[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"gaucher-disease-type-i\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:gaucher-disease-type-i":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,41,52,77],{"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":4,"briefSummary":23,"conditions":24,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"100054061","oxidative-stress-and-inflammatory-biomarkers-in-gaucher-disease-100054061",false,"NCT02437396","Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease","Novel Inflammatory Biomarkers Complement 5A and Hepcidin in Patients With Gaucher Disease (GD)","Inclusion criteria:\n\n1. All participants must be 18 years or older.\n2. All enrollees must understand and cooperate with requirements of the study in the opinion of the investigators and must be able to provide written informed consent.\n3. Individuals with Gaucher disease who are medically stable for participation in study in the opinion of the investigator.\n4. GD subjects must be stable on a specific ERT and\u002For SRT therapy at a specific dose (for e.g. on a units\u002Fkg basis) for at least 2 years or be naïve to these therapies (no therapy for 2 years).\n5. GD1 patients, who have had a change in therapy i.e. a change in dose or switch from one drug to another, can be enrolled after at least 6 months have elapsed since the change and is considered stable in the opinion of the clinician providing care to the patient.\n6. All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study.\n\nExclusion Criteria:\n\n1. Medically unstable conditions in any group as determined by the investigators\n2. Concurrent disease; medical condition; or an extenuating circumstance that, in the opinion of the investigator, might compromise subject safety, study compliance, completion of the study, or the integrity of the data collected for the study.\n3. Females who are pregnant or lactating or of child-bearing age who are not using acceptable forms of contraception\n4. History of asthma that is presently being treated\n5. Subjects who cannot or are unwilling to have blood drawn\n6. Unable to adhere to study protocol for whatever reason","ALL","18 Years","75 Years",{"count":20,"type":21},34,"ESTIMATED","OBSERVATIONAL","The objective of this study is to evaluate oxidative stress and\u002For inflammation in patients with Gaucher disease type I using a series of biomarkers and correlate with measurements of currently used diagnostic biomarkers.",[25,26,27],"Gaucher Disease Type I","Oxidative Stress","Inflammation","RECRUITING","2026-07-10",{"date":31,"type":32},"2026-07-13","ACTUAL",{"date":34,"type":32},"2015-10",{"date":36,"type":21},"2026-10-30",{"name":38,"class":39},"University of Minnesota","OTHER",1,{"id":42,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":43,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":23,"conditions":44,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":45,"lastUpdatePostDateStruct":46,"startDateStruct":48,"completionDateStruct":49,"leadSponsor":51,"locationsCount":40},"100242677",{"count":20,"type":21},[25,26,27],"2026-03-16",{"date":47,"type":32},"2026-03-19",{"date":34,"type":32},{"date":50,"type":21},"2026-06-28",{"name":38,"class":39},{"id":53,"slug":54,"hasResults":11,"nctId":55,"briefTitle":56,"officialTitle":57,"acronym":58,"eligibilityCriteria":59,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":60,"enrollmentInfo":61,"targetDuration":4,"studyType":63,"phases":64,"briefSummary":66,"conditions":67,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":40},"100528558","a-study-of-the-safety-and-preliminary-efficacy-of-ly-m001-injection-in-the-treatment-of-adult-patients-with-gaucher-disease-type-i-100528558","NCT06162338","A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I","A Prospective, Single-center, Open-arm, Single-arm Study of the Safety and Preliminary Efficacy of Single Intravenous Infusion Administration of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I","LY-M001","Inclusion Criteria:\n\n1. Age ≥ 18 years and ≤ 60 years, male or female.\n2. The subjects should fully understand the purpose, nature and method of this study as well as possible adverse reactions, and sign the informed consent form (ICF) voluntarily.\n3. Patients with GD1 who have confirmed double mutations in the Gba1 allele by laboratory testing and meet the standard for clinical diagnosis of Gaucher disease (i.e., reduced GCase enzyme activity to less than 30% of normal values).\n4. The subjects were type I patients with Gaucher disease. Patients with type I Gaucher disease who had received specific treatment in the past required 5 half-lives of elution\n5. Negative pregnancy test for female subjects of childbearing potential 6.6.The subject and his\u002Fher partner have no plans to have children during the screening period and within 6 months after the end of the study, and voluntarily take effective contraceptive measures (such as abstinence, condom, etc.); and the subject had no plans to donate sperm or eggs.\n\n7.Subjects are not to donate blood during the study and for at least 1 year after the end of the study.\n\nExclusion Criteria:\n\n1. AAV8 neutralizing antibody is strongly positive.\n2. Patients with clinically suspected Gaucher disease type II (GD2) or Gaucher disease type III (GD3).\n3. Active and progressive bone disease that is expected to require surgical treatment within the next 6 months.\n4. Subject has idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenic purpura (TTP), thrombocytopenia, anemia, hepatomegaly, splenomegaly, and\u002For osteoporosis unrelated to Gaucher disease as judged by the investigator.\n5. Treatment or disposition of an investigational drug or investigational device for another clinical investigation within 28 days or 5 half-lives (only for drugs), whichever is longer, prior to Screening.\n6. Evidence of clinically significant liver disease, fragile liver, or history of hepatotoxin exposure, meeting at screening, but not limited to, any of the following:\n\n   * Progressive hepatomegaly and greater than 3 times normal volume.\n   * History of stage 2 or greater liver fibrosis.\n   * AST, ALT or TBIL greater than 1.5 times the upper limit of normal (ULN).\n   * History of alcohol or drug abuse within the previous 2 years.\n   * Hepatitis B surface antigen (HBsAg) positive, and hepatitis B virus DNA positive (HBV-DNA \\> 103 copies\u002FmL); or taking hepatitis B virus drugs (such as interferon, lamivudine, adefovir and entecavir); or hepatitis C virus (HCV) antibody positive.\n7. Human immunodeficiency virus (HIV) antibody positive or Treponema pallidum antibody positive.\n8. Severe hyperlipidemia (triglycerides \\> 1000 mg\u002FdL).\n9. Uncontrolled concomitant disease or infectious disease (need to be judged by the investigator based on clinical practice).\n10. Subject had undergone splenectomy and were scheduled to undergo splenectomy during the study period.\n11. Karnofsky score (KPS) \\\u003C 70.\n12. The subject has received or plans to receive bone marrow transplantation, hematopoietic stem cell transplantation and\u002For major organ transplantation, including but not limited to liver transplantation, kidney transplantation, etc.\n13. Subject has received erythropoietin, transfusion, or red blood cell transfusion within 3 months prior to screening ;or platelet transfusion within 1 month prior to screening.\n14. Clinically diagnosed or significant cardiovascular disease as judged by the investigator (e.g., New York Heart Association \\[NYHA\\] class ≥ 3 heart failure).\n15. Hypersensitivity to any component of LY-M001 Injection.\n16. Previous treatment with any type of gene therapy or cell therapy.\n17. Use of systemic immunosuppressive agents or steroid therapy other than those required by the protocol for prophylactic administration within 3 months prior to dosing.\n18. History of cancer within 5 years of screening, except for completely resected non-melanoma skin cancer, non-metastatic prostate cancer, and completely treated ductal carcinoma in situ.\n19. Has received a live attenuated vaccine within 4 months prior to screening or plans to receive a live attenuated vaccine during the clinical trial.\n20. Other conditions that, in the opinion of the investigator, make the subject unsuitable for the study.","60 Years",{"count":62,"type":21},3,"INTERVENTIONAL",[65],"NA","This is a prospective single-center, open, single-arm, single-dose intravenous infusion study to evaluate the safety and initial efficacy, pharmacodynamic characteristics, immunogenicity, biodistribution, and viral shedding of LY-M001 injection.This study mainly includes the main study stage and the long-term follow-up study stage.",[25],"2025-02-11",{"date":70,"type":32},"2025-02-14",{"date":72,"type":32},"2023-11-15",{"date":74,"type":21},"2031-12-17",{"name":76,"class":39},"He Huang",{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":82,"acronym":83,"eligibilityCriteria":84,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":85,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":87,"conditions":88,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":91,"lastUpdatePostDateStruct":92,"startDateStruct":94,"completionDateStruct":96,"leadSponsor":98,"locationsCount":100},"100304243","gaucherite---a-study-to-stratify-gaucher-disease-100304243","NCT03240653","Gaucherite - A Study to Stratify Gaucher Disease","Predictive Measures to Stratify Clinical Outcomes in Children and Adults With Gaucher Disease and Responses to Specific Therapies","Gaucherite","Inclusion Criteria:\n\nEach patient must meet all of the following criteria to be enrolled in this study:\n\n1. Confirmed biochemical diagnosis of Type I, Type II or Type III Gaucher disease\n2. Written Ethics Committee (EC) approved informed consent obtained from the patient, or patient's parent or legal guardian and patient assent if appropriate\n3. Male or Female patients, no age limitation\n4. Willing and able to comply with study schedule and procedures\n5. Deceased patients for whom the EC determines that patient data can be collected without a new consent from the patient\n\nExclusion Criteria:\n\nPatients meeting any of the following criteria will be excluded from the study:\n\n1. Unrelated co-morbid condition limiting life expectancy to less than 6 months\n2. Patient or if applicable, parent or legal guardian is unable to comprehend, sign and date the EC approved informed consent form and patient assent as appropriate\n3. If determined unsuitable for the study by the investigator",{"count":86,"type":21},250,"The purpose of this research is to review data already collected and to collect new data from adults and children in England with Gaucher Disease to determine clinical factors which predict severity and response to therapy of Gaucher disease especially in the areas of bone, cancer and brain conditions.",[89,90],"Gaucher Disease, Type I","Gaucher Disease, Type III","2024-08-05",{"date":93,"type":32},"2024-08-06",{"date":95,"type":32},"2014-01-01",{"date":97,"type":21},"2028-12-31",{"name":99,"class":39},"Cambridge University Hospitals NHS Foundation Trust",8]