[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"non-malignant-disorders\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:non-malignant-disorders":33},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,47,84,108],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100641447","phase-2-naive-t-cell-deplete-grafts-for-gvhd-prevention-in-non-malignant-diseases-100641447",false,"NCT07660783","Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases","A Phase II Prospective Study Evaluating Selective Depletion of CD45RA+ (Naïve) T Cells (TND) From Peripheral Blood Stem Cell (PBSC) Grafts for Prevention of Graft Versus Host Disease (GVHD) in Non-Malignant Diseases (NMDs)","Inclusion Criteria:\n\n* Considered appropriate candidate for allogeneic HCT following low dose (4Gy) TBI containing-conditioning and have one of the following diagnoses: A) BMF B)Hemoglobinopathies C)PID D) Autoimmune cytopenias E) Immune dysregulation F) HLH G) Other NMD treatable by HCT and NMD that is not clearly defined (a patient with a NMD for whom genetic testing has been done and a genetic mutation responsible for their NMD phenotype has not been identified) are eligible for the study following discussion with and approval by the protocol PI\n* Patients aged 6 months- 5 years old (inclusive) at the time of informed consent\n* Patient with suitable HCT donor (see inclusion criteria below)\n* Recipient informed consent\u002Fassent (13 years and older), and\u002For legal guardian permission must be obtained\n\nExclusion Criteria:\n\n* Patient with aplastic anemia\n* Patients with severe combined immunodeficiency (SCID)\n* Fanconi anemia\n* Dyskeratosis congenita\n* Patient weight \\> 100 kg\n* Patients who are positive for HIV-1, HIV-2\n* Patients with current neoplastic disorders\n* Patients with uncontrolled infections for whom HCT is considered contraindicated by the consulting infectious disease physician.\n* Patients with organ dysfunction including A) Renal insufficiency B) Impaired cardiac function C)Impaired pulmonary function D) Liver dysfunction\n* Patients who are pregnant or breast-feeding\n* Patients on other experimental protocols for prevention of GVHD\n* Patients of childbearing age who are presumed to be fertile and are unwilling to use an effective birth control method or refrain from sexual intercourse during and for 12 months post-HCT\n* Patients with any other significant medical conditions that would make them unsuitable for transplantation, as determined by the PI\n* Patients with a known hypersensitivity to tacrolimus or MMF","ALL","6 Months","50 Years",{"count":20,"type":21},40,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant diseases requiring hematopoietic cell transplantation. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.",[27,28,29,30,31,32,33],"Bone Marrow Failure","Hemoglobinopathies","Primary Immune Deficiency (PID)","Autoimmune Cytopenia","Immune Dysregulation","Hemophagocytic Lymphohistiocytosis (HLH)","Non Malignant Disorders","NOT_YET_RECRUITING","2026-06-16",{"date":37,"type":38},"2026-06-22","ACTUAL",{"date":40,"type":21},"2026-09",{"date":42,"type":21},"2035-09",{"name":44,"class":45},"Fred Hutchinson Cancer Center","OTHER",2,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":53,"eligibilityCriteria":54,"healthyVolunteers":11,"sex":16,"minAge":55,"maxAge":56,"enrollmentInfo":57,"targetDuration":4,"studyType":22,"phases":59,"briefSummary":61,"conditions":62,"keywords":68,"overallStatus":74,"whyStopped":4,"lastUpdateSubmitDate":75,"lastUpdatePostDateStruct":76,"startDateStruct":78,"completionDateStruct":80,"leadSponsor":82,"locationsCount":5},"100295674","phase-1-reduced-intensity-conditioning-and-familial-hla-mismatched-bmt-for-non-malignant-disorders-100295674","NCT03128996","Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders","A Phase I\u002FII Trial of Reduced Intensity Conditioning and Familial HLA-Mismatched Bone Marrow Transplantation in Children With Non-Malignant Disorders","FAM BMT","Inclusion Criteria:\n\n* Nonmalignant disorder requiring bone marrow transplant including bone marrow failure syndromes, metabolic disorders, immunologic disorders, or hemoglobinopathy\n* For patients with sickle cell disease, must have one of the following severe manifestations:\n\n  1. Overt or silent stroke or persistently elevated transcranial doppler velocities despite transfusion therapy\n  2. Recurrent acute chest syndrome with significant respiratory compromise each time\n  3. Sickle nephropathy\n  4. Recurrent admissions for vaso-occlusive episodes resulting in prolonged opioid use and poor quality of life with interrupted school attendance activity\n  5. Red cell alloimmunization with the need for chronic transfusions\n  6. Recurrent osteonecrosis or multiple joint involvement from avascular necrosis\n* Patients with sickle cell disease must have hemoglobin S \\\u003C 30% within 30 days prior to beginning alemtuzumab\n* Age \\\u003C\u002F= 20.99 years at the time of enrollment\n* Performance score \\>\u002F= 50\n* Left ventricular ejection fraction \\> 40% or left ventricular shortening fraction \\> 26% by echocardiogram\n* DLCO \\> 40% (corrected for hemoglobin) or pulse oximetry with a baseline O2 saturation of \\>\u002F= 90% on room air if too young to perform PFTs\n* Serum creatinine \\\u003C\u002F= 1.5x upper limit of normal for age and\u002For GFR \\> 70 mL\u002Fmin\u002F1.73m2\n* Direct bilirubin \\\u003C 2x upper limit of normal for age\n* ALT and AST \\\u003C 5x upper limit of normal for age\n* Participants who have or are receiving \\>\u002F= 8 packed red blood cell transfusions for \\>\u002F= 1 year or \\>\u002F= 20 packed red blood cell transfusions (lifetime cumulative) will undergo liver MRI for estimation of hepatic iron content.\n\n  1\\. Liver biopsy is indicated for hepatic iron content \\>\u002F= 7mg Fe\u002Fmg liver dry weight by liver MRI. Histologic examination of the liver must document for the absence of cirrhosis, bridging fibrosis, and active hepatitis\n* Female subjects of childbearing potential, must agree to practice 2 methods of contraception at the same time from the time of signing of informed consent through 12 months post transplant. Male subjects must agree to practice effective barrier contraception or practice true abstinence from the time of signing informed consent through 12 months post transplant.\n* Written informed consent must be obtained from all recipients in accordance with the guidelines of the institution's Human Studies Committee.\n\nExclusion Criteria:\n\n* Patients who have an HLA-identical sibling who is able and willing to donate bone marrow\n* Patients with cirrhosis or established bridging fibrosis of the liver or active hepatitis\n* Uncontrolled bacterial, viral, or fungal infection within 6 weeks prior to enrollment\n* Evidence of HIV infection or known HIV positive serology\n* Patients who have received a previous stem cell transplant\n* Patients who have received an investigational drug or device or off-label use of a drug or device within 3 months of enrollment\n* Females who are pregnant or breast feeding\n* Patients with active autoimmune disease (e.g. sarcoidosis, lupus, scleroderma)","1 Day","21 Years",{"count":58,"type":21},29,[60,24],"PHASE1","This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.",[63,64,65,66,28,67],"Severe Sickle Cell Disease","Bone Marrow Failure Syndromes","Metabolic Disorders","Immunologic Disorders","Non-malignant Disorders",[69,70,71,72,73],"Bone marrow transplant","Transplant","Transplantation","Reduced Intensity","Familial HLA mismatched","RECRUITING","2026-05-26",{"date":77,"type":38},"2026-05-29",{"date":79,"type":38},"2017-03-20",{"date":81,"type":21},"2033-04",{"name":83,"class":45},"Washington University School of Medicine",{"id":85,"slug":86,"hasResults":11,"nctId":87,"briefTitle":88,"officialTitle":88,"acronym":4,"eligibilityCriteria":89,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":90,"enrollmentInfo":91,"targetDuration":4,"studyType":93,"phases":4,"briefSummary":94,"conditions":95,"keywords":4,"overallStatus":74,"whyStopped":4,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":107},"100582280","treosulfan-therapeutic-drug-monitoring-in-pediatric-hematopoietic-stem-cell-transplant-recipients-100582280","NCT06861257","Treosulfan Therapeutic Drug Monitoring in Pediatric Hematopoietic Stem Cell Transplant Recipients","Inclusion Criteria:\n\n* Age range 0 - 18 years.\n* Life expectancy \\> 12 weeks.\n* Diagnosis of malignant or non-malignant disorder.\n* Pre-HSCT Lansky \u002F Karnofsky score ≥ 40%.\n* Indication to allogeneic or autologous HSCT with TREO as part of the pre-transplant conditioning regimen.\n* Negativity of pregnancy test for female patients.\n* Written informed consent signed by the parents or guardians.\n\nExclusion Criteria:\n\n* Absence of written informed consent signed by the parents or guardians.\n* Current clinically active infectious disease (including positive HIV serology or viral RNA).\n* Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction \\\u003C40%).\n* Liver dysfunction (AST\u002FALT ≥ 3 times institutional upper limit normal value -ULN- or bilirubin \\> 3 times ULN).\n* Renal dysfunction: serum creatinine \\> 1.5 times ULN or calculated creatinine clearance \\\u003C 60 ml\u002Fmin\u002F1.73 m2\n* End stage irreversible multi-system organ failure.\n* Pregnant or breast feeding female patient.","18 Years",{"count":92,"type":21},70,"OBSERVATIONAL","One of the major challenges to improve the outcome of hematopoietic stem cell transplantation (HSCT) is the reduction of toxicity and non-relapse mortality caused by the pre-transplant conditioning regimen, while maintaining efficacy. Treosulfan (TREO) (L-treitol-1,4-bis-methanesulfonate) is a busulfan analogue with a distinct site of alkylation that results in a more favourable toxicity profile in comparison with busulfan and total body irradiation. TREO is the prodrug of L-epoxybutane, a water-soluble bifunctional alkylating agent with remarkable myeloablative and immunosuppressive properties. The use of TREO, in combination with other chemotherapy agents, as part of the conditioning regimen for hematopoietic stem cell transplantation (HSCT) in children has progressively increased during the last decade for both malignant and non-malignant disorders. Data on TREO pharmacokinetics in the pediatric population are still scarce. To date, only a few studies, including small numbers of pediatric patients, have investigated the PK profile of TREO. These studies reported high variability of TREO pharmacokinetics, and the relationship between TREO exposure, toxicity and clinical outcome is still unresolved. Therefore, therapeutic drug monitoring with a personalized approach may be an important tool to optimize outcomes in the pediatric population. The aim of the investigators' study is to characterize TREO PK\u002FPD profiles in children undergoing HSCT and to evaluate the relationship between TREO exposure and early toxicity and clinical outcome.",[96,97,67],"Pediatric Hematopoietic Stem Cell Transplantation","Malignant Disorders","2026-04-23",{"date":100,"type":38},"2026-04-24",{"date":102,"type":38},"2021-02-24",{"date":104,"type":21},"2027-07-31",{"name":106,"class":45},"Fondazione IRCCS Policlinico San Matteo di Pavia",10,{"id":109,"slug":110,"hasResults":11,"nctId":111,"briefTitle":112,"officialTitle":113,"acronym":4,"eligibilityCriteria":114,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":115,"enrollmentInfo":116,"targetDuration":4,"studyType":22,"phases":118,"briefSummary":119,"conditions":120,"keywords":122,"overallStatus":74,"whyStopped":4,"lastUpdateSubmitDate":133,"lastUpdatePostDateStruct":134,"startDateStruct":136,"completionDateStruct":138,"leadSponsor":140,"locationsCount":141},"100126972","phase-1-campathfludarabinemelphalan-transplant-conditioning-for-non-malignant-diseases-100126972","NCT00920972","Campath\u002FFludarabine\u002FMelphalan Transplant Conditioning for Non-Malignant Diseases","A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime","Inclusion Criteria:\n\nStratum 1: Patient must have non-malignant disorder, excluding thalassemia. Must be receiving a 8\u002F8 HLA-matched bone marrow, related or unrelated Stratum 2: Patient must have thalassemia receiving 8\u002F8 HLA-matched bone marrow or 5-8\u002F8 HLA-matched UCB. Related or unrelated.\n\nStratum 3: Patient must have a hemoglobinopathy receiving 7\u002F8 HLA-matched bone marrow or 5-8\u002F8 HLA-matched UCB. Related or unrelated.\n\nStratum 4: Patient must have a non-malignant disorder (excluding hemoglobinopathy) receiving 7\u002F8 HLA-matched bone marrow or 5-8\u002F8 HLA-matched UCB. Related or unrelated.\n\nAll strata:\n\n* Recipient age \\\u003C 21 years\n* Lansky\u002FKarnofsky \\>\u002F= 40\n* Adequate pulmonary, renal, liver, and other organ function as defined in protocol\n* Negative pregnancy test\n* Adequate total nucleated cell or CD34+ dose of product as defined in protocol\n* If sickle cell, Hemoglobin S \\\u003C30%\n\nExclusion Criteria:\n\n* HIV positive\n* Invasive infection\n* Pregnancy\u002Flactating","20 Years",{"count":117,"type":21},220,[60,24],"The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells. It is also to determine major toxicities from these conditioning regimens, within the first 100 days after transplantation.",[65,121,28,67],"Hematologic, Immune, or Bone Marrow Disorders",[123,70,71,124,125,126,127,128,129,130,131,132],"Bone marrow","Hematopoietic","Umbilical cord","Related","Unrelated","Reduced","Non-myeloablative","Nonmyeloablative","Non-malignant","Nonmalignant","2026-03-24",{"date":135,"type":38},"2026-03-25",{"date":137,"type":38},"2001-12",{"date":139,"type":21},"2031-12",{"name":83,"class":45},28]