[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"chronic-granulomatous-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:chronic-granulomatous-disease":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,47,74,126],{"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":29,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100453852","phase-1-nadph-oxidase-correction-in-mrna-transfected-granulocyte-enriched-cells-in-chronic-granulomatous-disease-cgd-100453852",false,"NCT05189925","NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)","NADPH Oxidase Correction in mRNA Transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)","* INCLUSION CRITERIA:\n\nIndividuals must meet all of the following criteria to be eligible for study participation:\n\n* Males aged 18 to 75 years\n* CGD confirmed by DHR and deficiency subtype confirmed by protein analysis and\u002For genetic sequencing\n* Has a physician at home for follow-up care\n* Able to provide informed consent\n* For men who engage in activities that can result in pregnancy, agree to use contraception when engaging in sexual activities that can result in pregnancy. Contraception must be used from screening through 3 months after the CGD-Grans infusion. Acceptable methods of contraception include the following:\n\n  * Hormonal contraception\n  * Male or female condom\n\nEXCLUSION CRITERIA:\n\nIndividuals meeting any of the following criteria will be excluded from study participation:\n\n* Clinically unstable due to moderate to severe acute systemic infections as defined by persistent resting tachypnea, tachycardia, or hypoxia of \\>20% from baseline and hypotension.\n* Current or history of stage 4 chronic kidney disease or estimated glomerular filtration rate \\[eGFR\\] \\\u003C30 mL\u002Fmin\u002F1.73 m\\^2 within 90 days of baseline.\n* Unstable diabetes mellitus with hemoglobin A1c \\>7.0% and fasting serum glucose \\>200 mg\u002FdL at screening.\n* Current or history of heart failure stage D as defined by the American College of Cardiology Foundation\u002FAmerican Heart Association guidelines.\n* History of arrhythmias that are symptomatic and deemed clinically unsafe for participation by NIH CC Cardiology consultation.\n* Current or history of invasive cancers that require chemotherapy within 5 years of screening.\n* Active hepatitis B, C, or HIV infections at screening.\n* Unstable hypertension requiring addition of new anti-hypertensives within 2 weeks of screening.\n* Impaired renal function that is unstable, with serum creatinine \\>3.0 mg\u002FdL and rising.\n* Serum transaminases and bilirubin that are \\>3 x the upper limit of normal.\n\nNOTE: For prospective subjects who, per PI assessment at screening, have abnormal liver function tests, and\u002For a significant history of liver disease, and\u002For liver-related complications of CGD, and who otherwise meet eligibility criteria \\[i.e. those who do NOT meet any of the exclusion set forth herein\\], a hepatology consult will be required at screening, and participation must be approved in writing by hepatology to the PI.\n\n* Electrocardiogram abnormalities indicative of acute myocardial injury, or arrhythmias that presents anesthetic risks, at screening.\n* Anemia with hemoglobin \\\u003C8 g\u002FdL (transfusions to correct anemia permitted).\n* Thrombocytopenia (platelets \\\u003C50 x10\\^9 cells\u002FL) (platelet transfusions to correct thrombocytopenia permitted).\n* Profound thrombocytopenia (platelet counts \\\u003C10,000\u002Fmicroliter) that is not reversible with platelet transfusions.\n* Abnormal prothrombin time\u002Fpartial thromboplastin time (PT\u002FPTT) values outside the ranges accepted at the NIH CC that are not corrected or that cannot be attributed to presence of Lupus anticoagulant (commonly found in CGD patients).\n* Inherited bleeding disorder that precludes line placement.\n* Severe oxygen-dependent pulmonary disease that increases risks of procedures that may require sedation.\n* History of or current evidence of alcohol or illicit drug abuse or dependence.\n* Participation in a clinical protocol that includes an intervention that, in the opinion of the investigator, may affect the results of the current study.\n\nSubjects will be selected in an equitable manner from the available pool of potentially eligible individuals, without regard to factors such as gender, race, ethnicity, socioeconomic status, etc, except for age and sex.","MALE","18 Years","75 Years",{"count":20,"type":21},25,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","Background:\n\nCGD is caused by a gene mutation. For people with CGD, their cells cannot kill germs well, so they can get frequent or life-threatening infections. Researchers want to see if a new procedure can help a person s cells kill germs for a short time. It uses messenger RNA (mRNA) to deliver correct instructions for the gene mutation to the cells.\n\nObjective:\n\nTo test a procedure in which mRNA is added to a person s blood cells.\n\nEligibility:\n\nMales aged 18-75 with CGD with a mutation in the gene that makes the protein gp91phox.\n\nDesign:\n\nParticipants will be screened with:\n\nMedical history\n\nPhysical exam\n\nBlood and urine tests\n\nSwab to test for strep throat\n\nSome screening tests will be repeated during the study.\n\nParticipants will be admitted to the NIH Clinical Center hospital for at least 7 days. They will have apheresis. For this, a medicine is injected under their skin to prepare their white blood cells for collection. An IV line is placed into an arm vein. Blood goes through the IV line into a machine that divides whole blood into red blood cells, plasma, and white blood cells. The white blood cells are removed, and the rest of the blood is returned to the participant through an IV line in their other arm. The next day, they will get their mRNA-corrected cells via IV. They will be monitored for 3 more days.\n\nAfter discharge, participants will keep a symptom diary. They will be contacted weekly for one month, and then once a month. They will have a follow-up visit 3 months after the infusion.",[27,28],"Chronic Granulomatous Disease","Infection",[30,31,32,33],"Primary Immune Deficiency","systemic infection","autologous transfusion","Apheresis","RECRUITING","2026-06-23",{"date":37,"type":38},"2026-06-24","ACTUAL",{"date":40,"type":38},"2022-07-22",{"date":42,"type":21},"2026-07-01",{"name":44,"class":45},"National Institute of Allergy and Infectious Diseases (NIAID)","NIH",1,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":54,"minAge":4,"maxAge":4,"enrollmentInfo":55,"targetDuration":4,"studyType":22,"phases":57,"briefSummary":59,"conditions":60,"keywords":61,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":46},"100335289","lentiviral-gene-therapy-for-cgd-100335289","NCT03645486","Lentiviral Gene Therapy for CGD","Lentiviral Gene Therapy for Chronic Granulomatous Disease (CGD)","Inclusion Criteria:\n\n1. CGD patients \\>= 0 years of age\n2. Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or significantly reduced biochemical activities of the NADPH-oxidase\n3. Karnofsky-Index \\> =70%\n4. At least one prior, ongoing or refractory severe infection and\u002For inflammatory complications requiring hospitalization despite drug intervention\n5. Written informed consent for adult patient, and assent for pediatric subjects seven years or older\n\nExclusion Criteria:\n\n1. Contraindication for leukapheresis (anaemia Hb \\\u003C8g\u002Fdl, cardiovascular instability, severe coagulopathy) or for administration of conditioning medication\n2. Female patients who are pregnant or lactating as determined by history and\u002For positive pregnancy test","ALL",{"count":56,"type":21},10,[58],"NA","This is a Phase I\u002FII clinical trial of gene therapy for treating Chronic Granulomatous Disease using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the TYF-CGD gene transfer clinical protocol.",[27],[62,63],"Chronic granulomatous disease","Lentiviral vector","2026-06-18",{"date":66,"type":38},"2026-06-22",{"date":68,"type":38},"2025-07-01",{"date":70,"type":21},"2029-12-31",{"name":72,"class":73},"Shenzhen Geno-Immune Medical Institute","OTHER",{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":78,"acronym":79,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":54,"minAge":81,"maxAge":82,"enrollmentInfo":83,"targetDuration":4,"studyType":22,"phases":84,"briefSummary":86,"conditions":87,"keywords":102,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":117,"lastUpdatePostDateStruct":118,"startDateStruct":120,"completionDateStruct":122,"leadSponsor":124,"locationsCount":46},"100614826","phase-2-hematopoietic-stem-cell-transplantation-hsct-for-common-variable-immunodeficiency-cvid-and-other-autoimmune-manifestations-of-primary-immune-regulatory-disorders-pird-100614826","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.","5 Years","40 Years",{"count":20,"type":21},[85],"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).",[88,89,90,91,92,93,94,95,96,97,98,99,100,101,27],"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",[103,104,105,106,107,108,109,110,111,112,91,113,27,92,93,114,115,96,97,116],"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)","2026-05-13",{"date":119,"type":38},"2026-05-15",{"date":121,"type":38},"2026-05-04",{"date":123,"type":21},"2031-02-01",{"name":125,"class":73},"Paul Szabolcs",{"id":127,"slug":128,"hasResults":11,"nctId":129,"briefTitle":130,"officialTitle":131,"acronym":4,"eligibilityCriteria":132,"healthyVolunteers":11,"sex":54,"minAge":133,"maxAge":134,"enrollmentInfo":135,"targetDuration":4,"studyType":22,"phases":137,"briefSummary":138,"conditions":139,"keywords":140,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":117,"lastUpdatePostDateStruct":144,"startDateStruct":146,"completionDateStruct":148,"leadSponsor":150,"locationsCount":46},"100474840","phase-1-allogeneic-hematopoietic-stem-cell-transplantation-for-chronic-granulomatous-disease-cgd-with-an-alemtuzumab-busulfan-and-tbi-based-conditioning-regimen-combined-with-cytokine-il-6---ifn-gamma-antagonists-100474840","NCT05463133","Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +\u002F- IFN-gamma) Antagonists","Phase I\u002FII Study Using Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +\u002F- IFN-gamma) Antagonists","* INCLUSION CRITERIA:\n\nIn order to be eligible to participate in this study, an individual must meet all the following criteria:\n\n* Must have the ability to comprehend and a willingness to sign the informed consent. For pediatric patients, must have a parent\u002Fguardian who can sign consent if the donor is a minor; assent will be obtained from minors as appropriate.\n* Must have confirmed diagnosis of CGD.\n* Must have sufficient complications from underlying disease to warrant undergoing transplantation (either a history of or ongoing inflammation\u002FCGD-related autoimmunity OR a CGD-related infection while on prophylaxis) OR have a Quartile 1 or 2 residual oxidase production level.\n* Ages 4 years-65 years.\n* HLA-matched family donor graft or an HLA-matched unrelated PBSC graft (10\u002F10 or 9\u002F10 mismatch) available.\n* Must be human immunodeficiency virus (HIV) negative.\n* When discharged from the hospital the participant must be able to stay within 1 hour s travel of the NIH for the first 3 months after transplantation.\n* Must have a family member or other designated care provider to assist with care during the post-transplant period when the patient is in the outpatient setting.\n* Must provide a durable power of attorney for health care decisions to an appropriate adult relative or guardian in accordance with NIH 200 'NIH Durable Power of Attorney for Health Care Decision Making.'\n* Females of child-bearing potential must agree to consistently use one form of contraception from 1 month prior to study entry and for at least 1 year post transplant. Male participants must agree to consistently use contraception for 1 year post transplant. Acceptable forms of contraception are:\n\n  * Contraceptive pills or patch, Norplant \\[Registered\\], Depo-Provera \\[Registered\\], or other FDA-approved contraceptive method.\n  * Male partner has previously undergone a vasectomy.\n  * Male participants will be advised to consistently use contraception throughout study participation and for 3 months post-transplant.\n* Stated willingness to comply with all study procedures and is available for protocol visits for the duration of the study when possible.\n* Patients who have a CRP of greater than 100 but otherwise meet inclusion criteria will be enrolled on the high-risk arm.\n\n  * CRP will be assessed no more than 7 weeks and no less than 6 weeks prior to anticipated transplant to determine on which arm the patient will be treated.\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n* Ejection fraction of less than 30% by echocardiography.\n* Forced expiratory volume (FEV1%) of less than 35% and\u002For an adjusted diffusing capacity of lung of carbon monoxide (adj DLCO) of less than 30%.\n* Transaminases \\>5x upper limit of normal based on the individual s clinical situation and at the discretion of the investigator.\n* Psychiatric disorder or mental deficiency severe enough as to make compliance with the HSCT treatment unlikely, and\u002For to make regulatorily and legally effective informed consent impossible.\n* Major anticipated illness or organ failure incompatible with survival from allogeneic peripheral blood stem cell (AlloPBSC) transplant.\n* Pregnant or lactating.\n* Uncontrolled seizure disorder per principal investigator (PI) discretion.\n* Individuals older than 65 years are excluded. It is known from standard transplantation that these individuals have a higher risk of morbidity and mortality related to transplantation. Given the investigational nature of this protocol, the risk-benefit ratio is not warranted to include these individuals at this time.\n* Active TB infection.\n* Any condition or circumstance that the PI feels would create difficulty in maintaining compliance with the requirements of this protocol.\n* Individuals who are not willing to submit their information as part of the alemtuzumab (Campath \\[Registered\\]) Distribution Program application or participants whom the Distribution Program committee has determined are not qualified to receive alemtuzumab.\n\nNOTE: Alemtuzumab (Campath-1H) (intravenous \\[IV\\] formulation) is no longer distributed commercially. To receive product, the physician must contact the program for the patient. If the patient is not willing to consent to submit their info (demographics, contact information, and rationale for use) to the program such that we can obtain the drug, then we cannot proceed with conditioning; therefore no transplant will occur on this protocol. http:\u002F\u002Fwww.campath.com\u002F","4 Years","65 Years",{"count":136,"type":21},50,[24,85],"Background:\n\nChronic granulomatous disease (CGD) affects the immune system. People with CGD are more likely to get infections. Drugs can help control infections, but these treatments can cause side effects including kidney failure and deafness. Stem cell transplants can cure CGD, but these don t always work.\n\nObjective:\n\nTo find out if a different drug treatment can improve the success rates of stem cell transplants in people with CGD.\n\nEligibility:\n\nPeople aged 4-65 years with CGD.\n\nDesign:\n\nParticipants will undergo screening. They will have a physical exam. They will have blood and urine tests and tests of their heart function and breathing. They will have imaging scans. They will have a bone marrow biopsy; a needle will be inserted into their hip to draw a sample of tissue from the bone.\n\nA tube called a catheter will be placed into a vein in the participant s chest. This catheter will remain in place for the transplant and recovery period. Blood for tests can be drawn from the catheter, and medications and the stem cells can be administered through it.\n\nParticipants will be in the hospital for either 10 or 21 days to receive 3 or 4 drugs before the transplant. They will get 2 doses of total body radiation on the same day.\n\nParticipants will receive donor stem cells through the catheter. They will remain in the hospital for 6 weeks afterward.\n\nParticipants will visit the clinic 2 to 3 times per week for 3 months after discharge.\n\nFollow-up visits will continue for 5 years.",[27],[141,142,143],"HLA Matched Related Donor","Matched Unrelated Donor","The National Marrow Donor Program",{"date":145,"type":38},"2026-05-14",{"date":147,"type":38},"2022-07-08",{"date":149,"type":21},"2032-12-31",{"name":44,"class":45}]