[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"primary-hyperoxaluria-type-1\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:primary-hyperoxaluria-type-1":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,55,82],{"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":24,"conditions":25,"keywords":28,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":44,"lastUpdatePostDateStruct":45,"startDateStruct":48,"completionDateStruct":50,"leadSponsor":52,"locationsCount":4},"100637819","phase-2-study-of-yolt-203-in-children-and-adults-with-primary-hyperoxaluria-type-1-ph1-100637819",false,"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).","ALL","6 Years",{"count":19,"type":20},36,"ESTIMATED","INTERVENTIONAL",[23],"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.",[26,27],"Primary Hyperoxaluria Type 1","PH1",[29,30,31,32,33,34,35,36,37,38,39,40,41,42],"ph1","primary hyperoxaluria Type 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","NOT_YET_RECRUITING","2026-05-07",{"date":46,"type":47},"2026-05-14","ACTUAL",{"date":49,"type":20},"2026-06",{"date":51,"type":20},"2028-02",{"name":53,"class":54},"YolTech Therapeutics Co., Ltd","INDUSTRY",{"id":56,"slug":57,"hasResults":11,"nctId":58,"briefTitle":59,"officialTitle":60,"acronym":61,"eligibilityCriteria":62,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":63,"targetDuration":4,"studyType":21,"phases":65,"briefSummary":66,"conditions":67,"keywords":69,"overallStatus":71,"whyStopped":4,"lastUpdateSubmitDate":72,"lastUpdatePostDateStruct":73,"startDateStruct":75,"completionDateStruct":77,"leadSponsor":79,"locationsCount":81},"100407053","phase-2-safety--efficacy-of-dcr-phxc-in-patients-with-ph1-and-esrd-100407053","NCT04580420","Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD","A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 and Severe Renal Impairment, With or Without Dialysis","PHYOX7","Note: Currently, the DCR-PHXC-204 study is only enrolling potential participants under 6 years of age.\n\nInclusion Criteria:\n\n1. Four age groups of participants will be enrolled:\n\n   1. adults and adolescents (aged ≥ 12 years)\n   2. children 6 to 11 years of age\n   3. children 2 to 5 years of age\n   4. infants and newborns from birth to \\\u003C 2 years of age\n2. . Documented diagnosis of PH1, confirmed by genotyping\n3. Estimated GFR at Screening \\\u003C30mL\u002Fmin normalized to 1.73m\\^2 BSA\n4. Mean of 2 Plasma Oxalate \\>20μmol\u002FL during screening\n5. For participants receiving hemodialysis or peritoneal dialysis total duration of hemodialysis or peritoneal dialysis must be less than 24 months and hemodialysis or peritoneal dialysis regimen must have been stable for at least 2 weeks prior to Screening.\n6. Male or Female\n\n   1. Male participants:\n\n      * A male participant with a female partner of childbearing potential must agree to use contraception during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from donating sperm during this period.\n   2. Female participants:\n\n      * A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:\n\n      Not a woman of childbearing potential (WOCBP).\n      * OR\n      * A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 12 weeks after the last dose of study intervention and agrees to refrain from harvesting\u002Ffreezing eggs during this period.\n   3. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n7. Participant (and\u002For participant's parent or legal guardian if participant is a minor \\[defined as patient \\\u003C18 years of age, or younger than the age of majority according to local regulations\\]) is capable of giving signed informed consent, which includes compliance with the requirement and restrictions listed in the informed consent form (ICF) and in the protocol.\n\n   1. Adolescents (12 to \\\u003C 18 years of age, or older than 12 years but younger than the age of majority according to local regulations) must be able to provide written assent for participation.\n   2. For children younger than 12 years of age, assent will be based on local regulations\n8. Affiliated with or is a beneficiary of a health insurance system (if applicable per national regulations)\n\nExclusion Criteria:\n\n1. Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Prior renal transplantation is allowed.\n2. Documented evidence of clinical manifestations of severe systemic oxalosis (including preexisting retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)\n3. Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact patient safety including, but not restricted to:\n\n   1. Severe intercurrent illness\n   2. Known causes of active liver disease\u002Finjury (e.g., alcoholic liver disease, nonalcoholic fatty liver disease\u002Fsteatohepatitis)\n   3. Non-PH related conditions contributing to renal insufficiency\n   4. Physician concerns about intake of drugs of abuse or excessive alcohol intake, or history of excessive alcohol intake in the 2 years prior to enrollment (defined as ≥ 21 units of alcohol per week in men and ≥ 14 units of alcohol per week in women; where a \"unit\" of alcohol is equivalent to a 12-ounce beer, 4-ounce glass of wine, or 1ounce shot of hard liquor)\n4. Use of an RNAi drug, other DCR-PHXC, within the last 6 months\n5. History of one or more of the following reactions to an oligonucleotide-based therapy:\n\n   1. Severe thrombocytopenia (platelet count ≤ 100,000\u002FµL)\n   2. Hepatotoxicity, defined as alanine transaminase (ALT) or aspartate transaminase (AST) \\> 3 times the upper limit of normal (ULN) and total bilirubin \\> 2 × ULN or international normalized ratio (INR) \\>1.5\n   3. Severe flu-like symptoms leading to discontinuation of therapy\n   4. Localized skin reaction from the injection (graded severe) leading to discontinuation of therapy\n   5. Coagulopathy\u002Fclinically significant prolongation of clotting time\n6. Participation in any clinical study in which they received an investigational medicinal product (IMP) other than DCR-PHXC within 4 months before Screening.\n7. Liver function test abnormalities: ALT and\u002For AST \\>1.5 × ULN for age and gender\n8. Positive anti-double-stranded deoxyribonucleic acid (anti-dsDNA) antibody test at Screening\n9. Known hypersensitivity to DCR-PHXC or any of its ingredients\n10. Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations",{"count":64,"type":20},28,[23],"The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.",[26,68],"End Stage Renal Disease",[27,70],"ESRD","RECRUITING","2025-12-23",{"date":74,"type":47},"2025-12-24",{"date":76,"type":47},"2021-04-15",{"date":78,"type":20},"2032-01-30",{"name":80,"class":54},"Dicerna Pharmaceuticals, Inc., a Novo Nordisk company",18,{"id":83,"slug":84,"hasResults":11,"nctId":85,"briefTitle":86,"officialTitle":86,"acronym":87,"eligibilityCriteria":88,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":89,"targetDuration":4,"studyType":21,"phases":91,"briefSummary":93,"conditions":94,"keywords":97,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":101,"lastUpdatePostDateStruct":102,"startDateStruct":104,"completionDateStruct":106,"leadSponsor":108,"locationsCount":4},"100551852","phase-3-evaluation-of-the-efficacy-and-safety-of-stiripentol-in-patients-6-years-and-older-with-primary-hyperoxaluria-type-1-2-or-3-100551852","NCT06465472","Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3","CRYSTAL","Screening Criteria :\n\n* 1\\. Male or female patients aged ≥ 6 years at the time of consent signature\n* 2\\. Diagnosed with primary hyperoxaluria (type 1, 2 or 3) documented as per standard methods\n* 3\\. With last estimated Glomerular Filtration Rate ≥ 45 mL\u002Fmin\u002F1.73 m2\n* 4\\. Able to understand and willing to comply with study requirements and to provide written informed consent. In case of patient under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent as per local and national requirements\n* 5\\. Female patients with contraception (intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, surgical sterilization of male partner, established use of oral, implantable, injectable, or transdermal hormonal methods of contraception, a double-barrier method (combination of male condom with cap, diaphragm, or sponge, in conjunction with spermicide) or true sexual abstinence\n\nInclusion Criteria:\n\n* 1\\. Diagnosed with primary hyperoxaluria disease and subtype (type 1, 2 or 3) confirmed by genetic testing\n* 2\\. Receiving optimal management of the disease through standard of care strategies (e.g., increased fluid intake, vitamin B6, potassium citrate) with or without approved target medications (e.g., lumasiran). Patients not receiving lumasiran can only be enrolled if they are not eligible for treatment with lumasiran for the specific following reasons: contraindications, previous treatment discontinued due to lack of efficacy or poor tolerability, not meeting national or regional eligibility criteria for treatment, investigator judgement\n* 3\\. With mean 24-hour urinary oxalate excretion from 2 valid 24-hour urine collections ≥ 0.70 mmol\u002F24h\u002F1.73m²\n* 4\\. With estimated Glomerular Filtration Rate ≥ 45 mL\u002Fmin\u002F1.73 m2 (Schwartz et al., 2009 in pediatric patients and CKD-EPI in adults)\n* 5\\. Pubescent and adult female patients must have a negative urine or serum pregnancy test within 60 days prior to first dose of study treatment if of childbearing potential. If the urine pregnancy test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. The serum pregnancy test must be negative for the patient to be eligible\n* 6\\. In France, patient affiliated with or who benefits from a social security scheme\n\nExclusion Criteria:\n\n* 1\\. Any relevant change in the use of any component of the standard of care (fluid intake, vitamin B6, potassium citrate) in the 4 weeks prior to inclusion or if such change is planned to occur during the first 6 months of the study\n* 2\\. If under approved targeted medications (e.g., lumasiran), treatment should have been administered for at least 6 months, with no change in dose or regimen in the 3 months prior to inclusion or ifsuch change is planned it should not occur during the first 12 months of the study\n* 3\\. History of kidney or liver transplant\n* 4\\. Presenting any of the following liver function tests abnormalities during the screening period:\n\n  1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST)\\> 2 × upper limit of normal (ULN)\n  2. Total bilirubin \\> 1.5 x ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if the total bilirubin is \\\u003C 2 x ULN\n* 5\\. Recent (4 weeks before the screening visit) or planned change in eating habits\n* 6\\. Intermittent fasting planned during the 6 first months of the study period (e.g., Ramadan)\n* 7\\. Other medical conditions or comorbidities, treatment, which in the opinion of the investigator, would interfere with study compliance or data interpretation\n* 8\\. Presenting any significant biological or clinical anomalies that are not compatible with participation in the study according to the investigator\n* 9\\. History of severe allergy, asthma, skin rashes, intolerance to lactose or hypersensitivity to the study treatments\n* 10\\. Treatment affecting hepatic metabolism (i.e., cimetidine, ketoconazole, fluconazole, itraconazole, phenytoin, rifampicin, rifabutin) that is ongoing or has been taken in the month prior to the selection visit\n* 11\\. Treatment affecting the renal tubule (probenecid, β-lactam, etc.,) that is ongoing or has been taken in the two weeks prior to the start of the study\n* 12\\. Contraindications to stiripentol as defined in the applicable Investigator's Brochure (i.e. patients presenting a hypersensitivity to the active substance or any excipients)\n* 13\\. Patient at risk of pregnancy, pregnant or breastfeeding female\n* 14\\. Patient under guardianship or curatorship\n* 15\\. Patient under the protection of the Court or deprived of liberty\n* 16\\. Patient participating in another interventional clinical trial which could interfere with the trial's results or impact the other trial's results; or within the last 30 days or 5 half-lives of the study investigational treatment, whichever is longer, prior to the urinary sampling during the screening period, or are in follow-up of another clinical study prior to randomization\n* 17\\. Patient whose current state of health does not allow him\u002Fher to give consent",{"count":90,"type":20},42,[92],"PHASE3","Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3.",[26,95,96],"Primary Hyperoxaluria Type 2","Primary Hyperoxaluria Type 3",[98,99,100],"Stiripentol","Primary Hyperoxaluria","Oxalate","2024-06-12",{"date":103,"type":47},"2024-06-18",{"date":105,"type":20},"2024-08",{"date":107,"type":20},"2030-08",{"name":109,"class":54},"Biocodex"]