[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Paul Szabolcs\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":285},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,70,127,160,199,220,251],{"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":42,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":69},"100614826","phase-2-hematopoietic-stem-cell-transplantation-hsct-for-common-variable-immunodeficiency-cvid-and-other-autoimmune-manifestations-of-primary-immune-regulatory-disorders-pird-100614826",false,"NCT07284641","Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)","CVID\u002FPIRD","Inclusion Criteria:\n\n1. Patient, parent, or legal guardian must have given written informed consent. For pediatric subjects who are developmentally able, assent or affirmation will be obtained.\n2. Male or female, 5 through 40 years old, inclusive, at the time of informed consent.\n3. Patients must have evidence of common variable immunodeficiency (CVID) or other autoimmune manifestation of a primary immune regulatory disorder (PIRD). Genetic screening is required by a targeting gene panel to determine presence of genetic variations that may lead to inborn errors of immunity.\n\n   Examples of such diseases include, but are not limited to:\n   * Common variable immunodeficiency (CVID)\n   * Combined Immunodeficiency (CID)\n   * Immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX syndrome), IPEX like syndromes\n   * Combined immunodeficiency with defects in T-cell-mediated immunity, including Omenn syndrome and DiGeorge Syndrome\n   * Chronic Granulomatous Disease (CGD)\n   * Signal Transducer and Activator of Transcription (STAT 1) Gain of Function (STAT1 GOF)\n   * Signal Transducer and Activator of Transcription (STAT 3) Gain of Function (STAT3 GOF)\n   * Hypomorphic Recombination-Activating Genes (RAG) 1 and RAG 2\n   * CD40 or CD40L deficiency\n   * Mendelian Susceptibility to Mycobacterial Disease\n   * GATA-binding factor 2 (GATA2) Associated Immunodeficiency\n   * Mouth and Genital Ulcers with Inflamed Cartilage Syndrome (MAGIC)\n4. Must have previously failed, due to lack of response or intolerance, mycophenolate mofetil and a B cell-depleting antibody, such as Rituximab\n5. Glomerular Filtration Rate (GFR) ≥50 mL\u002Fmin\u002F1.73 m2\n6. Aspartate Aminotransferase (AST) ≤4x upper limit of normal\n7. Alanine Aminotransferase (ALT) ≤4x upper limit of normal\n8. Direct bilirubin ≤ 2.5 mg\u002FdL\n9. Human Immunodeficiency Virus (HIV) negative by serology and PCR\n10. Human T-cell Lymphotropic Virus (HTLV) negative by serology\n11. Cardiac ejection fraction ≥ 40% or shortening fraction ≥26%\n12. Forced Vital Capacity (FVC) and Forced Expiratory Volume in 1 second (FEV1) ≥40% predicted for age\n13. Peripheral Capillary Oxygen Saturation (SpO2) of \\>92% at rest on room air\n14. Subjects must be a minimum of 8 weeks post-solid organ transplant prior to start of conditioning, if applicable\n15. Negative pregnancy test for females \\>10 years old or who have reached menarche, unless surgically sterilized.\n16. All females of childbearing potential and sexually active males must agree to use a FDA approved method of birth control for up to 12 months after stem cell transplant or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause birth defects.\n17. Subject and\u002For parent guardian informed of the potential risks of infertility following stem cell transplant and advised to discuss sperm banking or oocyte harvesting.\n18. Transplant endorsement from clinical immunologist\n\nExclusion Criteria:\n\n1. Allergy to Dimethylsulfoxide (DMSO) or any other ingredient used in the manufacturing of the stem cell product\n2. Uncontrolled systemic infection, as determined by the appropriate confirmatory testing e.g. blood cultures, Polymerase chain reaction (PCR) testing, etc.\n3. Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of stem cell transplant\n4. Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the subject's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.","ALL","5 Years","40 Years",{"count":20,"type":21},25,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradiation (TBI) in those diagnosed with Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD).",[27,28,29,30,31,32,33,34,35,36,37,38,39,40,41],"Common Variable Immunodeficiency (CVID)","Primary Immune Regulatory Disorder","Immune Dysregulation","DiGeorge Syndrome","STAT 1 Gain of Function","STAT 3 Gain of Function","Hypomorphic RAG1 Deficiency","CD40 Ligand Deficiency","Mendelian Susceptibility to Mycobacterial Disease","GATA2 Associated Immunodeficiency","CD40 Deficiency","Hypomorphic RAG2 Deficiency","Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome","Omenn Syndrome","Chronic Granulomatous Disease",[43,44,45,46,47,48,49,50,51,52,30,53,41,31,32,54,55,35,36,56],"HSCT","CVID","PIRD","primary immune regulatory disorder","common variable immunodeficiency","IPEX like syndromes","Immune dysregulation","polyendocrinopathy","enteropathy","X-linked (IPEX syndrome)","Combined Immunodeficiency","Hypomorphic RAG 1 and RAG 2","CD40 or CD40L deficiency","Inflamed Cartilage Syndrome (MAGIC)","RECRUITING","2026-05-13",{"date":60,"type":61},"2026-05-15","ACTUAL",{"date":63,"type":61},"2026-05-04",{"date":65,"type":21},"2031-02-01",{"name":67,"class":68},"Paul Szabolcs","OTHER",1,{"id":71,"slug":72,"hasResults":11,"nctId":73,"briefTitle":74,"officialTitle":75,"acronym":76,"eligibilityCriteria":77,"healthyVolunteers":11,"sex":16,"minAge":78,"maxAge":79,"enrollmentInfo":80,"targetDuration":4,"studyType":82,"phases":4,"briefSummary":83,"conditions":84,"keywords":90,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":119,"lastUpdatePostDateStruct":120,"startDateStruct":122,"completionDateStruct":124,"leadSponsor":126,"locationsCount":69},"100403058","data-collection-study-of-patients-with-non-malignant-disorders-undergoing-ucbt-bmt-or-pbsct-with-ric-100403058","NCT04528355","Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC","A Prospective Outcomes Study of Pediatric and Adult Patients With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation With a Reduced-Intensity Conditioning Regimen (PRO-RIC)","PRO-RIC","Inclusion Criteria:\n\n1. Patient, parent, or legal guardian must have given written informed consent.\n2. Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.\n3. Patients should have a non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to the following:\n\n   A. Primary Immunodeficiency Syndromes\n   * Severe Combined Immune Deficiency (SCID) with NK cell activity\n   * Omenn Syndrome\n   * Bare Lymphocyte Syndrome (BLS)\n   * Combined Immune Deficiency (CID) syndromes\n   * Combined Variable Immune Deficiency (CVID) syndrome\n   * Wiskott-Aldrich Syndrome\n   * Leukocyte adhesion deficiency\n   * Chronic granulomatous disease (CGD)\n   * Hyper IgM (XHIM) syndrome\n   * IPEX syndrome\n   * Chediak-Higashi Syndrome\n   * Autoimmune Lymphoproliferative Syndrome (ALPS)\n   * Hemophagocytic Lymphohistiocytosis (HLH) syndromes\n   * Lymphocyte Signaling defects\n\n   B. Congenital Bone Marrow Failure Syndromes\n   * Congenital Amegakaryocytic Thrombocytopenia (CAMT)\n   * Osteopetrosis\n\n   C. Inherited Metabolic Disorders (IMD)\n   * Mucopolysaccharidoses\n\n     * Hurler syndrome (MPS I)\n     * Hunter syndrome (MPS II)\n   * Leukodystrophies\n\n     * Krabbe Disease, also known as globoid cell leukodystrophy\n     * Metachromatic leukodystrophy (MLD)\n     * X-linked adrenoleukodystrophy (ALD)\n   * Other inherited metabolic disorders\n\n     * Alpha Mannosidosis\n     * Gaucher Disease\n     * Other inheritable metabolic diseases where HSCT may be beneficial\n\n   D. Hereditary Anemias\n   * Thalassemia major\n   * Sickle cell disease (SCD)\n   * Diamond Blackfan Anemia (DBA)\n\n   E. Inflammatory Conditions\n   * Crohn's Disease or Inflammatory Bowel Disease\n   * IPEX or IPEX-like Syndromes\n   * Rheumatoid Arthritis\n   * Other inflammatory conditions where HSCT may be beneficial\n4. Subjects receive either umbilical cord blood, bone marrow, or peripheral blood stem cell transplant with an alemtuzumab, melphalan, thiotepa, fludarabine and hydroxyurea-based, reduced-intensity conditioning regimen, according to clinical practice at UPMC Children's Hospital of Pittsburgh.\n\nThere are no exclusion criteria.","2 Months","60 Years",{"count":81,"type":21},50,"OBSERVATIONAL","This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.",[85,86,87,88,89],"Primary Immunodeficiency (PID)","Congenital Bone Marrow Failure Syndromes","Inherited Metabolic Disorders (IMD)","Hereditary Anemias","Inflammatory Conditions",[91,40,92,93,94,95,96,97,98,99,100,101,102,103,104,105,106,107,108,109,110,111,112,113,114,115,116,117,118],"Severe Combined Immune Deficiency (SCID) with NK cell activity","Bare Lymphocyte Syndrome (BLS)","Combined Immune Deficiency (CID) syndromes","Wiskott-Aldrich Syndrome","Leukocyte adhesion deficiency","Chronic granulomatous disease (CGD)","Hyper IgM (XHIM) syndrome","IPEX syndrome","Chediak-Higashi Syndrome","Autoimmune Lymphoproliferative Syndrome (ALPS)","Hemophagocytic Lymphohistiocytosis (HLH) syndromes","Lymphocyte Signaling defects","Congenital Amegakaryocytic Thrombocytopenia (CAMT)","Osteopetrosis","Hurler syndrome (MPS I)","Hurler syndrome (MPS II)","Krabbe Disease, also known as Globoid Cell Leukodystrophy","Metachromatic leukodystrophy (MLD)","X-linked adrenoleukodystrophy (ALD)","Alpha Mannosidosis","Gaucher Disease","Thalassemia major","Sickle cell disease (SCD)","Diamond Blackfan Anemia (DBA)","Crohn's Disease","Inflammatory Bowel Disease","IPEX or IPEX-like Syndromes","Rheumatoid Arthritis","2026-01-12",{"date":121,"type":61},"2026-01-13",{"date":123,"type":61},"2020-08-20",{"date":125,"type":21},"2028-06-30",{"name":67,"class":68},{"id":128,"slug":129,"hasResults":11,"nctId":130,"briefTitle":131,"officialTitle":132,"acronym":4,"eligibilityCriteria":133,"healthyVolunteers":11,"sex":16,"minAge":134,"maxAge":79,"enrollmentInfo":135,"targetDuration":4,"studyType":22,"phases":137,"briefSummary":139,"conditions":140,"keywords":143,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":119,"lastUpdatePostDateStruct":153,"startDateStruct":154,"completionDateStruct":156,"leadSponsor":158,"locationsCount":159},"100324187","phase-1-lung-and-bone-marrow-transplantation-for-lung-and-bone-marrow-failure-100324187","NCT03500731","Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure","Lung Transplant in Tandem With Bone Marrow Transplant for Combined Lung and Bone Marrow Failure","Inclusion Criteria:\n\nIndividuals must meet all of the following criteria in order to be eligible for this study.\n\n1. Subject must be able to understand and provide informed consent.\n2. Male or female, 18 through 60 years old, inclusive, at the time of informed consent.\n3. Meet criteria for UNOS listing for lung transplantation.\n4. Patients must have evidence of end stage lung disease. Examples of such diseases include but are not limited to:\n\n   * Pulmonary Fibrosis\n   * COPD\u002FEmphysema\n5. Patients must have evidence of bone marrow failure with abnormal low cell count in at least one hematopoietic line, making the patient a poor candidate for long-term immunosuppressive therapy. Eligible patients must meet at least one of the following criteria:\n\n   * Unexplained, non-drug induced neutropenia with absolute neutrophils counts of \\\u003C1500\u002FµL the previous year, confirmed by repeat testing\n   * Unexplained, non-drug induced thrombocytopenia with mean platelets counts of \\\u003C100,000\u002FµL the previous year, confirmed by repeat testing\n   * Unexplained, non-hemolytic anemia, with a hemoglobin level of \\\u003C 12 g\u002FdL the previous year, confirmed by repeat testing\n6. GFR ≥45 mL\u002Fmin\u002F1.73 m2.\n7. AST, ALT ≤4x upper limit of normal, total bilirubin ≤ 2.5 mg\u002FdL, normal INR, albumin \\>3.0 g\u002FdL\n8. Cardiac ejection fraction ≥ 40% or shortening fraction ≥26%.\n9. Negative pregnancy test for females, unless surgically sterilized.\n10. All females of childbearing potential and sexually active males must agree to use a FDA approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause birth defect.\n11. Subject will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting.\n\nExclusion Criteria:\n\nIndividuals who meet any of these criteria are not eligible for this study.\n\n1. Inability or unwillingness of a participant to give written informed consent or comply with study protocol.\n2. Patients who have underlying malignant conditions.\n3. Patients who have non-malignant conditions not requiring BMT.\n4. HIV positive by serology or PCR, HTLV positive by serology. If HTLV serology is positive, it will be confirmed by nucleic acid testing (NAT). If HTLV NAT is negative, subject will remain eligible regardless of HTLV serology result.\n5. Females who are pregnant or who are lactating.\n6. Allergy to DMSO or any other ingredient used in the manufacturing of the stem cell product.\n7. Uncontrolled pulmonary infection, as determined by radiographic findings and\u002For significant clinical deterioration. NOTE: Pulmonary colonization with multiple organisms is common and will not be considered an exclusion criterion.\n8. Uncontrolled infection, as determined by the appropriate imaging and\u002For confirmatory testing e.g. blood cultures, PCR testing, etc.\n9. Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of transplant.\n10. Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.","18 Years",{"count":136,"type":21},8,[138,24],"PHASE1","The purpose of this study is to determine whether a lung transplantation prior to bone marrow transplantation (BMT) would allow for restoration of pulmonary function prior to BMT, allowing to proceed to BMT, to restore hematologic function.",[141,142],"Idiopathic Pulmonary Fibrosis","Emphysema or COPD",[144,145,141,142,146,147,148,149,150,43,151,152],"Lung Transplantation","Stem Cell Transplantation","Bone marrow transplantation","Cadaveric donor","Unrelated donor","HLA-Mismatch","BMT","IPF","Pulmonary fibrosis",{"date":121,"type":61},{"date":155,"type":61},"2018-04-19",{"date":157,"type":21},"2028-12",{"name":67,"class":68},2,{"id":161,"slug":162,"hasResults":11,"nctId":163,"briefTitle":164,"officialTitle":165,"acronym":166,"eligibilityCriteria":167,"healthyVolunteers":11,"sex":16,"minAge":78,"maxAge":168,"enrollmentInfo":169,"targetDuration":4,"studyType":22,"phases":171,"briefSummary":172,"conditions":173,"keywords":176,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":191,"lastUpdatePostDateStruct":192,"startDateStruct":194,"completionDateStruct":196,"leadSponsor":198,"locationsCount":69},"100206313","phase-2-reduced-intensity-conditioning-for-non-malignant-disorders-undergoing-ucbt-bmt-or-pbsct-100206313","NCT01962415","Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT","A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults ≤55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation","HSCT+RIC","Inclusion:\n\n1. A 4\u002F6, 5\u002F6 or 6\u002F6 HLA matched related or unrelated UCB unit available that will deliver a pre-cryopreservation total nucleated cell dose of ≥ 3 x 10e7 cells\u002Fkg, or double unit grafts, each cord blood unit delivering at least 2 x 10e7 cells\u002Fkg OR an 8 of 8 or 7 of 8 HLA allele level matched unrelated donor bone marrow or peripheral blood progenitor graft.\n2. Adequate organ function as measured by:\n\n   1. Creatinine ≤ 2.0 mg\u002FdL and creatinine clearance ≥ 50 mL\u002Fmin\u002F1.73 m2.\n   2. Hepatic transaminases (ALT\u002FAST) ≤ 4 x upper limit of normal (ULN).\n   3. Adequate cardiac function by echocardiogram or radionuclide scan (shortening fraction \\> 26% or ejection fraction \\> 40% or \\> 80% of normal value for age).\n   4. Pulmonary evaluation testing demonstrating CVC or FEV1\u002FFVC of ≥ 50% of predicted for age and\u002For resting pulse oximeter ≥ 92% on room air or clearance by the pediatric or adult pulmonologist. For adult patients DLCO (corrected for hemoglobin) should be ≥ 50% of predicted if the DLCO can be obtained.\n3. Written informed consent and\u002For assent according to FDA guidelines.\n4. Negative pregnancy test if pubertal and\u002For menstruating.\n5. HIV negative.\n6. A non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to:\n\n   1. Primary Immunodeficiency syndromes including but not limited to:\n\n      * Severe Combined Immune Deficiency (SCID) with NK cell activity\n      * Omenn Syndrome\n      * Bare Lymphocyte Syndrome (BLS)\n      * Combined Immune Deficiency (CID) syndromes\n      * Combined Variable Immune Deficiency (CVID) syndrome\n      * Wiskott-Aldrich Syndrome\n      * Leukocyte adhesion deficiency\n      * Chronic granulomatous disease (CGD)\n      * X-linked Hyper IgM (XHIM) syndrome\n      * IPEX syndrome\n      * Chediak - Higashi Syndrome\n      * Autoimmune Lymphoproliferative Syndrome (ALPS)\n      * Hemophagocytic Lymphohistiocytosis (HLH) syndromes\n      * Lymphocyte Signaling defects\n      * Other primary immune defects where hematopoietic stem cell transplantation may be beneficial\n   2. Congenital bone marrow failure syndromes including but not limited to:\n\n      * Dyskeratosis Congenita (DC)\n      * Congenital Amegakaryocytic Thrombocytopenia (CAMT)\n      * Osteopetrosis\n   3. Inherited Metabolic Disorders (IMD) including but not limited to:\n\n      * Mucopolysaccharidoses\n\n        * Hurler syndrome (MPS I)\n        * Hunter syndrome (MPS II)\n      * Leukodystrophies\n\n        * Krabbe Disease, also known as globoid cell leukodystrophy\n        * Metachromatic leukodystrophy (MLD)\n        * X-linked adrenoleukodystrophy (ALD)\n        * Hereditary diffuse leukoencephalopathy with spheroids (HDLS)\n      * Other inherited metabolic disorders\n\n        * alpha mannosidosis\n        * Gaucher Disease\n      * Other inheritable metabolic diseases where hematopoietic stem cell transplantation may be beneficial.\n   4. Hereditary anemias\n\n      * Thalassemia major\n      * Sickle cell disease (SCD) - patients with sickle disease must have one or more of the following:\n\n        * Overt or silent stroke\n        * Pain crises ≥ 2 episodes per year for past year\n        * One or more episodes of acute chest syndrome\n        * Osteonecrosis involving ≥ 1 joints\n        * Priapism\n      * Diamond Blackfan Anemia (DBA)\n      * Other congenital transfusion dependent anemias\n   5. Inflammatory Conditions\n\n      * Crohn's Disease\u002FInflammatory Bowel Disease\n\nExclusion:\n\n1. Allogeneic hematopoietic stem cell transplant within the previous 6 months.\n2. Any active malignancy or MDS.\n3. Severe acquired aplastic anemia.\n4. Uncontrolled bacterial, viral or fungal infection (currently taking medication and with progression of clinical symptoms).\n5. Pregnancy or nursing mother.\n6. Poorly controlled pulmonary hypertension.\n7. Any condition that precludes serial follow-up.","55 Years",{"count":170,"type":21},100,[24],"The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.",[85,86,87,88,89,174,175],"Systemic Juvenile Idiopathic Arthritis (sJIA)","Juvenile Rheumatoid Arthritis (JRA)",[177,40,92,93,178,94,95,96,179,98,180,100,101,102,181,103,104,182,105,183,184,185,108,109,186,111,112,113,114,115,116,187,188,189,190,174,175],"Severe Combined Immune Deficiency (SCID)","Combined Variable Immune Deficiency (CVID) syndrome","X-linked Hyper IgM (XHIM) syndrome","Chediak - Higashi Syndrome","Dyskeratosis Congenita (DC)","Mucopolysaccharidoses","Hunter syndrome (MPS II)","Leukodystrophies","Krabbe Disease","Alpha mannosidosis","Hematopoietic Stem Cell Transplant (HSCT)","Congenital transfusion dependent anemias","Globoid cell leukodystrophy","Hereditary diffuse leukoencephalopathy with spheroids (HDLS)","2025-12-08",{"date":193,"type":61},"2025-12-15",{"date":195,"type":61},"2014-02-04",{"date":197,"type":21},"2027-11",{"name":67,"class":68},{"id":200,"slug":201,"hasResults":11,"nctId":202,"briefTitle":203,"officialTitle":204,"acronym":4,"eligibilityCriteria":205,"healthyVolunteers":11,"sex":16,"minAge":206,"maxAge":79,"enrollmentInfo":207,"targetDuration":4,"studyType":22,"phases":209,"briefSummary":210,"conditions":211,"keywords":212,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":191,"lastUpdatePostDateStruct":214,"startDateStruct":215,"completionDateStruct":217,"leadSponsor":219,"locationsCount":159},"100109740","phase-1-autologous-stem-cell-transplantation-for-crohns-disease-100109740","NCT00692939","Autologous Stem Cell Transplantation for Crohn's Disease","Autologous Stem Cell Transplantation With CD34-Selected Peripheral Blood Stem Cells (PBSC) in Pediatric and Adult Patients With Severe Crohn's Disease","5.1 Inclusion Criteria\n\n1. Subject and\u002For guardian must be able to understand and provide informed consent.\n2. Male or female, 10 through 60 years old, inclusive at time of informed consent.\n3. Examples of subjects for whom stem cell transplant therapy would be appropriate include, but are not limited to:\n\n   * Patients who have had prior surgery and subsequent severe recurrent disease in spite of aggressive maintenance therapy, necessitating consideration of further extensive surgical resections.\n   * Patients who have diffuse small bowel and colonic disease and who are refractory to aggressive medical treatment, and not eligible for treatment using a surgical approach without the risk of precipitating short bowel syndrome and dependence of parenteral nutrition or who have other conditions that preclude surgery\n   * Patients with a persistently high Harvey Bradshaw Index (HBI) (\\>6), CDAI (\\>250) or Pediatric CD Activity Index (PCDAI\\>45) (44) score or those in the lower, moderate range (HBI ≤ 6), (CDAI \\\u003C 250), (PCDAI 30-45), but who are dependent on daily doses of corticosteroids, that are unable to be withdrawn, and aggressive medical treatment to maintain moderate disease status.\n   * Patients who have resistant complications of CD unresponsive to medical management including multiple enteric fistulas, enterovesicular or enterovaginal fistulas, severe perianal disease, debilitating arthritis, severe skin lesions (pyoderma), and severe bony complications of the disease and therapy (aseptic necrosis, pathologic fractures).\n   * Patients who developed severe complications to while receiving medical management such as pancreatitis following 6-Mercaptopurine, colitis following 5-ASA or those with severe hypersensitivity to TNFalpha inhibitors (infliximab, adalimumab, certolizumab pegol), anti-integrin agents (natalizumab, vedolizumab) or anti-IL12\u002F23 agents (ustekinumab).\n   * Patients with stomas are eligible.\n4. No surgical therapeutic option secondary to risk of short bowel syndrome or patient refusal.\n5. Harvey Bradshaw Index (HBI) or CD activity score \\>5, CDAI \\>250 or PCDAI \\>30.\n6. Platelet count greater than 100,000\u002Fmm3.\n7. Absolute neutrophil count greater than 1500\u002Fmm3 (unless secondary to 6MP therapy).\n8. Creatinine ≤ 2.0 mg\u002FdL.\n9. No history of coronary artery disease; resting LVEF ≥ 40% or shortening fraction ≥ 26%.\n10. FEV1\u002FFVC ≥ 60% predicted for age; DLCO ≥ 60% predicted value for age.\n11. Negative pregnancy test for females ≥ 10 years old or who have reached menarche, unless surgically sterilized.\n12. All females or childbearing potential and sexually active males must agree to use a FDA approved method of birth control for up to 24 months after PBSC transplant or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause a birth defect.\n\n5.2 Exclusion Criteria\n\n1. Patients who have not been treated with adequate dosing of 6-MP, 5-ASA products and metronidazole.\n2. Patients who achieved a sustained, corticosteroid free response to anti-TNF alpha therapy, anti-integrin therapy or anti-IL12\u002F23 therapy after a 4 month course of treatment.\n3. Toxic megacolon, intestinal perforation\n4. Conjugated bilirubin \\> 2.0 mg\u002FdL.\n5. Pregnancy or nursing mother\n6. HIV\u002FHTLV seropositive, HBsAg, or HCV RNA positive by PCR\n7. Active infection, as determined by the appropriate confirmatory testing e.g. blood cultures, PCR testing, etc., within two weeks of mobilization and high dose chemotherapy.\n8. Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.","10 Years",{"count":208,"type":21},20,[138,24],"The objective of this study is to evaluate the safety and effectiveness of administering high-dose chemotherapy followed by infusion of autologous CD34-selected peripheral blood stem cells (PBSC) in pediatric and adult patients with severe Crohn's disease.",[115],[213,115],"Stem cell transplantation",{"date":193,"type":61},{"date":216,"type":61},"2012-06-26",{"date":218,"type":21},"2027-12",{"name":67,"class":68},{"id":221,"slug":222,"hasResults":11,"nctId":223,"briefTitle":224,"officialTitle":225,"acronym":226,"eligibilityCriteria":227,"healthyVolunteers":11,"sex":16,"minAge":228,"maxAge":79,"enrollmentInfo":229,"targetDuration":4,"studyType":22,"phases":230,"briefSummary":231,"conditions":232,"keywords":237,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":242,"lastUpdatePostDateStruct":243,"startDateStruct":245,"completionDateStruct":247,"leadSponsor":249,"locationsCount":250},"100334119","phase-2-autologous-stem-cell-transplantation-in-patients-with-systemic-sclerosis-100334119","NCT03630211","Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis","Autologous Stem Cell Transplantation With CD34-Selected Peripheral Blood Stem Cells (PBSC) in Patients With Treatment Resistant Systemic Sclerosis (SSc)","SSc","Cohort 1: Children, Adolescents and Young Adults (Cohort 1)\n\nInclusion:\n\nIndividuals must meet all the following criteria to be eligible for this study.\n\n1. Patient, parent, or legal guardian must have given written informed consent. For patients ≥ 168 years of age who are developmentally able, assent or affirmation will be obtained.\n2. Age 8-24, inclusive, at time of consent.\n3. Diagnosed with Systemic Sclerosis (SSc) at the age of ≤19.\n4. Failure to respond, specifically no improvement or progression of disease, to at least 2 disease-modifying antirheumatic drugs (DMARDS) within 12 months of consent with any of the following conditions:\n\n   1. Progression of skin thickening over the past 6 months or Modified Rodnan skin score (mRSS) ≥ 20\n   2. Progression of ILD within 18 months prior to consent. Progression to be determined by either of the following:\n\n      * CT scan showing increased ground glass opacities or reticulations OR\n      * Pulmonary function testing (PFTs) showing a decrease in FVC% or DLCO% predicted value of ≥10%.\n   3. Myositis - CPK \\> 2x upper limit of normal or MRI consistent with myositis\n   4. Childhood Myositis Assessment Score \\\u003C 30\n   5. Arthritis\n   6. Digital tip ulcerations\n5. Cardiology clearance to undergo stem cell transplantation (documented in subject's medical chart)\n6. Negative for human immunodeficiency virus (HIV), hepatitis B virus and hepatitis C virus, all confirmed by PCR testing.\n7. Negative pregnancy test for females. who have reached menarche.\n\n87\\. All females of childbearing potential and sexually active males must agree to use an FDA approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause a birth defect.\n\nExclusion:\n\nIndividuals who meet any of these criteria are not eligible for this study.\n\n1. FVC \\\u003C35%, determined by pulmonary function tests for those able to complete spirometry adequately (per investigator's determination)\n2. O2 sat \\\u003C92% at rest in room air\n3. Estimated CrCl \\\u003C40 mL\u002Fmin,using Cockcroft-Gault formula based on actual body weight.\n4. Active, untreated SSc renal crisis at the time of consent.\n5. ALT \\> 4x upper limit of normal.\n6. Active, uncontrolled infection that would be a contraindication to safe use of high-dose immunosuppressive therapy or cyclophosphamide.\n7. Hematologic abnormalities as defined by any of the following peripheral blood counts:\n\n   1. ANC \\\u003C 1500 cell\u002FµL.\n   2. Platelets \\\u003C 100,000 cells\u002F µL.\n   3. Hemoglobin \\\u003C 9.0 g\u002FdL.\n8. Malignancy within 2 years prior to enrollment, excluding adequately treated squamous cell cancer, basal cell carcinoma or carcinoma in situ. Treatment should have been completed with cure\u002Fremission status documented for at least 2 years.\n9. Past or current medical problems or findings from medical history, physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.\n\nCohort 2 for Adults\n\nInclusion:\n\nIndividuals must meet all the following criteria to be eligible for this study.\n\n1. Patient, parent, or legal guardian must have given written informed consent. For patients ≥ 16 years of age who are developmentally able, assent or affirmation will be obtained.\n2. Age 1618-705560, inclusive, at time of consent. Patients up to age 24, diagnosed with SSc at age ≤ 19, will be included in Cohort 1 and evaluated according to the Pediatric and Young Adult criteria listed in sections 3.1.1 and 3.1.2.\n3. Diagnosed with Systemic Sclerosis (SSc), according to the 2013 ACR\u002FEULAR criteria (van den Hoogen et al., 2013).\n4. All patients must meet either the following skin or ILD criteria. Disease duration is defined as time from first non-Raynaud symptom.\n\n   Skin Criteria: Diffuse SSc, defined by presence of proximal skin thickening and:\n\n   A. If disease duration is of \\\u003C2 years, patients must have a calculated mortality risk prediction score which places them in the intermediate or high- risk category (Domsic et al., 2016). Refer to Appendix 5 for calculation criteria.\n\n   B. If disease duration is of \\>2 years, patients must have evidence of active cutaneous disease based upon 1) a worsening Modified Rodnan Skin Score (MRSS) in the preceding three months or 2) the presence of palpable tendon friction rubs.\n\n   ILD Criteria:\n\n   A. The presence of recognized fibrosis on imaging of \\\u003C2 years AND either \\> 10% of lung involvement by CT scan or FVC% pred \\\u003C80% or B. Fibrosis on imaging of any duration with a decline in FVC% pred of ≥10% over the preceding 12-18 months.\n5. Negative for human immunodeficiency virus (HIV), hepatitis B virus and hepatitis C virus, all confirmed by PCR testing.\n6. Negative pregnancy test for females.\n7. All females of childbearing potential and sexually active males must agree to use an FDA approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause a birth defect.\n\nExclusion Criteria Individuals who meet any of these criteria are not eligible for this study.\n\n1. Moderate to severe cardiac involvement defined by any of the following:\n\n   1. New York Heart Association classification of heart failure ≥3.\n   2. Left ventricular ejection fraction (LVEF) \\\u003C50% as determined by cardiac MRI.\n   3. Significant pulmonary hypertension, for subjects ≥ 18 years of age, defined as mean PASP ≥30 mmHg determined by right heart catheterization, or for subjects ≤ 17 years of age, defined as mean PASP \\>45 mmHg, determined by echocardiogram.\n   4. Atrial tachycardia, atrial fibrillation or atrial flutter of ≥1-minute duration, determined by electrocardiogram (EKG) or, cardiac event monitor and\u002For implanted loop recorder (if applicable), or on anti-arrhythmic therapy for the arrhythmias listed above.\n   5. Ventricular tachycardia of ≥6 beats at rate of ≥100 beats per minute, determined by EKG or, cardiac event monitor and\u002For implanted loop recorder (if applicable), or on an anti-arrhythmic therapy for any ventricular arrhythmia.\n   6. Left bundle branch block, bifascicular heart block, Mobitz 2 heart block, complete heart block or infarction pattern as determined by EKG or, cardiac event monitor and\u002For implanted loop recorder\n   7. Presence of pacemaker or implantable cardioverter defibrillator.\n2. Moderate to severe pulmonary involvement defined by any of the following:\n\n   1. Hemoglobin-corrected DLCO \\\u003C45%, determined by pulmonary function tests.\n   2. FVC \\\u003C45%, determined by pulmonary function tests.\n   3. pO2 \\\u003C70 mmHg, determined by an arterial blood gas (not applicable for subjects ≤17 years of age).\n   4. pCO2 ≥45 without supplemental O2 determined by an arterial blood gas (not applicable for subjects ≤17 years of age).\n   5. O2 sat \\\u003C92% at rest without supplemental O2, determined by an arterial blood gas (not applicable for subjects ≤17 years of age).\n   6. Six-minute walk (6MW) results \\\u003C400 feet.\n3. Steroid therapy defined by either of the following:\n\n   1. Subjects who received \\> 10 mg\u002Fday prednisone or equivalent within 30 days prior to start of conditioning regimen on Day -21.\n   2. Subjects who have been treated for concurrent illnesses (eg, asthma) with the equivalent of prednisone 1 mg\u002Fkg\u002Fday or its equivalent for \\> 5 days on \\> 2 occasions during the previous 12 months (prior to conditioning) or \\> 1 occasion in the prior 6 months (prior to conditioning).\n4. Estimated CrCl \\\u003C40 mL\u002Fmin,using Cockcroft-Gault formula based on actual body weight.\n5. Serum creatinine \\>2.0 mg\u002FdL.\n6. Active, untreated SSc renal crisis at the time of consent.\n7. Dependence on nutritional supplementation\u002Fhyperalimentation.\n8. Active gastric antral vascular ectasia (GAVE), defined by a decrease in hemoglobin greater than 1 g\u002FdL in the preceding 60 days, attributed to GAVE.\n9. Active hepatitis defined by any of the following:\n\n   1. AST \\> 2x upper limit of normal.\n   2. ALT \\> 2x upper limit of normal.\n   3. Bilirubin \\>2x upper limit of normal.\n10. Evidence of moderate to severe periportal fibrosis, determined by liver biopsy, if applicable.\n11. Active, uncontrolled infection that would be a contraindication to safe use of high-dose immunosuppressive therapy or cyclophosphamide.\n12. Hematologic abnormalities as defined by any of the following peripheral blood counts:\n\n    1. ANC \\\u003C 1500 cell\u002FµL.\n    2. Platelets \\\u003C 100,000 cells\u002F µL.\n    3. Hemoglobin \\\u003C 9.0 g\u002FdL.\n13. Evidence of myelodysplasia (MDS), confirmed by bone marrow aspirate, if applicable.\n14. Malignancy within 2 years prior to enrollment, excluding adequately treated squamous cell cancer, basal cell carcinoma or carcinoma in situ. Treatment should have been completed with cure\u002Fremission status documented for at least 2 years, with the exception of hormonal therapy for breast cancer.\n15. Females who are pregnant or who are lactating.\n16. Tobacco use, by subject admission, within previous 4 weeks of time of consent.\n17. History of sensitivity to murine proteins or E. coli proteins.\n18. Known history of substance abuse, determined by medical record or subject admission, within 6 months of time of consent.\n19. Patient with systemic reaction to anti-thymocyte globulin or any other equine gamma globulin preparation\n20. Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.","8 Years",{"count":136,"type":21},[24],"The purpose of this study is to determine whether a regimen of high-dose immunoablative therapy will demonstrate safety that is consistent or improved with other published regimens in SSc patients, while maintaining a treatment effect.",[233,234,235,236],"Systemic Sclerosis","Diffuse Sclerosis Systemic","Interstitial Lung Disease","Pulmonary Hypertension",[145,233,238,235,239,236,240,241],"Scleroderma","ILD (Interstitial Lung Disease)","BMT ( bone marrow transplantation)","Autologous","2025-10-13",{"date":244,"type":61},"2025-10-15",{"date":246,"type":61},"2018-07-31",{"date":248,"type":21},"2026-08-01",{"name":67,"class":68},3,{"id":252,"slug":253,"hasResults":11,"nctId":254,"briefTitle":255,"officialTitle":256,"acronym":4,"eligibilityCriteria":257,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":258,"targetDuration":4,"studyType":22,"phases":260,"briefSummary":261,"conditions":262,"keywords":266,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":277,"lastUpdatePostDateStruct":278,"startDateStruct":280,"completionDateStruct":282,"leadSponsor":284,"locationsCount":69},"100335893","phase-1-t-cell-depleted-alternative-donor-bone-marrow-transplant-for-sickle-cell-disease-scd-and-other-anemias-100335893","NCT03653338","T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias","T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias","Inclusion Criteria\n\n1. Patient, parent, or legal guardian must have given written informed consent and\u002For assent according to FDA guidelines.\n2. Ages 5 years to 40 years, at time of consent.\n3. Diagnosis of Sickle Cell Disease (Hemoglobin SS, Sβ0-thalassemia) complicated by any of the following:\n\n   * Recurrent acute painful episodes (also known as vaso-occlusive crises; VOC) despite supportive care, minimum of 2 new pain events per year requiring hospitalization for parenteral pain management in the previous 2 years.\n   * Recurrent acute chest syndrome (ACS) despite supportive care, minimum of 2 episodes in preceding 2-year period.\n   * Stroke or neurologic event lasting \\> 24 hours with an accompanying infarct on MRI in any patient for all ages; Brain MRI with silent infarct without clinical event in patients ≤ 16 years.\n   * Chronic transfusion therapy defined as \\> 8 packed red blood cell transfusions per year in the year prior to enrollment and\u002For evidence of red blood cell alloimmunization.\n   * Elevated transcranial Doppler velocities - \\> 200 cm\u002Fs, via the non-imaging technique or \\> 185 cm\u002Fs by the imaging technique measured on 2 separate occasions ≥ 1-month apart\n   * Elevated TRV \\> 2.6m\u002Fs in patients ≥ 16 years old.\n   * Sickle-related renal insufficiency and\u002For sickle hepatopathy and\u002For any irreversible end-organ damage in patients ≥ 16 years old.\n\n   OR Diagnosis of beta-thalassemia or Diamond-Blackfan anemia complicated by transfusion dependence with evidence of iron overload.\n4. A minimum donor match of 4\u002F8 via high resolution HLA typing at HLA-A, -B, -C, -DRB1 loci in the related setting or minimum donor match of 6\u002F8 via high resolution HLA typing at HLA-A, -B, -C, -DRB1 loci (with the DRB1 locus as a full match requirement). An unrelated donor and cord blood search must have been completed without an eligible 8\u002F8 matched unrelated donor or 6\u002F8 cord blood unit available. Patients who may have acceptable cord blood donor options (4\u002F6 or better) but are limited by cell dose of a single cord will also be eligible for the proposed study.\n5. Adequate function of other organ systems as measured by:\n\n   * Creatinine clearance or GFR ≥ 45 ml\u002Fmin\u002F1.73m.\n   * Hepatic transaminases (ALT\u002FAST) ≤ 3 x upper limit of normal.\n   * Liver MR imaging for iron content should be performed in all patients with Ferritin \\> 500 ng\u002FmL. If hepatic iron content \\> 10mg Fe\u002Fg liver should have hepatology consultation and liver biopsy to confirm absence of cirrhosis, fibrosis or hepatitis.\n   * Adequate cardiac function as measure by echocardiogram (shortening fraction \\> 26% or ejection fraction \\> 40% or \\>80% of age-specific normal).\n   * Pulmonary evaluation testing demonstrating FEV1\u002FFVC ≥ 60% of predicted for age and\u002For resting pulse oximeter ≥ 92% on room air.\n   * Cardiology clearance to proceed with conditioning regimen and HSCT.\n   * Pulmonology clearance to proceed with conditioning regimen and HSCT.\n6. Subjects must be human immunodeficiency virus (HIV) negative by PCR.\n7. Negative pregnancy test for females ≥10 years old or who have reached menarche, unless surgically sterilized.\n8. All females of childbearing potential and sexually active males must agree to use an FDA approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause a birth defect.\n9. Subject and\u002For parent guardian will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting (Refer to section,\n10. Hydroxyurea must have been trialed and failed in patients with sickle cell disease.\n\nPatient Exclusion Criteria\n\n1. Patients with alternate, superior donor options (matched sibling donor or matched unrelated donor).\n2. Patients who have undergone stem cell transplantation in the 6 months prior to anticipated conditioning.\n3. Patients with history of a central nervous system (CNS) event within six months prior to start of conditioning (patient will be delayed until eligible).\n4. Patients who are pregnant or lactating\n5. Patients with uncontrolled bacterial, viral or fungal infection\n6. Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.",{"count":259,"type":21},5,[138,24],"The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and\u002For haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and\u002For haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.",[263,264,265],"Sickle Cell Anemia","Beta-thalassemia Major","Diamond-blackfan Anemia",[267,268,269,270,213,271,272,273,274,275,276],"Sickle Cell","Diamond-Blackfan","Beta-thalassemia","Anemia","Unrelated Donor","haploidentical","hematopoietic stem cell transplant (HSCT)","bone marrow transplant (BMT)","mismatched","t-cell depletion","2025-08-07",{"date":279,"type":61},"2025-08-12",{"date":281,"type":61},"2018-08-02",{"date":283,"type":21},"2027-08-01",{"name":67,"class":68},""]