[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Lingyi Biotech Co., Ltd.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":72},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,45],{"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":26,"conditions":27,"keywords":29,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100642688","phase-1-phase-iii-clinical-study-to-evaluate-the-safety-tolerability-and-efficacy-of-ly-m003-injection-in-adult-patients-with-wilsons-disease-100642688",false,"NCT07641140","Phase I\u002FII Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease","A Multicenter, Open, Single-arm, Single-dose, Phase I\u002FII Study Evaluating the Safety, Tolerability, and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease","Inclusion Criteria:\n\n1. The subject fully comprehends the purpose, design, methods and possible adverse events of the study, agrees to participate voluntarily and signs the informed consent form (ICF).\n2. Patients with confirmed diagnosis of Wilson's disease (WD).\n3. Subjects with Wilson's disease (WD) confirmed by laboratory testing to have biallelic ATP7B gene mutation or deletion.\n4. The subjects are treated patients with Wilson's disease (WD) who have received standard therapy (e.g., D-penicillamine or zinc acetate) continuously for at least 6 months prior to screening.\n5. Subjects have maintained a low-copper diet for at least 6 consecutive months prior to screening and will continue this dietary restriction throughout the study.\n6. Subjects must agree to refrain from donating blood, organs, tissues or cells at any time after treatment.\n7. Female subjects of childbearing potential (WOCBP) must have a negative pregnancy test.\n8. Subjects and their partners must have no plans for pregnancy from screening through 6 months after study completion, and will voluntarily use effective contraception (e.g., abstinence, condoms). Subjects shall not plan to donate sperm or ova.\n\nExclusion Criteria:\n\n1. AAV8 neutralizing antibody titer \\> 1:10 .\n2. History of active gastrointestinal bleeding within the past 3 months.\n3. Decompensated liver cirrhosis or advanced liver disease presenting with portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy, etc.\n4. Subjects with other concomitant liver diseases as judged by the investigator, including autoimmune hepatitis, alcoholic liver disease, primary biliary cholangitis, primary sclerosing cholangitis, and\u002For drug- or toxin-induced liver disease.\n5. Subjects with severe hypersplenism complicated and requiring splenectomy as assessed by the investigator.\n6. Model for End-Stage Liver Disease (MELD) score \\> 13.\n7. Other disorders of copper metabolism, such as chronic cholestatic liver diseases, disorders of glycosylation, copper metabolism disorders, etc.\n8. A history of non-compliance with copper chelators or zinc agents as assessed by the investigator within 6 months prior to screening.\n9. Previously treated WD subjects with ALT and\u002For AST levels more than 5 times the upper limit of normal (ULN).\n10. Subjects with severe neurological deficits or impairments that, in the investigator's judgment, compromise their safety and\u002For ability to participate in the study.\n11. Hemoglobin \\\u003C 90g\u002FL.\n12. Subjects with positive hepatitis B surface antigen (HBsAg), positive hepatitis C virus (HCV) antibody, positive human immunodeficiency virus (HIV) antibody or positive treponema pallidum antibody.\n13. Subjects with end-stage renal disease on dialysis (Chronic Kidney Disease Stage 3 and above), or creatinine clearance \\\u003C 60 mL\u002Fmin.\n14. Severe hyperlipidemia (triglycerides \\>1000 mg\u002FdL);\n15. Subjects who have received or plan to undergo bone marrow transplantation, hematopoietic stem cell transplantation and\u002For major organ transplantation, including but not limited to liver transplantation and renal transplantation.\n16. Subjects with clinically diagnosed severe cardiovascular diseases or those deemed by the investigator to have such conditions (e.g., New York Heart Association \\[NYHA\\] heart failure classification ≥ Class 3).\n17. Subjects with uncontrolled concomitant diseases or infectious diseases as assessed by the investigator.\n18. Subjects who are allergic to any ingredient of LY-M003 Injection.\n19. Prior receipt of any type of gene therapy or cell therapy.\n20. Use of systemic immunosuppressants or steroids within 3 months prior to administration (except for prophylactic immunosuppressive therapy specified in the protocol).\n21. History of cancer within 5 years prior to screening, excluding completely resected non-melanoma skin cancer, non-metastatic prostate cancer and fully cured ductal carcinoma in situ.\n22. Received live attenuated vaccines within 4 months prior to screening, or planned to receive such vaccines during the clinical trial.\n23. Received treatment or intervention with other investigational drugs or study devices within 28 days or 5 half-lives (for drugs only) prior to screening, whichever is longer.\n24. Pregnant women (or women planning pregnancy) or breastfeeding women.\n25. Other conditions that, in the investigator's opinion, render the subject ineligible for study participation.","ALL","18 Years","60 Years",{"count":20,"type":21},18,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This is a multicenter, open-label, single-arm, single-dose Phase I\u002FII clinical study. It aims to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-M003 Injection in patients with Wilson's Disease (WD).",[28],"Wilson's Disease",[28,30,31],"LY-M003 Injection","Gene Therapy","NOT_YET_RECRUITING","2026-06-30",{"date":35,"type":36},"2026-07-02","ACTUAL",{"date":38,"type":21},"2026-06-15",{"date":40,"type":21},"2032-12-30",{"name":42,"class":43},"Lingyi Biotech Co., Ltd.","INDUSTRY",1,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":51,"eligibilityCriteria":52,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":53,"targetDuration":4,"studyType":22,"phases":55,"briefSummary":56,"conditions":57,"keywords":59,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":64,"startDateStruct":66,"completionDateStruct":68,"leadSponsor":70,"locationsCount":71},"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.",{"count":54,"type":21},12,[24,25],"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.",[58],"Gaucher Disease Type 1",[60,61],"anaemia","Gene therapy","RECRUITING","2026-01-22",{"date":65,"type":36},"2026-01-26",{"date":67,"type":36},"2024-07-05",{"date":69,"type":21},"2031-07-30",{"name":42,"class":43},3,""]