACEI and\u002For ARB therapy for IgAN OR has not received ACEI and\u002For ARB therapy within the last 12 months.\n* The patient has a systolic BP ≤150 mmHg and ≥100 mmHg, and diastolic blood pressure ≤100 mmHg and ≥60 mmHg at screening.\n* Women of childbearing potential (WOCBP), beginning at menarche, must agree to the use of one highly reliable (ie, can achieve a failure rate of \\\u003C1% per year) method of contraception from 7 days prior to the first dose of trial medication until 90 days after the last dose of trial medication. Highly reliable contraception methods include stable oral, implanted, transdermal, or injected contraceptive hormones associated with inhibition of ovulation, or an intrauterine device (IUD) in place for at least 3 months. One additional barrier method must also be used during sexual activity, such as a diaphragm or diaphragm with spermicide (preferred), or male partner's use of male condom or male condom with spermicide), from Day 1 until 90 days after the last dose of trial medication.\n\nWOCBP are defined as those who are fertile, following menarche and until becoming postmenopausal unless permanently sterile; permanent sterilisation methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy. A postmenopausal state is defined as amenorrhoea for more than 24 consecutive months without an alternative medical cause; women on hormone replacement therapy must have a documented plasma follicle-stimulating hormone level ≥40 mIU\u002FmL. All WOCBP must have a negative pregnancy test at Visit 1 (serum test) and Visit 2 (urine, with positive results confirmed by serum).\n\nExclusion Criteria:\n\n* The patient has IgAN secondary to another condition (eg, systemic lupus erythematosus, liver cirrhosis).\n* The patient, in the opinion of the Investigator, has a rapidly progressive glomerulonephritis (rapid decline in GFR and crescents on biopsy).\n* The patient has a history of type 1 diabetes mellitus, uncontrolled type 2 diabetes mellitus (haemoglobin A1c \\[HbA1c\\] \\>8%), or nonfasting blood glucose \\>10 mmol\u002FL (180 mg\u002FdL) at screening.\n* The patient has undergone any organ transplantation, with the exception of corneal transplants.\n* The patient requires any of the prohibited concomitant medications (see Section 14.4).\n* The patient has been taking any systemic immunosuppressive medications for \\>2 weeks within 6 months prior to screening.\n* The patient has a documented history of heart failure (New York Heart Association Class II-IV) and\u002For previous hospitalisation for heart failure or unexplained dyspnoea, orthopnoea, paroxysmal nocturnal dyspnoea, ascites, and\u002For peripheral oedema.\n* The patient has clinically significant cerebrovascular disease (transient ischemic attack or stroke) and\u002For coronary artery disease (hospitalisation for myocardial infarction or unstable angina, new onset of angina with positive functional tests, coronary angiogram revealing stenosis, or a coronary revascularisation procedure) within 6 months prior to screening.\n* The patient has jaundice, hepatitis, or known hepatobiliary disease (including asymptomatic cholelithiasis), or alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>2 times the upper limit of the normal range at screening.\n* The patient has a history of malignancy other than adequately treated basal cell or squamous cell skin cancer or cervical carcinoma within the past 2 years.\n* The patient has a screening haematocrit value \\\u003C27% or haemoglobin value \\\u003C90 g\u002FL (9 g\u002FdL).\n* The patient has a screening potassium value of \\>5.5 mmol\u002FL (5.5 mEq\u002FL).\n* The patient has a history of alcohol or illicit drug use disorder (as defined in the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition).\n* The patient has a history of serious side effects or allergic response to any AngII or ERA, including sparsentan, or has a hypersensitivity to any of the excipients in the IMP.\n* The female patient is pregnant, plans to become pregnant during the course of the trial, or is breastfeeding.\n* The patient has participated in a trial of any investigational product within 28 days prior to screening, or plans to participate in such a trial during the course of this trial.\n* The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the trial, including the ability to swallow the IMP whole.\n* The patient, in the opinion of the Investigator, has a medical condition or abnormal clinically significant laboratory screening value not listed above that may interfere with the evaluation of sparsentan safety or activity.\n* Patients with a medical condition or abnormal clinically significant laboratory screening value not listed above that may interfere with the evaluation of sparsentan safety or activity will be reviewed before consideration of the patient for enrolment.\n\nFor Cohort B (Recurrent IgAN following kidney transplantation)\n\nInclusion Criteria:\n\n* Male and female aged ≥18 years\n* Diagnosis of recurrent IgAN based on histological analysis of a transplanted kidney biopsied within the last 6 months\n* A time period of \\>12 months since kidney transplantation\n* UPCR ≥50 mg\u002Fmmol (≥0.44 g\u002Fg) and eGFR value ≥25 mL\u002Fmin\u002F1.73 m2\n* For patients on an ACEI and\u002For ARB, and\u002For SGLT2 inhibitor, the dosing regimen is stable for at least 6 weeks prior to and during the screening period\n* Tacrolimus treatment as part of standard of care immunosuppression following kidney transplantation\n* Systolic BP ≤150 mmHg and ≥100 mmHg, and diastolic blood pressure ≤100 mmHg and ≥60 mmHg at screening.\n* Female patients not of childbearing potential, or of childbearing potential and agreeing to use the contraceptive methods listed in Section 5.1\n\nExclusion Criteria:\n\n* The patient has recurrent IgAN secondary to another condition or cause (eg, systemic lupus erythematosus, liver cirrhosis).\n* Evidence of alternative pathology on the kidney transplant biopsy as the main cause for proteinuria (e.g. diabetic nephropathy, chronic transplant glomerulopathy, mTORi treatment)\n* Patient has multiorgan transplants (with the exception of corneal transplants)\n* Immunosuppressive therapy (IST) regimen for kidney transplant or other chronic immunosuppressive therapies that is not stable for \\>6 weeks prior to Day 1. Exceptions include routine protocol tapering and for tacrolimus, changes in dose to meet target level\n* Treatment with enteric budesonide (nefecon) within 6 months prior to screening, or planned use of enteric budesonide (nefecon) at any time during the study.\n* Current treatment for surgical complications\n* \\\u003C3 months after anti-rejection treatment or active rejection\n* Active bacterial, fungal or viral infection and\u002For active treatment of infection including BKV, CMV, HIV, Hepatitis B and C \\\u003C3 months prior to and during the screening period\n* Current treatment for surgical complications\n* Uncontrolled diabetes mellitus (defined by HbA1C \\>8% (\\>64 mmol\u002Fmol)\n* History of heart failure (New York Heart Association (NYHA) Class II-IV)\n* Jaundice, hepatitis, or known hepatobiliary disease\n* Malignancy within the past 2 years with the exception of adequately treated basal cell carcinoma or non-metastatic squamous cell carcinoma of the skin, with no evidence or recurrence\n* Haematocrit \\\u003C27%, haemoglobin \\\u003C90 g\u002FL (9 g\u002FdL), or potassium \\>5.5 mmol\u002FL (5.5 mEq\u002FL)\n* History of alcohol or illicit drug use disorder (as defined in the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition)\n* History of serious side effects or allergic response to any angiotensin II antagonist or endothelin receptor antagonist (ERA) or dual endothelin and angiotensin receptor antagonist (DEARA e.g. sparsentan)\n* The female patient is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.\n* The patient has participated in a study of any investigational product within 28 days prior to screening, or plans to participate in such a study during the course of this study.\n* The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study, including the ability to swallow the IMP whole.\n* The patient, in the opinion of the Investigator, has a medical condition or abnormal clinically significant laboratory screening value not listed above that may interfere with the evaluation of sparsentan safety or activity.",{"count":204,"type":21},24,[24],"To determine the nephroprotective potential of treatment with sparsentan in (1: Cohort A) patients newly-diagnosed with immunoglobulin A nephropathy (IgAN) (ie, incident patients) who have not received prior angiotensin-converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) therapy, and in (2: Cohort B) patients with recurrent IgAN following kidney transplantation.",[208,209,210,211,31,32],"Immunoglobulin A Nephropathy","Kidney Diseases","Glomerulonephritis, IGA","Glomerulonephritis",{"date":161,"type":40},{"date":214,"type":40},"2020-12-10",{"date":216,"type":21},"2027-12-31",{"name":218,"class":47},"University of Leicester",6,{"id":221,"slug":222,"hasResults":11,"nctId":223,"briefTitle":224,"officialTitle":225,"acronym":4,"eligibilityCriteria":226,"healthyVolunteers":11,"sex":16,"minAge":87,"maxAge":18,"enrollmentInfo":227,"targetDuration":4,"studyType":22,"phases":229,"briefSummary":230,"conditions":231,"keywords":232,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":233,"lastUpdatePostDateStruct":234,"startDateStruct":236,"completionDateStruct":238,"leadSponsor":240,"locationsCount":137},"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.",{"count":228,"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",[65,96,32,95,94],[67,98,70,68,69],"2026-05-01",{"date":235,"type":40},"2026-05-04",{"date":237,"type":40},"2020-09-22",{"date":239,"type":21},"2027-11-30",{"name":79,"class":80},{"id":242,"slug":243,"hasResults":11,"nctId":244,"briefTitle":245,"officialTitle":246,"acronym":247,"eligibilityCriteria":248,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":249,"targetDuration":251,"studyType":116,"phases":4,"briefSummary":252,"conditions":253,"keywords":267,"overallStatus":269,"whyStopped":4,"lastUpdateSubmitDate":270,"lastUpdatePostDateStruct":271,"startDateStruct":273,"completionDateStruct":275,"leadSponsor":277,"locationsCount":137},"100513772","clonal-hematopoiesis-of-immunological-significance-100513772","NCT05969821","Clonal Hematopoiesis of Immunological Significance","Immuno-inflammatory Manifestations With or Without Clonal Hematopoiesis: Ambispective Cohort Study","CHIS","Inclusion Criteria:\n\n* Age \\>=18 years old;\n* Confirmed dysimmune manifestations: clinical or biological abnormality or systemic disease;\n* Presence or absence of myeloid or lymphoid blood disease according to World Health Organization (WHO) classification\n\nExclusion Criteria:\n\n* Persons benefiting from special protection: adults under guardianship and curatorship;\n* People hospitalized without their consent and not protected by law; persons deprived of liberty;\n* Persons not affiliated to the social security system",{"count":250,"type":21},5000,"10 Years","Ambispective, national, multicenter observational cohort study aimed at characterizing the satellite dysimmune manifestations of clonal hematopoiesis, including Vexas (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome.",[32,31,254,255,256,257,258,259,260,261,262,263,65,264,265,266],"Inflammation","Autoinflammatory Diseases","Vexas Syndrome","Hematopoiesis Clonal","Clonal Hematopoiesis of Indeterminate Potential","Hematologic Diseases","Myelodysplastic-Myeloproliferative Diseases","Leukemia Myelomonocytic Chronic","Myelodysplastic Syndromes","Myeloproliferative Disorders","Lymphoma","Leukemia","Monoclonal Gammopathy of Undetermined Significance",[254,255,268,31,258,260,65,266],"Vexas syndrome","NOT_YET_RECRUITING","2026-03-19",{"date":272,"type":40},"2026-03-23",{"date":274,"type":21},"2026-04",{"date":276,"type":21},"2045-09",{"name":278,"class":47},"Assistance Publique - Hôpitaux de Paris",{"id":280,"slug":281,"hasResults":11,"nctId":282,"briefTitle":283,"officialTitle":284,"acronym":4,"eligibilityCriteria":285,"healthyVolunteers":11,"sex":16,"minAge":286,"maxAge":287,"enrollmentInfo":288,"targetDuration":4,"studyType":22,"phases":289,"briefSummary":291,"conditions":292,"keywords":307,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":313,"lastUpdatePostDateStruct":314,"startDateStruct":316,"completionDateStruct":318,"leadSponsor":320,"locationsCount":137},"100560306","phase-1-a-study-to-investigate-safety-and-effectiveness-of-porcine-pancreatic-cells-opf-310-in-patients-with-type-1-diabetes-mellitus-100560306","NCT06575426","A Study to Investigate Safety and Effectiveness of Porcine Pancreatic Cells (OPF-310) in Patients With Type 1 Diabetes Mellitus","A Phase I\u002FIIa, Single Site, Open-Label, Ascending Dose Study to Evaluate the Safety and Efficacy of OPF-310 [Encapsulated Porcine Islet Cells for Xenotransplantation] in Subjects With Type 1 Diabetes Mellitus","Inclusion Criteria:\n\n1. Subject must be aged 35 to 65 years of age inclusive, at the time of signing the informed consent.\n2. Subject has an established diagnosis of type 1 diabetes mellitus (T1DM)(in accordance with the American Diabetes Association's criteria), with a minimum duration since diagnosis of 5 years.\n3. If one of the following criteria (either a or b) applies:\n\n   1. Subject has unstable T1DM, not achieving adequate control after receiving CLS (CGM:Dexcom G6, insulin pump: Omnipod® 5 or t:slim X2) under care of a qualified diabetes team for at least 6 months prior to enrollment.\n   2. Subject has unstable T1DM, not achieving adequate control after receiving CLS (CGM:Dexcom G7, insulin pump: Omnipod® 5, t:slim X2, iLet Bionic Pancreas or The Tandem Mobi System) under care of a qualified diabetes team for at least 6 months prior to enrollment.\n4. If one of the following criteria (either a, b or c) applies:\n\n   1. Subject has had a Level 3 (severe) hypoglycemic episode (defined as having cognitive impairment requiring external assistance for recovery) at least three times within the 1 year prior to enrollment recorded in the medical record or patient log.\n   2. Subject has had a Level 3 (severe) hypoglycemic episode at least once within the 1 year prior to enrollment and demonstrates a Clarke Score ≥4, assessed by trained study personnel. The SHE(s) and Clarke Score must be recorded in the medical record or patient log.\n   3. Subject has had TBR \\>1% at glucose levels below 70mg\u002FdL and demonstrates a Clarke Score≥4, assessed by trained study personnel. TBR data used for screening and Clarke score must be recorded in the medical record or patient log.\n5. Subject has C-peptide \\\u003C0.3 ng\u002FmL following a mixed meal tolerance test or undetectable fasting C-peptide.\n6. Hemoglobin A1C (HbA1c) ≤ 9.0\n7. Contraceptive use must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies\n8. Subject who can agree to cooperate with lifetime follow-up after transplantation.\n9. Subject is capable of providing signed informed consent\n\nExclusion Criteria:\n\n1. Previous history of insulin resistance (defined as an average insulin dose requirement ≥ 0.8 unit\u002Fkg\u002Fday for 1 week prior to enrollment).\n2. Subject has latent autoimmune diabetes in adults (LADA), ketosis-prone (Flatbush) diabetes, or maturity onset diabetes of the young (MODY).\n3. CRP ≥ 10 mg\u002FL.\n4. Clinically unstable thyroid disease (thyroid stimulating hormone (TSH)\\\u003C the lower limit of the normal range of TSH at the site.) Patients with subclinical hyperthyroidism can be rescreened once TSH levels normalize due to treatment or other factors. In addition, patients with transiently abnormal TSH levels may undergo rescreening only once during the screening period.\n5. History of malignancies within the past 5 years, excluding basal and squamous cell carcinoma\n6. Positive serologies or nucleic acid testing for human immunodeficiency virus (HIV), hepatitis C, and hepatitis B.\n7. Active or untreated proliferative diabetic retinopathy. Subjects may be rescreened once they are successfully treated.\n8. Serious comorbid conditions that are likely to affect participation in the study, including:\n\n   1. Within the last 12 months, peripheral vascular disease with previous amputation.\n   2. History of New York Heart Association (NYHA) class II, III or IV congestive heart failure (CHF) and\u002For chronic atrial fibrillation.\n   3. Chronic obstructive pulmonary disease (COPD) or asthma with previous hospitalization for decompensation; a requirement for mechanical ventilation at any stage; or long- term treatment with oral corticosteroids.\n   4. Macroalbuminuria (\\> 300 mg albumin\u002Fgm creatinine).\n   5. Estimated glomerular filtration rate (eGFR) cut-off of \\\u003C 30 ml\u002Fmin for all per Kidney Disease Improving Global Outcomes (KDOQI) and Kidney Disease Outcomes Quality Initiative (KDIGO) consensus.\n9. Use of warfarin or other anticoagulant therapy (except aspirin), or prothrombin time and international normalized ratio (PT-INR) \\> 1.5\n10. Adrenal insufficiency being treated with corticosteroids\n11. Previous pan-peritonitis\n12. Previous cardiovascular or cerebrovascular disease. NOTE: For the purposes of this exclusion criterion, \"previous cardiovascular disease\" is defined as the presence of co-existing cardiac disease, characterized by any of the following conditions:\n\n    1. Recent myocardial infarction (within past one year), or\n    2. Angiographic evidence of non-correctable coronary artery disease, or\n    3. Evidence of ischemia on functional cardiac exam (with a stress echo test recommended for subjects with a history of ischemic disease), or\n    4. Heart failure \\> NYHAII\n13. Patients with hematopoietic stem cell abnormalities (e.g., aplastic anemia, myelodysplastic syndrome)\n14. Patients who received a blood transfusion in the previous 90 days, are anticipated to undergo surgery during the 1-year study period that may require transfusion, or have donated blood within the previous 90 days.\n15. Previous receipt of an organ, skin allograft, or other tissue transplant from an allogeneic human or animal donor.\n16. Treatment with immunosuppressive medication.\n17. Previous abdominal surgery, excluding uncomplicated appendectomy, cholecystectomy, exploratory laparoscopy and hernia repair performed prior to 12 weeks prior to enrollment.\n18. Treatment with any hypoglycemic medication prescribed for glycemic control, other than insulin therapy.\n19. Treatment with acetaminophen or hydroxycarbamide.\n20. Use of any investigational products within 12 weeks of enrollment (before entering run-in) or 5 half-lives of the investigational product, whichever is greater.\n21. Subject has history of allergy to antibiotics (Amphotericin B, Cefazolin, Ciprofloxacin, Gentamicin), which are used during manufacture of OPF-310.\n22. Previous history of insulin allergy (including porcine insulin), pork product allergy or alginate\u002Fseaweed allergy.\n23. Panel reactive antibodies (PRA) \\> 80 %.\n24. Active drug, substance or alcohol addiction.\n25. Body mass index (BMI) \\>27 kg\u002Fm2.\n26. Any other condition that, in the opinion of the Investigator, may interfere with adherence to the study protocol, including dementia, psychiatric disorder, medical condition, or a history of non-adherence to appointments or treatments","35 Years","65 Years",{"count":194,"type":21},[290,24],"PHASE1","This study is First In Human study for Encapsulated Porcine Islet Cells for Xenotransplantation (OPF-310). The purpose of this study to assess the safety, tolerability, and efficacy of OPF-310 transplantation and to define the recommended Phase 2 dose (RP2D) in adult subjects with unstable Type 1 Diabetes Mellitus (T1DM) and a level 3 (severe) hypoglycemic episode at least three times within the 1 year prior to enrollment despite treatment with a closed loop system (CLS) for at least 6 months.",[293,294,295,296,297,298,299,300,27,301,302,303,304,305,32,31,306],"Diabetes Mellitus, Type 1","Hypoglycemia","Islet Cell Transplantation","Type 1 Diabetes","Type 1 Diabetes Mellitus","T1D","T1DM","T1DM - Type 1 Diabetes Mellitus","Severe Hypoglycemia","Xenotransplantation","Hypoglycemic Episode","Islet Transplantation in Diabetes Mellitus Type 1","Glucose Metabolism Disorders (Including Diabetes Mellitus)","Metabolic Disease",[308,309,299,294,310,311,302,312,297,296],"Diabetes Mellitus","Diabetes Mellitus, Type1","islet cell transplantation","pig islet cell transplantation","Porcine islet cell transplantation","2026-02-26",{"date":315,"type":40},"2026-03-02",{"date":317,"type":40},"2025-06-10",{"date":319,"type":21},"2027-06-30",{"name":321,"class":322},"Otsuka Pharmaceutical Factory, Inc.","INDUSTRY",{"id":324,"slug":325,"hasResults":11,"nctId":326,"briefTitle":327,"officialTitle":328,"acronym":4,"eligibilityCriteria":329,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":330,"targetDuration":4,"studyType":22,"phases":332,"briefSummary":334,"conditions":335,"keywords":336,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":342,"lastUpdatePostDateStruct":343,"startDateStruct":345,"completionDateStruct":347,"leadSponsor":349,"locationsCount":351},"100521437","implementation-of-support-in-the-care-of-adults-living-with-type-1-diabetes-100521437","NCT06069583","Implementation of Support in the Care of Adults Living With Type 1 Diabetes","Using the Support Online Platform for Self-management Education of Adults Living With Type 1 Diabetes as Part of Usual Care or Independently: an Implementation Study","Inclusion Criteria:\n\n* Diagnosis of T1D (including LADA - latent autoimmune diabetes in adults)\n* Have access to Internet\n* Use of an active email address\n* Comprehension of English or French\n* Live in Canada\n\nExclusion Criteria:\n\n\\- Unable to use the Support platform",{"count":331,"type":21},322,[333],"NA","The investigators will conduct a trial to evaluate if an online training and support platform can help adults living with type 1 diabetes (T1D) in their diabetes self-management. Investigators will compare a group that has access to the \"Support\" platform through their usual medical care to a group that accesses the platform independently. The first group will be recruited through four participating clinics in the province of Quebec (Canada). The second group will be composed of adults living with T1D across Canada. Participants will have access to the platform for 12 months and will be asked to complete online questionnaires at the beginning and after 6 and 12 months, and share their glucose reader data with the research team. A subgroup of participants as well as healthcare professionals from the four clinics will be invited to participate in an individual interview aiming to understand the barriers and facilitators of integration \"Support\" in clinical care.",[296,306,30,31,32],[337,338,339,340,341],"self-management education","Hemaglobin A1c","Online education","adults","type 1 diabetes","2025-09-22",{"date":344,"type":40},"2025-09-25",{"date":346,"type":40},"2024-05-05",{"date":348,"type":21},"2027-10",{"name":350,"class":47},"McGill University",4,{"id":353,"slug":354,"hasResults":11,"nctId":355,"briefTitle":356,"officialTitle":357,"acronym":358,"eligibilityCriteria":359,"healthyVolunteers":11,"sex":16,"minAge":360,"maxAge":361,"enrollmentInfo":362,"targetDuration":4,"studyType":22,"phases":364,"briefSummary":365,"conditions":366,"keywords":367,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":342,"lastUpdatePostDateStruct":374,"startDateStruct":375,"completionDateStruct":377,"leadSponsor":378,"locationsCount":351},"100509239","support-t-online-training-in-youth-living-with-type-1-diabetes-transitioning-to-adult-care-100509239","NCT05910840","Support-t Online Training in Youth Living With Type 1 Diabetes Transitioning to Adult Care","Support-t, an Online Training and Peer Support Platform to Accompany Youth Living With Type 1 Diabetes Transitioning to Adult Healthcare","Support-t","Inclusion Criteria:\n\n* Adolescents with a clinical diagnosis of T1D\n* 14-16 years of age\n* Receiving diabetes care at one of 4 university teaching hospital-based pediatric diabetes clinics in Quebec: Montreal Children's Hospital-McGill University Health Centre, Centre hospitalier universitaire de Sherbrooke, Le Copain-Hôpital de Gatineau, Centre Hospitalier Universitaire de Québec\n* Having access to internet\n* Having an active email address\n* Fluent in English or French\n\nExclusion Criteria:\n\n* Severe neurocognitive disabilities\n* Patients with conditions associated with shortened erythrocyte survival, such as hemolytic anemia or other conditions associated with inaccurate HbA1c","14 Years","16 Years",{"count":363,"type":21},200,[333],"The investigators will conduct a randomized controlled trial (RCT) to examine how an online training and peer support platform could help the preparation to transition to adult care. Among 14-16 year old youth with Type 1 Diabetes (T1D), the investigators aim to assess the effect of an online training and peer support platform (Support-t) integrated in usual care, compared with usual care on Hemoglobin A1c (HbA1c), adverse outcomes and psychosocial measures during the preparation for transition to adult care. The investigators will conduct a multi-site, parallel group, blinded (outcome assessors, data analysts), superiority RCT of adolescents with T1D (14-16 years of age) followed at one of 4 university teaching hospital-based pediatric diabetes clinics in the province of Quebec.",[308,293,305,306,30,31,32],[368,369,370,371,338,372,373],"Transition Care","Pediatric","Adolescent","Education","Self-management","Randomized Controlled Trial",{"date":344,"type":40},{"date":376,"type":40},"2024-10-07",{"date":348,"type":21},{"name":379,"class":47},"Anne-Sophie Brazeau",{"id":381,"slug":382,"hasResults":11,"nctId":383,"briefTitle":384,"officialTitle":385,"acronym":386,"eligibilityCriteria":387,"healthyVolunteers":11,"sex":16,"minAge":57,"maxAge":4,"enrollmentInfo":388,"targetDuration":389,"studyType":116,"phases":4,"briefSummary":390,"conditions":391,"keywords":397,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":404,"lastUpdatePostDateStruct":405,"startDateStruct":407,"completionDateStruct":409,"leadSponsor":411,"locationsCount":413},"100543224","abrocitinib-taiwan-treatment-pattern-and-real-world-study-in-atopic-dermatitis-attract-registry-100543224","NCT06353087","Abrocitinib Taiwan Treatment Pattern and Real World Study in ATopiC Dermatitis (ATTRACT Registry)","The Real-world Treatment Patterns and Clinical Outcomes in Moderate-to-severe Atopic Dermatitis (AD) Patients Receiving Abrocitinib","ATTRACT","Inclusion Criteria:\n\nPatients must meet all of the following inclusion criteria to be eligible for inclusion in the study:\n\n1. Patients aged ≥12 years\n2. Patients with confirmed diagnosis of moderate-to-severe AD as assessed by the physician\n3. Patients for whom the physician's decision has been made to newly prescribe abrocitinib in usual clinical practice conditions\n4. Evidence of a personally signed and dated informed consent\u002Fassent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study\n\nExclusion Criteria:\n\nPatients meeting any of the following criteria will not be included in the study:\n\n1. Any prior use of abrocitinib\n2. Simultaneous participation in a study that includes administration of any investigational drug or procedure",{"count":363,"type":21},"12 Months","This study is to describe the real-world treatment patterns and clinical outcomes in moderate-to-severe AD patients receiving abrocitinib over a 12-month observation period, and to describe patient demographic and baseline characteristics.",[392,393,394,395,32,396],"Dermatitis, Atopic","Dermatitis","Eczema","Skin Diseases","Janus Kinase Inhibitors",[398,399,400,401,402,403],"Atopic Dermatitis","Abrocitinib","JAK inhibitors","Real world","Registry","Taiwan","2025-09-02",{"date":406,"type":40},"2025-09-03",{"date":408,"type":40},"2024-07-01",{"date":410,"type":21},"2026-05-15",{"name":412,"class":322},"Pfizer",8,{"id":415,"slug":416,"hasResults":11,"nctId":417,"briefTitle":418,"officialTitle":419,"acronym":4,"eligibilityCriteria":420,"healthyVolunteers":56,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":421,"targetDuration":4,"studyType":116,"phases":4,"briefSummary":423,"conditions":424,"keywords":442,"overallStatus":269,"whyStopped":4,"lastUpdateSubmitDate":448,"lastUpdatePostDateStruct":449,"startDateStruct":451,"completionDateStruct":453,"leadSponsor":455,"locationsCount":4},"100557135","digital-diagnostics-and-intervention-services-for-parkinsons-disease-100557135","NCT06534177","Digital Diagnostics and Intervention Services for Parkinson's Disease","Development of Digital Diagnostics and Intervention Services for Parkinson's Disease","Inclusion Criteria:\n\n* Diagnosis of idiopathic Parkinson's disease (UK Brain Bank Criteria) or other appropriate condition specific scale \\[stroke, multiple sclerosis, arthritis or osteoporosis\\]\n* Able to self-report history of daily gait freezing and\u002For festination for people with PD or gait and\u002For transfers affected by condition\n* Able to walk unsupported or using an aid for at least 5 minutes and satisfactory completion of the Canadian PARQ and if over 69 used to carrying out this level of exercise\n* Adult (+18 years old)\n* Normal or corrected-to-normal vision (Snellen Visual Acuity \\> 12\u002F18) or safe to mobilise with support\n* Montreal Cognitive assessment score \\>21 or ability to follow 2 stage commands Healthy participants \\[Phase 1,2,3\\]\n* With no long-term conditions affecting movement\n* Able to walk unsupported or using an aid for at least 3 minutes and satisfactory completion of the Canadian PARQ and\n* if over 69 used to carrying out this level of exercise\n* Adult (+18 years old)\n* Normal or corrected-to-normal vision (Snellen Visual Acuity \\> 12\u002F18) or safe to mobilise with support\n* Montreal Cognitive assessment score \\>21 or ability to follow 2 stage commands\n\nExclusion Criteria:\n\n* Participants with long-term conditions affecting movement\n* Any physical or mental condition affecting ability to safely participate in this level of activity and capacity to understand\n* testing as demonstrated by ability to safely follow commands and pass the PARQ by the research team.\n* Cognitive impairment affecting ability to safely participate and follow instructions\n* Any injury or disorder that may affect balance (other than Parkinson's or referring primary condition)\n* Any skin conditions or broken skin in the calf and behind knee area\n* Deep brain stimulation or pacemaker implants or other implant that may interfere with the measurement system Healthy participants\n* Any physical or mental condition affecting ability to safely participate in this level of activity and capacity to understand\n* testing as demonstrated by ability to safely follow commands and pass the PARQ by the research team.\n* Cognitive impairment affecting ability to safely participate and follow instructions\n* Any injury or disorder that may affect balance (other than Parkinson's or referring primary condition)\n* Any skin conditions or broken skin in the calf and behind knee area\n* Deep brain stimulation or pacemaker implants or other implants that may interfere with the measurement system",{"count":422,"type":21},80,"People with Parkinson's have infrequent clinical consultation (once every 12-18 months) and limited rehabilitation.\n\nAssessment play an important role in these consultations to help clinicians understand patients' health status and disease progression necessary to adjust treatment plans. The current way of measuring is the UPDRS which needs a clinician to do this and takes 30 minutes. There is a strong need for more frequent and accurate Parkinson's assessments in the clinic and at home to detect changes early and then give appropriate support and drug and physiotherapy quickly. There is a need to develop good home digital physiotherapy tools to increase the amount of therapy. Here the investigators are testing new digital technologies to do these assessments in the home and clinic and a new digital physiotherapy device in the home. The investigators aim to conduct a clinical study with 50 people with Parkinson's (50 from UK) with the UPDRS, (a rating scale that is commonly used in clinical settings to evaluate the progression of Parkinson's disease) and 30 healthy adults. The investigators will develop and investigate if two new digital devices, one the MachineMD that measures eye movement and one the gaitQ that measures gait can be used instead of the MDS-UPDRS (motor) using digital gait and ophthalmic features in the clinic setting. The investigators will investigate the effect of a physiotherapy gait intervention gaitQ Tempo in the home context for two weeks and of doing the gait measure at home. The investigators will determine the potential of the gaitQ intervention to improve key gait metrics in order to collect clinical evidence and of using the gaitQ as a cuing system over a 2-week period on gait and other movement measures in the home and community",[425,426,427,428,429,430,431,432,433,434,435,436,31,32,437,438,439,440,441],"Brain Diseases","Central Nervous System Diseases","Nervous System Diseases","Joint Diseases","Musculoskeletal Diseases","Parkinson Disease","Basal Ganglia Diseases","Movement Disorders","Synucleinopathies","Neuro-Degenerative Disease","Demyelinating Disease, Autoimmune, CNS","Demyelinating Disease","Bone Diseases, Metabolic","Bone Diseases","Arthritis","Osteoporosis","Multiple Sclerosis",[443,444,445,446,447],"parkinson's disease","osteoarthritis","stroke","MS","osteoporosis","2024-08-06",{"date":450,"type":40},"2024-08-09",{"date":452,"type":21},"2024-10-15",{"date":454,"type":21},"2026-03-01",{"name":456,"class":47},"University of Exeter",{"id":458,"slug":459,"hasResults":11,"nctId":460,"briefTitle":461,"officialTitle":462,"acronym":4,"eligibilityCriteria":463,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":88,"enrollmentInfo":464,"targetDuration":4,"studyType":22,"phases":466,"briefSummary":467,"conditions":468,"keywords":480,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":482,"lastUpdatePostDateStruct":483,"startDateStruct":485,"completionDateStruct":487,"leadSponsor":489,"locationsCount":137},"100536932","phase-1-a-study-to-evaluate-the-safety-pkpd-of-oricar-017-in-subjects-with-rrmm---rigel-study-100536932","NCT06271252","A Study to Evaluate the Safety, PK\u002FPD of (OriCAR-017) in Subjects With RR\u002FMM - RIGEL Study","A Phase I\u002FII, Open-label, Multicenter Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of Anti-GPRC5D CAR-T Cell Product (OriCAR-017) in Subjects With Relapsed\u002FRefractory Multiple Myeloma.","Inclusion Criteria:\n\nCapable of giving signed informed consent\n\nSubjects aged 18 to 75 years (inclusive) at Screening (signing the ICF).\n\nExpected survival period is \\>12 weeks.\n\nDiagnosis of MM according to the IMWG criteria (2016 version).\n\nOne of the following criteria must be met:\n\nIf immunoglobulin (Ig)G type MM, then serum M protein \\>10 g\u002FL; if IgA, IgD, IgE or IgM type MM, then serum M protein \\>5 g\u002FL\n\nUrine M protein level \\>200 mg\u002F24 hour\n\nIf light chain type MM, then serum free light chain (sFLC) \\>100 mg\u002FL and K\u002Fλ FLC ratio is abnormal.\n\nExtramedullary lesions (\\>1 cm for diameter of the short axis).\n\nFor Phase I (dose-escalation) - Subjects who had received at least 3 prior lines of therapy, had previous exposure to BCMA-Ag+ therapies, and were refractory to the last line of therapy.\n\nFor Phase I (dose-expansion) and Phase II: Subjects with previous exposure to BCMA directed therapies including BCMA bispecific antibody (e.g., teclistamab), BCMA antibody directed conjugate (such as BLENREP), and BCMA-CAR-T (such as CARVYKT1TM)\n\nSubjects with adequate hematologic, renal, hepatic, pulmonary and cardiac function.\n\nSubject and partners willing to take and or use effective contraceptive measures until 2 years post IMP infusion.\n\nExclusion Criteria:\n\nPregnant or breastfeeding.\n\nSeropositive for history of human immunodeficiency virus Active Hepatitis B infection and or Hepatitis C infection\n\nKnown active or prior history of CNS involvement\n\nHistory of autoimmune diseases (such as Crohn's disease, rheumatoid arthritis, systemic lupus erythematosus) caused damage to terminal organs or required systemic application of immunosuppressive or other drugs in the past 2 years\n\nPresence of uncontrolled active infection\n\nSubjects who received autologous hematopoietic stem cell transplantation (ASCT) within 8 weeks of Screening Visit or who plan to undergo ASCT during the study.\n\nSubjects who received allogeneic stem cell therapy.\n\nAny condition that in the opinion of the Investigator, would interfere with evaluation of the IMP.\n\nReceived Bendamustine treatment 1 year prior to Screening Visit.",{"count":465,"type":21},81,[290],"The is a first clinical study for Oricell Therapeutics Inc. in the United States to evaluate the safety, PK, PD and preliminary efficacy of our anti-GPRC5D cell product (OriCAR-017) in subjects with relapsed\u002Frefractory multiple myeloma.\n\nRIGEL Study",[469,470,471,472,473,474,475,476,259,477,65,478,32,479],"Neoplasms, Plasma Cell","Neoplasms by Histologic Type","Neoplasms","Hemostatic Disorders","Vascular Diseases","Cardiovascular Diseases","Paraproteinemias","Blood Protein Disorders","Hemorrhagic Disorders","Immunoproliferative Disorders","Multiple Myeloma",[481],"R\u002FR MM, CAR-T","2024-08-01",{"date":484,"type":40},"2024-08-02",{"date":486,"type":40},"2024-04-03",{"date":488,"type":21},"2028-04-12",{"name":490,"class":322},"OriCell Therapeutics Co., Ltd."]