[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"YolTech Therapeutics Co., Ltd\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":100},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,55,77],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":23,"conditions":24,"keywords":27,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":43,"lastUpdatePostDateStruct":44,"startDateStruct":47,"completionDateStruct":49,"leadSponsor":51,"locationsCount":54},"100652573","phase-1-phase-i-exploratory-study-of-yolt-203-in-patients-with-primary-hyperoxaluria-type-1-ph1-100652573",false,"NCT07776626","Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1)","Inclusion Criteria:\n\nSubjects must meet ALL of the following criteria to be eligible for participation in this study:\n\nAge ≥ 6 years at the time of signing the informed consent form. Documented confirmation of Primary Hyperoxaluria Type 1 (PH1) diagnosis via genetic analysis prior to enrollment.\n\nMean 24-hour urinary oxalate excretion level ≥ 0.7 mmol\u002F24 h\u002F1.73 m² obtained from at least two valid 24-hour urine collections.\n\nIf the subject is receiving vitamin B6, the stable treatment regimen must have been maintained for a minimum of 90 days prior to enrollment, and the subject is willing to continue this regimen from administration of study drug through the end of the study.\n\nThe subject is capable of understanding, willing, and able to comply with study requirements, and provides written informed consent. For subjects below the legal age of consent, a legal guardian must sign the informed consent form, and the subject shall provide assent in accordance with local and national requirements.\n\nExclusion Criteria:\n\n* Subjects with any of the following conditions will be excluded:\n\nPatients who respond well to vitamin B6 (pyridoxine) treatment, with urinary oxalate excretion returning to normal after treatment.\n\nClinical evidence of extrarenal systemic oxalosis.\n\nAny of the following laboratory test results observed at screening:\n\n1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 2 × upper limit of normal (ULN).\n2. Total bilirubin \\> 1.5 × ULN. Subjects diagnosed with Gilbert syndrome whose total bilirubin \\\u003C 2 × ULN may be enrolled.\n3. International normalized ratio (INR) \\> 1.5 (subjects taking oral anticoagulants such as warfarin with INR \\\u003C 3.5 are allowed to participate).\n\nKnown history of HIV infection or evidence of HIV infection (positive HIV antibody); active or chronic hepatitis C virus (HCV) infection (positive HCV antibody and positive HCV RNA polymerase chain reaction) or hepatitis B virus (HBV) infection (positive hepatitis B surface antigen \\[HBsAg\\]).\n\nEstimated glomerular filtration rate (eGFR) \\\u003C 45 mL\u002Fmin\u002F1.73 m² at screening (for subjects ≥ 18 years old: calculated using the 2021 Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation; for subjects \\\u003C 18 years old: calculated using the Schwartz bedside equation).\n\nReceiving dialysis treatment (peritoneal dialysis \\[PD\\] and\u002For hemodialysis \\[HD\\]) or expected to require dialysis treatment during the study period.\n\nReceived an investigational drug within 30 days or 5 half-lives (whichever is longer) prior to administration of the study drug, or participated in follow-up of another clinical study before enrollment.\n\nPrior receipt of gene editing therapy, or receipt of small interfering ribonucleic acid (siRNA) therapy within the following timeframes (based on drug half-life and dosing frequency). RNAi therapy is prohibited unless administered as rescue therapy:\n\ni. Lumasiran: less than 15 months from the last dose to administration of the study drug.\n\nii. Nedosiran: less than 150 days from the last dose to administration of the study drug.\n\nHistory of kidney or liver transplantation, or expected to require liver and\u002For kidney transplantation during the study period.\n\nOther medical conditions or comorbidities that, in the Investigator's judgment, may interfere with study compliance or data interpretation.\n\nHistory of multiple drug allergies or hypersensitivity reactions to oligonucleotides or lipid nanoparticles (LNPs).\n\nUnwilling to comply with contraception requirements throughout the entire study participation period until 6 months after the end of the study.\n\nFemale subjects who are pregnant, planning to become pregnant, or breastfeeding.\n\nUnwilling or unable to limit alcohol intake throughout the study period. Daily alcohol intake exceeding 2 standard units during the study (1 standard unit: approximately 125 mL wine = approximately 29 mL spirits = approximately 284 mL beer).\n\nHistory of alcohol abuse or substance abuse within 12 months prior to screening as judged by the Investigator.\n\nInability to adhere to standard supportive care (adequate water intake, potassium citrate, dietary requirements, etc.).","ALL","6 Years",{"count":18,"type":19},9,"ESTIMATED","INTERVENTIONAL",[22],"PHASE1","This is a single-arm, open-label, single-dose dose-escalation study. It aims to evaluate the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 (PH1), and to preliminarily assess the effect of a single administration of YOLT-203 on 24-hour urinary oxalate excretion.\n\nThe clinical trial will first evaluate the safety and tolerability in adult patients. After the safety profile is preliminarily confirmed, the trial will be sequentially conducted in adolescents, older children, and younger children.",[25,26],"Primary Hyperoxaluria Type 1","PH1",[25,28,29,30,31,32,33,34,35,36,37,38,39,40,41],"PH 1","yoltech","yolt203","primary hyperoxaluria","recurrent kidney stones","rare kidney stones","YOLT-203","genetic kidney stones","gene editing","gene therapy","AGXT gene","CRISPR-Cas9","Alanine Glyoxylate Aminotransferase Gene","ph1","RECRUITING","2026-08-17",{"date":45,"type":46},"2026-08-20","ACTUAL",{"date":48,"type":19},"2026-08",{"date":50,"type":19},"2028-01",{"name":52,"class":53},"YolTech Therapeutics Co., Ltd","INDUSTRY",1,{"id":56,"slug":57,"hasResults":11,"nctId":58,"briefTitle":59,"officialTitle":60,"acronym":4,"eligibilityCriteria":61,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":62,"targetDuration":4,"studyType":20,"phases":64,"briefSummary":66,"conditions":67,"keywords":68,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":71,"startDateStruct":73,"completionDateStruct":74,"leadSponsor":76,"locationsCount":54},"100637819","phase-2-study-of-yolt-203-in-children-and-adults-with-primary-hyperoxaluria-type-1-ph1-100637819","NCT07587021","Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)","A Randomized, Double-blind, Placebo-controlled Study Followed by a Treatment Extension to Evaluate the Efficacy and Safety of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1","Inclusion Criteria\n\n* You are 6 years old or older.\n* You have been diagnosed with PH1 (primary hyperoxaluria type 1) through genetic testing.\n* Urine tests show that your oxalate levels are not within certain limits (based on at least two 24-hour urine collections).\n* If you take vitamin B6, your dose has been stable for at least 3 months, and you are willing to keep it the same during the study.\n* You (and\u002For your parent or legal guardian, if you are under 18) understand the study and agree to follow all study requirements, including signing a consent form.\n\nExclusion Criteria\n\n* You have signs that oxalate has affected parts of your body outside the kidneys.\n* Your liver blood tests are not within certain limits.\n* You have certain abnormal blood clotting results (unless controlled with medication like warfarin).\n* You have HIV, or active or chronic hepatitis C.\n* You are pregnant, planning to become pregnant, or breastfeeding.\n* You are not willing to use birth control during the study and for 6 months after (if applicable).\n* You are unable to limit alcohol intake to no more than about 2 drinks per day.\n* You have had alcohol or drug abuse in the past year.\n* You are unable to follow standard treatments for PH1 (such as drinking plenty of fluids, taking prescribed medications like potassium citrate, or following dietary advice).",{"count":63,"type":19},36,[65],"PHASE2","This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1.\n\nAfter the initial randomized, 6-month double-blind, placebo-controlled period, participants who were initially assigned to placebo will receive a single-dose of YOLT-203 treatment whereas participants in the YOLT-203 group will receive a single-dose of placebo infusion.\n\nFor patients interested in additional information on how to participate, please follow this link: https:\u002F\u002Fmytomorro.ws\u002Fph1trial.",[25,26],[41,69,29,30,31,32,33,34,35,36,37,38,39,40],"primary hyperoxaluria Type 1","2026-08-07",{"date":72,"type":46},"2026-08-12",{"date":48,"type":19},{"date":75,"type":19},"2028-02",{"name":52,"class":53},{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":81,"acronym":4,"eligibilityCriteria":82,"healthyVolunteers":11,"sex":15,"minAge":83,"maxAge":84,"enrollmentInfo":85,"targetDuration":4,"studyType":20,"phases":87,"briefSummary":88,"conditions":89,"keywords":4,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":92,"lastUpdatePostDateStruct":93,"startDateStruct":95,"completionDateStruct":97,"leadSponsor":99,"locationsCount":5},"100557522","phase-1-a-phase-iiiaopen-label-single-ascending-dose-and-dose-expansion-clinical-study-to-evaluate-the-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-yolt-201-in-patients-with-transthyretin-amyloidosis-polyneuropathy-attr-pn-or-transthyretin-amyloidosis-cardiomyopathy-attr-cm-100557522","NCT06539208","A Phase I\u002FIIa，Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)","Inclusion Criteria:\n\n1. Age 18 - 80 years old (including the critical values), regardless of gender;\n2. Body weight at the time of screening is between 40 - 90kg (including the critical values);\n3. TTR gene mutation is confirmed by genetic testing;\n4. At the time of screening, the following laboratory standards must be met:\n\n   1. AST, ALT, and TBIL ≤ the upper limit of the normal value (ULN);\n   2. For subjects with Gilbert syndrome, TBIL ≤ 2 times ULN;\n   3. Glomerular filtration rate (GFR) ≥ 45 mL\u002Fmin\u002F1.73m2 (calculated according to the CKD-EPI formula);\n   4. Platelet count ≥ 100 × 109\u002FL;\n   5. Partial thromboplastin time (APTT), prothrombin time (PT), and thrombin generation time (TGT) are all within the reference value range, fibrinogen (FIB) ≥ the lower limit of the normal value (LLN) and ≤ 1.5\\*ULN, the international normalized ratio (INR) ≤ ULN, and if taking anticoagulant drugs, it is ≤ 2.5\\*ULN;\n   6. Vitamin A and vitamin B12 ≥ the lower limit of the reference value (LLN);\n   7. Low-density lipoprotein cholesterol (LDL) \\\u003C 200 mg\u002FdL (5.17 mmol\u002FL).\n5. Drugs approved for the treatment of ATTR are not accessible (Criterion A) and\u002For the disease still progresses despite the use of drugs approved for the treatment of ATTR (Criterion B):\n\n   * Criterion A: Meeting one or more of the following criteria:\n\n     1. Drugs for the treatment of ATTR are not marketed in China;\n     2. Unable to receive the approved drugs for ATTR treatment (e.g., intolerance or other medical, cost and\u002For other reasons);\n   * Criterion B: Subjects have received ATTR drug treatment for at least 3 months, but the subject's condition has progressed as assessed by the investigator, and meets any of the following criteria:\n\n   ATTR-CM: a. Increased number of hospitalizations related to heart failure; b. Worsening of NYHA classification; c. Decrease in KCCQ score by at least 5 points; d. Decrease in 6-MWT by at least 30m; e. Increase in NT-proBNP by 30%; f. Increase in Troponin I by 30%; g. Echocardiography indicates an increase in left ventricular wall thickness by 2mm; h. Echocardiography indicates a decrease in left ventricular ejection fraction by ≥ 5% or a decrease in global longitudinal strain by ≥ 1% or a decrease in stroke volume by ≥ 5%; i. New conduction block appears; ATTR-PN: a. PND score increase by ≥ 1 point; b. FAP increases by 1 stage; c. NIS score increase by ≥ 5 points; d. NIS-Lower Limb score increase by ≥ 5 points; e. mBMI decrease by ≥ 25 kg\u002Fm2×g\u002FL; f. 10-MWT decrease by ≥ 0.1 m\u002Fs; g. Electroneurophysiological examination (electromyography) worsens compared to the previous.\n6. Agree to stop drinking alcohol within the screening period to 28 days after administration;\n7. Female subjects need to be menopausal (absence of menstruation for at least 1 year) or have undergone uterine\u002Fovarian resection surgery; Male subjects and their partners have no fertility plans from the screening period to 6 months after the end of the trial and agree to take effective non-pharmaceutical contraceptive measures during the trial;\n8. The subject himself\u002Fherself (or his\u002Fher legally recognized representative) understands and signs the informed consent form;\n9. Agree not to receive other ATTR drug intervention treatment within at least 8 weeks after administration of YOLT-201;\n\n   For ATTR-PN only:\n10. Diagnosed as ATTR-PN according to the \"Consensus on the Diagnosis and Treatment of Transthyretin Amyloidosis Polyneuropathy\", and the NIS score at the screening is ≥ 5 and ≤ 130, and the PND score is ≤ IIIb;\n11. NT-proBNP \\\u003C 600pg\u002Fml at the screening;\n\n    For ATTR-CM only:\n12. Diagnosed as ATTR-CM according to the \"Expert Consensus on the Diagnosis and Treatment of Transthyretin Cardiac Amyloidosis\";\n13. The New York Heart Association (NYHA) cardiac function classification is grade II - III;\n14. The 6-minute walk test (6-MWT) is ≥ 150 m at the screening;\n15. NT-proBNP is ≥ 600pg\u002FmL and ≤ 3000pg\u002FmL at the screening;\n16. At the screening, echocardiography suggests evidence of cardiac involvement: the thickness of the interventricular septum and\u002For the posterior wall of the left ventricle is ≥ 12 mm.\n\nExclusion Criteria:\n\n1. Amyloidosis is not caused by TTR protein, such as light chain amyloidosis;\n2. There is meningeal transthyretin amyloidosis;\n3. Allergic to any lipid nanoparticle (LNP) component or has previously received LNP and experienced treatment-related laboratory abnormalities or adverse events;\n4. Use any of the following ATTR treatments within the prescribed time:\n\n   * In the dose escalation stage of the first stage, the use history of Patisiran, Inotersen, and Vutrisiran is excluded;\n   * In the dose expansion stage of the second stage, the following are excluded: Patisiran is used within 90 days before the administration of the investigational drug; Inotersen is used within 160 days before the administration of the investigational drug; Vutrisiran has a previous use history;\n   * Tafamidis: used within 10 days before the administration of the investigational drug;\n   * Diflunisal: used within 3 days before the administration of the investigational drug;\n   * Doxycycline and\u002For taurodeoxycholic acid: used within 14 days before the administration of the investigational drug;\n   * Previous use history of investigational gene editing drugs;\n   * Other drugs for the treatment of ATTR: the last use is less than 30 days or 5 half-lives before the administration of the investigational drug, whichever is longer.\n5. Unable or unwilling to supplement vitamin A during the trial;\n6. History of multiple myeloma;\n7. Ophthalmological examination results at the screening are consistent with vitamin A deficiency;\n8. Abnormal thyroid function test with clinical significance judged by the investigator;\n9. Known or suspected systemic infection (viral, parasitic or fungal infection) within 14 days before screening;\n10. History of past hepatitis B virus, hepatitis C virus, acquired immunodeficiency syndrome or positive HBsAg, HCV-Ab, and HIV-Ab at the screening;\n11. History of previous liver, heart or other organ transplantation or bone marrow transplantation or expected transplantation within 1 year (except for the history of corneal transplantation or planned corneal transplantation);\n12. History of bleeding or coagulation disorders (such as cirrhosis, malignant hematological disease, antiphospholipid antibody syndrome);\n13. History of acute thrombosis within 6 months before screening (such as acute myocardial infarction, acute cerebral infarction), or positive Leiden factor V and\u002For prothrombin gene test;\n14. History of malignant tumor within 5 years before screening (except for basal cell carcinoma of the skin, radicalized squamous cell carcinoma of the skin, and carcinoma in situ of the cervix);\n15. Planned invasive cardiovascular surgery during the trial (such as coronary artery stent\u002Fcoronary artery bypass, pacemaker placement, etc.); those who have undergone cardiovascular invasive surgery within 90 days before screening or have been hospitalized due to heart failure;\n16. History of alcohol abuse within 3 years before screening (definition of alcohol abuse: women drink ≥ 4 glasses\u002Fday or 8 glasses\u002Fweek, men ≥ 5 glasses or 15 glasses\u002Fweek, where 1 glass = 14g of pure alcohol);\n17. Expected survival period is less than 1 year;\n18. Other situations that the investigator deems inappropriate to enter this trial;\n\n    For ATTR-PN only:\n19. Other known diseases that cause motor or sensory neuropathy (such as diabetic neuropathy, neuropathy related to autoimmune diseases, etc.);\n20. Diagnosed with type 1 diabetes or type 2 diabetes for ≥ 5 years;\n21. NYHA cardiac function classification is grade III or IV within 90 days before screening;\n\n    For ATTR-CM only:\n22. NYHA cardiac function classification is grade IV within 90 days before screening;\n23. PND score is grade IIIa, IIIb or IV at the screening;\n24. Suffering from other cardiomyopathies not caused by TTR (such as hypertensive cardiomyopathy, valvular heart disease, cardiomyopathy caused by ischemic heart disease, etc.).","18 Years","80 Years",{"count":86,"type":19},31,[22,65],"This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of YOLT-201 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and participants with hereditary transthyretin amyloidosis with cardiomyopathy (ATTRv-CM).",[90,91],"Transthyretin Amyloidosis Polyneuropathy","Transthyrexin Amyloidosis Cardiomyopathy","2024-08-01",{"date":94,"type":46},"2024-08-06",{"date":96,"type":46},"2024-05-23",{"date":98,"type":19},"2026-06-30",{"name":52,"class":53},""]