[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"gaucher-disease-type-1\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:gaucher-disease-type-1":56},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,42,65,76,87,113],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100053288","phase-1-a-clinical-trial-of-pr001-ly3884961-in-patients-with-peripheral-manifestations-of-gaucher-disease-proceed-100053288",false,"NCT05487599","A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)","An Open-label, Dose-Finding, Phase 1\u002F2 Study to Evaluate the Safety and Tolerability of a Single Intravenous Dose of LY3884961 in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)","Inclusion Criteria:\n\n1. Age greater or equal to 18 years at the time of informed consent.\n2. Bi-allelic pathogenic GBA1 variants must be centrally confirmed.\n3. On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening.\n4. Capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.\n5. Females and males will be eligible for this study. Men and women of childbearing potential must use a highly effective method of contraception consistently and correctly for the duration of the study, including the long-term follow-up.\n6. Patients must agree to abstain from blood, tissue and organ donation; and must agree to abstain from tissue and organ donation for the duration of the study, including long-term follow-up.\n\nExclusion Criteria:\n\n1. Clinically significant neurological signs and symptoms and\u002For behavioral disturbances.\n2. Active and progressive bone disease expected to require surgical treatment in the next 6 months.\n3. History of total splenectomy or planned total splenectomy during the first 18 months of the study. (Partial splenectomy not exclusionary).\n4. Splenomegaly \\> 10 MN as evaluated by centrally read abdominal magnetic resonance imaging (MRI)\n5. Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins.\n6. Thrombocytopenia with platelet count \\\u003C 40 × 10\\^3 per μL.\n7. Severe hyperlipidemia (triglycerides \\> 1,000 mg\u002FdL).\n8. Current diagnosis of unstable or clinically significant cardiovascular conditions based on Investigator assessment.\n9. History of certain cancers within 5 years of Screening.\n10. Concomitant disease, condition or treatment which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.\n11. Women of childbearing potential, pregnant (i.e., positive serum pregnancy result at Screening and\u002For Check-in) or breastfeeding or intending to become pregnant during the course of the trial.\n12. Use of any GD-related chaperone therapy within 4 weeks prior to Screening or expected need to initiate chaperone therapy during at least the first 18 months of the study.\n13. Any type of prior gene or cell therapy.\n14. Use of systemic immunosuppressant or steroid therapy other than protocol-specified immunosuppression.\n15. Participation in another therapeutic investigational drug or device study within 3 months or 5 half-lives of the study agent, whichever is longer.\n16. Have an anti-AAV9 antibody titer of \\>1:40 as determined by central laboratory.\n17. Clinically significant abnormalities in laboratory test results at Screening.\n18. Have any contraindications for MRI, including claustrophobia or the presence of contraindicated metal (ferromagnetic)implants\u002Fcardiac pacemaker.","ALL","18 Years",{"count":19,"type":20},15,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","Study J3Z-MC-OJAE is a Phase 1\u002F2, multicenter, open-label, dose-finding study of LY3884961 evaluating the safety and tolerability in adults with peripheral manifestations of GD.\n\nUp to 3 dose levels of LY3884961 will be assessed in 3 dose-finding cohorts of 3 patients. Following this, up to 6 patients may be enrolled in an expansion cohort.\n\nFor each enrolled patient, the study will be approximately 5 years in duration, including up to a 60-day screening period. During the first 18 months after dosing, subjects will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will be followed for an additional 42 months to monitor safety, immunogenicity, and selected biomarker and efficacy parameters.",[27,28],"Gaucher Disease","Gaucher Disease, Type 1","RECRUITING","2026-07-10",{"date":32,"type":33},"2026-07-13","ACTUAL",{"date":35,"type":33},"2022-12-20",{"date":37,"type":20},"2032-08-30",{"name":39,"class":40},"Prevail Therapeutics","INDUSTRY",9,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":48,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":21,"phases":52,"briefSummary":54,"conditions":55,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":57,"startDateStruct":58,"completionDateStruct":60,"leadSponsor":62,"locationsCount":64},"100054053","phase-3-a-gaucher-disease-gene-therapy-trial-with-flt201-100054053","NCT07223944","A Gaucher Disease Gene Therapy Trial With FLT201","A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1","GALILEO-3","Key Inclusion Criteria:\n\n* Aged ≥18 years at time of screening.\n* Clinical diagnosis of Gaucher disease type 1\n* Stable hemoglobin concentration at baseline\n* Stable platelet count at baseline\n* Receiving ERT or SRT without interruption for at least 2 years\n\nKey Exclusion Criteria:\n\n* Diagnosed or suspected Gaucher disease type 2 or type 3\n* Positive for AAVS3 neutralizing antibodies.\n* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study\n* Positive pregnancy test or lactating\n* History of hematopoietic stem cell transplant (HSCT)\u002Fbone marrow transplant or any solid organ transplant.\n* History of receiving any gene therapy or cell therapy.\n* History of total splenectomy. Note: Additional protocol defined Inclusion and Exclusion criteria apply",{"count":51,"type":20},45,[53],"PHASE3","This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study aims to confirm the efficacy and safety of FLT201 in this population after discontinuation of ERT\u002FSRT.",[56],"Gaucher Disease Type 1",{"date":32,"type":33},{"date":59,"type":33},"2026-04-07",{"date":61,"type":20},"2032-10-01",{"name":63,"class":40},"Spur Therapeutics",33,{"id":66,"slug":4,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":48,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":67,"targetDuration":4,"studyType":21,"phases":68,"briefSummary":54,"conditions":69,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":71,"startDateStruct":73,"completionDateStruct":74,"leadSponsor":75,"locationsCount":64},"100610158",{"count":51,"type":20},[53],[56],"2026-06-22",{"date":72,"type":33},"2026-06-23",{"date":59,"type":33},{"date":61,"type":20},{"name":63,"class":40},{"id":77,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":78,"targetDuration":4,"studyType":21,"phases":79,"briefSummary":25,"conditions":80,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":81,"lastUpdatePostDateStruct":82,"startDateStruct":84,"completionDateStruct":85,"leadSponsor":86,"locationsCount":41},"100476719",{"count":19,"type":20},[23,24],[27,28],"2026-05-19",{"date":83,"type":33},"2026-05-22",{"date":35,"type":33},{"date":37,"type":20},{"name":39,"class":40},{"id":88,"slug":89,"hasResults":11,"nctId":90,"briefTitle":91,"officialTitle":91,"acronym":4,"eligibilityCriteria":92,"healthyVolunteers":93,"sex":16,"minAge":94,"maxAge":95,"enrollmentInfo":96,"targetDuration":4,"studyType":98,"phases":4,"briefSummary":99,"conditions":100,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":102,"lastUpdatePostDateStruct":103,"startDateStruct":105,"completionDateStruct":107,"leadSponsor":109,"locationsCount":112},"100458741","prodromal-parkinsonian-features-in-gba1-mutation-carriers-100458741","NCT05253560","Prodromal Parkinsonian Features in GBA1 Mutation Carriers","Inclusion Criteria:\n\n* Willing to participate\n\nExclusion Criteria:\n\n* PD patients\n* Dementia",true,"40 Years","75 Years",{"count":97,"type":20},600,"OBSERVATIONAL","Objective of the trial. To define a sub-population which is at increased risk of developing Parkinson, beyond the fact of carrying Gaucher; in this sub-population the investigators shall conduct a comprehensive evaluation that includes a variety of non-invasive tests, whose purpose is to evaluate the state of the pre- Parkinson's disease signs, signs which can appear, even twenty years before the appearance of the disease, and also to compare them to a group of diagnosed Gaucher patients and a group of healthy people who are not carriers of Gaucher disease.\n\nA group of those carriers will be available for trial or for treatment, if there will be a medicine for the prevention of the development of Parkinson, obtainable.",[28,101],"Healthy","2026-04-23",{"date":104,"type":33},"2026-04-29",{"date":106,"type":33},"2017-05-16",{"date":108,"type":20},"2030-12-31",{"name":110,"class":111},"Shaare Zedek Medical Center","OTHER",1,{"id":114,"slug":115,"hasResults":11,"nctId":116,"briefTitle":117,"officialTitle":118,"acronym":119,"eligibilityCriteria":120,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":121,"enrollmentInfo":122,"targetDuration":4,"studyType":21,"phases":124,"briefSummary":125,"conditions":126,"keywords":127,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":130,"lastUpdatePostDateStruct":131,"startDateStruct":133,"completionDateStruct":135,"leadSponsor":137,"locationsCount":139},"100579018","phase-1-a-clinical-study-evaluating-ly-m001-injection-in-the-treatment-of-adult-patients-with-type-i-gaucher-disease-100579018","NCT06818838","A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease","A Multicenter, Open, Single-arm, Single-dose, Dose-escalation, and Expanded Phase I\u002FII Study Evaluating the Safety, Tolerability, and Efficacy of LY-M001 Injection in Adult Patients With Type I Gaucher Disease","GD","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 confirmed double mutations in the GBA1 allele through laboratory testing, and the glucocerebrosidase activity was reduced to less than 30% of the normal value(For example, the result of the dried blood spot (DBS) method is \\\u003C 1.19 μmol\u002FL\u002Fh), and meeting the standard clinical diagnosis criteria for GD1.\n4. Patients who meet a) or b) below:\n\n   1. Treated patients with Gaucher disease type I who had previously received enzyme replacement therapy (ERT) or substrate clearance therapy (SRT) with GD, were on stable medication, eluted 5 drugs for a half-life or more before administration, or were comprehensively judged to be stable by the investigator.\n   2. Newly treated or untreated GD1 patients who meet one or more of the following criteria at screening:\n\n      * Hemoglobin ≥80g\u002FL and less than the lower limit of normal;\n      * Platelets ≥40×10\\^9\u002FL and less than the lower limit of normal;\n      * Hepatomegaly;\n      * Splenomegaly.\n5. Negative pregnancy test for female subjects of childbearing potential (WOCBP). Notes: WOCBP is defined as the absence of postmenopausal status (continuous amenorrhea of at least 12 months with no identifiable cause other than menopause), and the absence of surgical (i.e., ovarian, salpingectomy, and\u002For hysterectomy) or Investigator-determined cause of permanent infertility due to other causes (e.g., lenticular hypoplasia) after menarche in female subjects.\n6. Subjects and their partners have no childbearing plans from the screening period to 6 months after the end of the study, and voluntarily adopt effective contraceptive measures (e.g., abstinence, condoms, etc.); subjects have no plans to donate sperm or eggs.\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 positive (Antibody titer \\> 1:40).\n2. Patients with clinically diagnosed Gaucher disease type II or III (GD2 or 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 GD as judged by the Investigator.\n5. Treatment or disposal of investigational drugs or investigational devices received in other clinical studies within 28 days prior to screening or within 5 half-lives (drugs only), whichever is older.\n6. Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins that meets, but is not limited to, any of the following at the time of screening:\n\n   * Progressive hepatomegaly larger than 3 times the normal volume.\n   * History of stage 2 or above liver fibrosis.\n   * AST, ALT, or TBIL are 1.5 times higher than ULN.\n   * A history of alcohol or drug abuse within the previous 2 years (defined as having consumed more than 14 standard units of alcohol per week \\[1 standard unit containing 14 g of alcohol, such as 360 mL beer, 45 mL spirits containing 40% or more alcohol, or 150 mL wine\\]).\n   * Hepatitis B surface antigen (HBsAg) positive and HBV deoxyribonucleic acid (HBV-DNA) positive (HBV-DNA\\>10\\^3 copy number \u002FmL); Or take hepatitis B drugs (such as interferon, lamivudine, adefovir and entecavir); Or antibodies to hepatitis C virus (HCV) and positive for hepatitis C virus RNA.\n7. Human immunodeficiency virus (HIV) antibody positive or Treponema pallidum antibody positive.\n8. Severe hyperlipidemia (triglycerides \\> 11.29mol\u002FL).\n9. Uncontrolled concomitant or infectious diseases (need to be determined by the investigator based on clinical practice).\n10. 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.\n11. 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.\n12. Clinically diagnosed or investigator-determined serious cardiovascular disease (such as heart failure ≥3 from the New York College of Cardiology \\[NYHA\\]).\n13. Hypersensitivity to any component of LY-M001 injection.\n14. Previous treatment with any type of gene therapy or cell therapy.\n15. Use of systemic immunosuppressive agents or steroid therapy other than those required by the protocol for prophylactic administration within 3 months prior to dosing.\n16. History of cancer within 5 years prior to screening, or currently active neoplastic disease, except for basal or squamous cell carcinoma of the skin or carcinoma in situ that has been definitively treated.\n17. Has received a live attenuated vaccine within 4 months prior to screening or plans to receive a live attenuated vaccine during the clinical trial.\n18. Other conditions that, in the opinion of the Investigator, make the subject unsuitable for the study.","60 Years",{"count":123,"type":20},12,[23,24],"Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate, Glucosylceramide (GlcCer), in macrophages in the liver, spleen, kidney, bone, lung, and even the brain, inducing their transformation into Gaucher cells whose cell cytoplasm presenting a characteristic \"crumpled tissue paper\" appearance, leading to pathological changes in involved tissues and organs.LY-M001 Injection is an rAAV8 vector gene therapy product. It can specifically transduce the target organ liver after a single intravenous administration and express the GCase protein in liver cells for a long period of time.",[56],[128,129],"anaemia","Gene therapy","2026-01-22",{"date":132,"type":33},"2026-01-26",{"date":134,"type":33},"2024-07-05",{"date":136,"type":20},"2031-07-30",{"name":138,"class":40},"Lingyi Biotech Co., Ltd.",3]