[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Shanghai Vitalgen BioPharma Co., Ltd.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":118},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,41,67,95],{"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":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":4},"100639064","phase-1-a-phase-iii-clinical-study-to-evaluate-the-safety-and-efficacy-of-vgn-r08b-in-patients-with-type-iii-gauchers-disease-100639064",false,"NCT07603050","A Phase I\u002FII Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease","A Phase I\u002FII Clinical Study to Evaluate the Tolerance, Safety and Efficacy of VGN-R08b Intracerebroventricular Injection in Patients With Type III Gaucher's Disease","Inclusion Criteria:\n\n1. The signing of the informed consent form should be completed when the subject is at least 2 years old but less than 18 years old.\n2. The subject has a documented medical history of Gaucher disease confirmed by GCase enzyme activity testing, and has a double GBA1 gene mutation.\n3. According to the investigator's assessment, the neurological signs and\u002For symptoms are consistent with type III Gaucher disease.\n4. At the time of enrollment, the subject has horizontal eye movement disorders (including gaze paralysis, or delayed or absent saccades), but there is no severe motor dysfunction resulting in bedridden status.\n5. The subject is currently receiving substrate reduction therapy (SRT) and\u002For high-dose ambroxol for Gaucher disease treatment. The subject is required to have been on stable treatment for at least 2 months before enrollment and the investigator determines that the treatment is ineffective for neurological symptoms, or is willing to discontinue the treatment at the time of enrollment (discontinuation 1 week before administration).\n6. The subject is currently receiving and willing to continue stable peripheral treatment (including imiglucerase or other ERT, or SRT), and the peripheral symptoms of Gaucher disease are stable at the time of screening, that is, all of the following conditions are met: hemoglobin level ≥ 11.0 g\u002FdL (female) or ≥ 12.0 g\u002FdL (male), platelet count ≥ 100×109\u002FL, spleen volume \\\u003C 10 times the normal value (MN), liver volume \\\u003C 1.5 MN, and no bone crisis or asymptomatic bone disease (such as bone necrosis and\u002For pathological fractures causing bone pain) within 3 months before screening.\n7. (Applicable) Male and female subjects with reproductive potential must continue to use an effective contraceptive method (including abstinence) correctly from the screening period until at least 1 year after the start of treatment, and not donate sperm or eggs.\n8. The subject (applicable) and their parents\u002Fguardians must understand the trial information, purpose and risks described in the informed consent form, and authorize the use of the subject's health information and provide an informed consent form with the signature and date of signing.\n9. The subject (applicable) and their parents\u002Fguardians are willing to participate in the study as information providers, providing the subject's health status, cognition and physical ability (including providing information for rating scales).\n\nExclusion Criteria:\n\n1. There are other serious neurological disorders that may cause symptoms of Gaucher disease or interfere with the research objectives;\n2. There are severe internal organ damages caused by Gaucher disease, which, after evaluation by the researchers, are considered to pose unacceptable risks to the subjects, or interfere with the subjects' research compliance, or interfere with the execution of the trial;\n3. Long-term ventilation or long-term nasogastric feeding (long-term ventilation is defined as: requiring tracheotomy for respiratory assistance, or continuous 14 days or more of non-invasive respiratory assistance for ≥ 16 hours per day, excluding acute reversible diseases that require assisted ventilation and perioperative ventilation. Long-term nasogastric feeding refers to the use of a nasogastric tube for feeding due to severe loss of swallowing function);\n4. There are severe immunodeficiencies or autoimmune diseases;\n5. There is active infection (including viral infections such as HIV, HBV, HCV or syphilis);\n\n   The following medication and treatment situations exist:\n6. Currently using drugs, herbs, or over-the-counter medications that have strong inhibitory or inducing effects on CYP3A4 or P-gp;\n7. Having received bone marrow or organ transplantation, or any gene or cell therapy;\n8. Having undergone immunization (live vaccines) within 4 weeks;\n9. Undergoing systemic immunosuppressive therapy or corticosteroid therapy other than that required by the protocol (local preparations for skin diseases can be used);","ALL","2 Years","18 Years",{"count":20,"type":21},12,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","A phase I\u002FII clinical study to evaluate the tolerance, safety and efficacy of VGN-R08b intracerebroventricular injection in patients with type III Gaucher's disease",[28],"Gaucher Disease Type 3","NOT_YET_RECRUITING","2026-05-20",{"date":32,"type":33},"2026-05-22","ACTUAL",{"date":35,"type":21},"2026-05-14",{"date":37,"type":21},"2032-08-16",{"name":39,"class":40},"Shanghai Vitalgen BioPharma Co., Ltd.","INDUSTRY",{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":4,"eligibilityCriteria":47,"healthyVolunteers":11,"sex":16,"minAge":18,"maxAge":48,"enrollmentInfo":49,"targetDuration":4,"studyType":22,"phases":51,"briefSummary":52,"conditions":53,"keywords":55,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":59,"lastUpdatePostDateStruct":60,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":66},"100637306","phase-1-a-safety-study-of-contralateral-eye-dosing-of-vgr-r01-in-participants-with-biettis-crystalline-dystrophy-bcd-100637306","NCT07586306","A Safety Study of Contralateral Eye Dosing of VGR-R01 in Participants With Bietti's Crystalline Dystrophy (BCD)","A Clinical Study Evaluating Subretinal Injection of VGR-R01 in the Contralateral Eye of Participants With Bietti's Crystalline Dystrophy Who Had Received VGR-R01 Administration","Key Inclusion Criteria:\n\n1. Participants received VGR-R01 administration in the VGR-R01-001 or VGR-R01-101 studies.\n2. Able to provide informed consent and comply with requirements of the study;\n3. Hand Motion ≤ BCVA ≤ 75 ETDRS letters in the second treated eye.\n\nKey Exclusion Criteria:\n\n1. Have insufficient viable retinal photoreceptor cells based on investigator's decision;\n2. Have current ocular or periocular infections, or endophthalmitis;\n3. Have any significant ocular disease\u002Fdisorder other than BCD, including age-related macular degeneration, diabetic retinopathy, optic neuropathy, significant lens opacity, glaucoma, uveitis, retinal detachment, etc;\n4. Have intraocular surgery history except cataract surgery in the study eye;\n5. Have or potentially require of systemic medications that may cause eye injure;\n6. Have contraindications for corticosteroids or immunosuppressant;\n7. Abnormal coagulation function or other clinically significant abnormal laboratory results;\n8. Have malignancies or history of malignancies;\n9. History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion\u002FExclusion criteria may apply.","69 Years",{"count":50,"type":21},15,[24,25],"This is a multi-centre, single- arm, non-randomized, open-label phase 1\u002F2 clinical trial which enables dosing of the fellow eyes of patients who received VGR-R01 administration in previous studies.",[54],"Bietti Crystalline Dystrophy",[56,57,58],"VGR-R01","CYP4V2","gene therapy","2026-05-12",{"date":35,"type":33},{"date":62,"type":21},"2026-06-01",{"date":64,"type":21},"2031-07-31",{"name":39,"class":40},2,{"id":68,"slug":69,"hasResults":11,"nctId":70,"briefTitle":71,"officialTitle":72,"acronym":4,"eligibilityCriteria":73,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":74,"enrollmentInfo":75,"targetDuration":4,"studyType":22,"phases":76,"briefSummary":77,"conditions":78,"keywords":81,"overallStatus":85,"whyStopped":4,"lastUpdateSubmitDate":86,"lastUpdatePostDateStruct":87,"startDateStruct":89,"completionDateStruct":91,"leadSponsor":93,"locationsCount":94},"100532826","phase-1-a-study-to-evaluate-the-tolerability-safety-and-efficacy-of-vgm-r02b-100532826","NCT06217861","A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b","An Open-Label, Dose-Escalation and Dose-Expansion Phase I Clinical Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b in Patients With Glutaric Acidemia Type I","Inclusion Criteria:\n\n1. Subjects must be ≤ 6 years;\n2. History of diagnosis of GA-I, and confirmed by gene mutation analysis with biallelic GCDH mutation;\n3. At the time of screening, there was one of the obvious neurological manifestations associated with the following diseases, including macrocephaly, dystonia, and motor\u002Fintellectual development Poor fertility, epilepsy, abnormal EEG;\n4. Those who are receiving standard treatment recommended by the guidelines and whose symptoms remain poorly controlled by the investigator;\n5. Plasma GA and 3-OHGA levels were higher than the normal range during screening;\n\nExclusion Criteria:\n\n1. Participation in gene therapy or stem cell transduction therapy at any time prior to screening for this trial or participation in any other clinical trial within 3 months prior to screening;\n2. Recurrent seizures that are not suitable for surgery, based on Investigator judgment;\n3. Current severe liver or kidney or cardiovascular disease or coagulation dysfunction, autoimmune deficiency, or uncontrolled autoimmune disease or need immunosuppressive long-term treatment, poorly controlled diabetes (HBA1C ≥7% at screening) or high blood pressure;\n4. Active viral infection (includes HIV or serology positive for hepatitis B or C or syphilis);\n5. Presence or history of malignancy;\n6. Received systemic immunosuppressive therapy within 3 months prior to screening;\n7. Received vaccine within 4 weeks prior to administration or plan to receive vaccine within 1 year after administration;\n8. Plan to receive surgery during the study;\n9. Current using medications including, drugs, herbal or OTC medications that strongly inhibit or induce CYP3A4 or P-glycoprotein (P-gp), e.g., metoclopramide, grapefruit juice, ketoconazole, erythromycin;\n10. Abnormal brain structure, not suitable for lateral ventricle administration;\n11. Abnormal laboratory test results, which are judged by the investigator not suitable for surgery;\n12. History of systemic hypersensitivity reaction to investigational product, the excipients contained in the formulation, or prophylactic immunosuppressant;\n13. Contraindicated use of corticosteroids and sirolimus;\n14. Contraindicated with general anesthesia or sedation;\n15. As judged by the investigator, unable to perform lateral ventricle puncture or Ommaya capsule implantation or lumbar puncture;\n16. Unable to perform CT or MRI;\n17. Poor compliance;\n18. Any other situation where, judged by the investigator, the subject is not suitable for participating in this study.","6 Years",{"count":20,"type":21},[24],"Phase I, open-label, single-arm, single-dose, trial of VGM-R02b (gene replacement therapy) in patients with Glutaric Acidemia Type I (GA-I) who meet enrollment criteria and are genetically confirmed by GCDH gene mutation. 1 to 3 patients aged≤ 6 years at the time of screening will be enrolled in each dose group in the dose escalation part. In the dose expansion part, the sample size will be statistically calculated and adjusted according to the efficacy and safety data in the dose escalation part.",[79,80],"Glutaric Acidemia Type I","Glutaric Aciduria Type I",[82,58,83,84],"AAV","ICV","GCDH","RECRUITING","2026-05-09",{"date":88,"type":33},"2026-05-13",{"date":90,"type":33},"2024-04-29",{"date":92,"type":21},"2027-08",{"name":39,"class":40},1,{"id":96,"slug":97,"hasResults":11,"nctId":98,"briefTitle":99,"officialTitle":100,"acronym":4,"eligibilityCriteria":101,"healthyVolunteers":11,"sex":16,"minAge":102,"maxAge":103,"enrollmentInfo":104,"targetDuration":4,"studyType":22,"phases":106,"briefSummary":107,"conditions":108,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":94},"100624796","phase-1-a-trial-to-evaluate-safety-and-efficacy-of-a-product-named-vgn-r08b-in-parkinsons-disease-patients-with-gba1-mutations-100624796","NCT07414290","A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R08b in Parkinson's Disease Patients With GBA1 Mutations","A Phase I\u002FII Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of VGN-R08b Intra-cerebroventricular Injection in Parkinson's Disease Patients With GBA1 Mutations","Inclusion Criteria:\n\n* Subjects must meet all the following inclusion criteria:\n\n  1. Male or female, aged 30 to 70 years (inclusive) at the time of signing the informed consent form.\n  2. Documented GBA1-mutant Parkinson's disease, confirmed by medical history: meeting the International Parkinson and Movement Disorder Society (MDS) diagnostic criteria for idiopathic Parkinson's disease, with the presence of at least one pathogenic GBA1 gene mutation (confirmed by investigator interpretation).\n  3. Glucocerebrosidase (GCase) enzyme activity below the normal range, as measured from past or screening dried blood spot tests.\n  4. Hoehn-Yahr stage of 3 to 4 in the \"OFF\" state, an MDS-UPDRS Part III (motor examination) score ≥33 points in the \"OFF\" state, and the ability to walk without relying on a walker or wheelchair.\n  5. Montreal Cognitive Assessment (MoCA) score meeting the following criteria: \\>13 (for ≤6 years of education), \\>15 (for 7-12 years of education), or \\>16 (for \\>12 years of education) .\n  6. On an optimized levodopa regimen at screening (defined as a regimen optimized with at least a combination of levodopa preparations plus a dopamine agonist or MAO-B inhibitor, with levodopa administered ≥3 times per day and at a total daily dose of ≥300 mg), yet still experiencing suboptimal symptom control or significant \"wearing-off\" (as evidenced by a diary documenting a daily \"OFF\" time of ≥2.5 hours for three consecutive days during screening).\n  7. Stable Parkinson's disease symptoms and stable optimized anti-Parkinson's medication regimen for ≥4 weeks prior to screening; patients with GD-PD receiving Gaucher disease (GD) therapy must have been on stable enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 3 months prior to screening.\n  8. Men and women of childbearing potential must agree to consistently and correctly use a highly effective method of contraception from the screening period until at least 1 year after dosing.\n  9. Men must agree not to donate sperm, and women must agree not to donate eggs, from the screening period until at least 1 year after dosing.\n  10. The patient and\u002For the patient's legal guardian demonstrates understanding of the trial information, purpose, and risks described in the informed consent form, and is able to authorize the use of the patient's health information by providing a signed and dated informed consent form.\n  11. The patient has a reliable study partner (e.g., family member, friend, caregiver) who is willing and able to assist with study visits when needed, and to help provide information regarding the patient's health status, and cognitive and physical abilities (including providing input for rating scales).\n\nExclusion Criteria:\n\n* Subject has any of the following diseases or disease history\n\n  1. Patients with atypical or secondary parkinsonian syndromes, including but not limited to those caused by trauma, brain tumors, infections, cerebrovascular diseases, or other neurological disorders; or symptoms confirmed by the investigator to be induced by drugs, chemicals, or toxins; or those with other serious neurological conditions deemed by the investigator to significantly compromise the safety and efficacy evaluation of the investigational drug.\n  2. Patients with active infections (including viral infections such as HBV, HCV, or syphilis) or a history of severe infections within 12 weeks prior to screening (e.g., pneumonia, sepsis, or central nervous system infections such as meningitis or encephalitis).\n  3. Patients with severe liver disease, severe immunodeficiency, or autoimmune diseases within 6 months prior to screening, or those requiring long-term immunosuppressive therapy.\n  4. Patients with poorly controlled diabetes or hypertension, judged by the investigator as unsuitable for dosing or likely to substantially impact the efficacy and safety analysis of the investigational drug.\n  5. Patients with a history of stroke or transient ischemic attack (TIA), unstable angina, myocardial infarction, chronic heart failure (NYHA Class III or IV), or clinically significant conduction abnormalities (e.g., unstable atrial fibrillation) within 1 year prior to screening.\n  6. Patients with a history of epileptic seizures or unexplained coma, deemed unsuitable by the investigator for participation in the trial.\n  7. Patients with a history of severe allergic reactions, or hypersensitivity to any inactive ingredient of the investigational drug or to immunosuppressants required by the trial protocol.\n  8. Patients with contraindications to corticosteroids or sirolimus, including but not limited to osteoporosis with vertebral fractures within 1 year prior to screening, poorly controlled hyperlipidemia or hypercholesterolemia, renal insufficiency, or interstitial lung disease.\n  9. Patients with newly diagnosed or unstable psychiatric disorders within 1 year prior to screening that may interfere with trial procedures and evaluations, including confusion, severe depression (HAMD score \\>35), or suicidal\u002Fself-harm tendencies.\n  10. Patients with a history of malignancy within 3 years prior to screening, except for completely resected non-melanoma skin cancer, non-metastatic prostate cancer, or fully cured carcinoma in situ that has remained stable for at least 6 months.\n  11. Patients with any other contraindications deemed by the investigator to potentially affect trial-related procedures, including lumbar puncture or intracerebral injection, such as spinal disorders, bleeding diathesis, clinically significant coagulation dysfunction, thrombocytopenia, or elevated intracranial pressure.","30 Years","70 Years",{"count":105,"type":21},17,[24,25],"A Phase I\u002FII Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of VGN-R08b Intra-cerebroventricular injection in Parkinson's Disease Patients with GBA1 Mutations",[109],"Parkinson Disease (PD)","2026-04-30",{"date":112,"type":33},"2026-05-01",{"date":114,"type":21},"2026-05-25",{"date":116,"type":21},"2031-09-01",{"name":39,"class":40},""]