[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"alport-syndrome\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:alport-syndrome":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,11,0,[8,41,77,116,144,177,206,235,264,365,387],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":21},"100609217","phase-2-a-study-to-learn-about-how-well-bay-3401016-works-in-adults-with-alport-syndrome-100609217",false,"NCT07211685","A Study to Learn About How Well BAY 3401016 Works in Adults With Alport Syndrome","A Randomized, Double-blind, Placebo-controlled, Parallel Group Phase 2a Study With an Extension Phase to Evaluate the Efficacy and Safety of BAY 3401016 in Participants Aged 18 to 45 With Alport Syndrome","ASSESS","Inclusion Criteria:\n\n* Participants must be 18 to 45 years of age inclusive\n* Participants with AS, either XLAS (male) or ARAS (male or female)\n* eGFR ≥ 45 mL\u002Fmin\u002F1.73m2\n* UACR ≥ 500mg\u002Fg\n\nExclusion Criteria:\n\n* Chronic kidney disease is different from AS\n* Clinically significant illness that could have influence on the safety of the participant and\u002For interfere with the study objectives\n* History or current existence of malignancy\n* Participants with history of severe allergies, multiple drug allergies or non-allergic drug reactions including allergies affecting the lower respiratory tract - allergic asthma, allergies requiring therapy with corticosteroids or urticaria\n* Participants with active skin disorders (e.g. atopic dermatitis, severe acne)\n* Systolic blood pressure above 140 mmHg\n* Diastolic blood pressure above 90 mmHg","ALL","18 Years","45 Years",{"count":21,"type":22},60,"ESTIMATED","INTERVENTIONAL",[25],"PHASE2","Alport syndrome (AS) is a rare genetic condition that causes kidney disease, hearing loss, and eye abnormalities that occur due to changes in specific genes (COL4A3, COL4A4, and COL4A5). These genes help in producing an important protein called collagen. People with AS have a high risk of developing chronic kidney disease (CKD), a condition in which there is progressive loss in kidney function over time. The kidneys soon lose their ability to remove waste products from the body properly, resulting in end-stage kidney disease. A common sign of decreasing kidney function is the presence of excess protein in the urine that is not usually found with healthy kidneys. This condition is known as proteinuria. The study drug, BAY 3401016 (a monoclonal antibody), is a type of medicine that blocks a protein called Semaphorin 3A (Sema3A), which is thought to be involved in causing kidney damage in AS. By blocking the action of the Sema3A protein, BAY 3401016 may prevent proteinuria and slow down the loss in kidney function due to AS.\n\nThe main purpose of this study is to learn more about how well BAY 3401016 works in slowing down the loss in kidney function in adults with a rapidly progressing AS.",[28],"Alport Syndrome","RECRUITING","2026-07-01",{"date":32,"type":33},"2026-07-02","ACTUAL",{"date":35,"type":33},"2025-11-19",{"date":37,"type":22},"2028-07-27",{"name":39,"class":40},"Bayer","INDUSTRY",{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":49,"targetDuration":4,"studyType":51,"phases":4,"briefSummary":52,"conditions":53,"keywords":62,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":67,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":76},"100638972","periodontal-disease-in-rare-renal-disorders-perio-ra-re-100638972","NCT07575347","Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)","Periodontal Inflammation in Rare Renal Disorders - A Cross-Sectional Controlled Observational Study Assessing the Burden and Phenotypes of Periodontal Disease","PERIO-RA-RE","Inclusion Criteria:\n\n* Age ≥18 years\n* Ability to provide written informed consent\n* At least 10 natural teeth present\n* Belonging to one of the predefined study groups:\n\n  1. Alport syndrome (genetically or clinically confirmed)\n  2. Fabry disease (enzymatically or genetically confirmed)\n  3. Tuberous sclerosis complex (according to established clinical or genetic criteria)\n  4. Systemic lupus erythematosus defined according to the 2019 EULAR\u002FACR or SLICC 2012 classification criteria, with renal involvement defined by at least one of the following: \\[1\\] Biopsy-proven lupus nephritis, \\[2\\] Persistent proteinuria (\\>0.5 g\u002Fday or equivalent), \\[3\\] Active urinary sediment (hematuria and\u002For cellular casts) consistent with lupus nephritis\n  5. Chronic kidney disease (CKD) of non-rare etiology: defined according to KDIGO criteria (eGFR \\\u003C60 ml\u002Fmin\u002F1.73 m² and\u002For markers of kidney damage)\n  6. Individuals without CKD, recruited from clinical or dental care settings as non-CKD controls\n\nExclusion Criteria:\n\n* Periodontal treatment within the last 6 months\n* Antibiotic therapy within the last 4 weeks\n* Pregnancy\n* Conditions precluding periodontal examination\n* Inability to comply with study procedures",{"count":50,"type":22},100,"OBSERVATIONAL","This study aims to evaluate the burden and phenotypic spectrum of periodontal disease in patients with rare kidney disorders (such as Alport syndrome, Fabry disease, and tuberous sclerosis complex) and systemic lupus erythematosus (SLE), compared with chronic kidney disease (CKD) controls and population controls.\n\nThis is a cross-sectional, case-control observational study. Participants will undergo a single structured evaluation including a full-mouth periodontal examination, a clinical questionnaire, and collection of relevant clinical and nephrological data.\n\nThe primary objective is to compare the prevalence of periodontitis across study groups. Secondary objectives include characterization of periodontal disease severity, prevalence of gingivitis and xerostomia, and identification of disease-specific oral phenotypes.\n\nExploratory analyses will assess associations between periodontal disease and clinical variables such as kidney function, proteinuria, and immunosuppressive exposure.",[54,55,56,57,28,58,59,60,61],"Periodontal Disease","Periodontitis","CKD","Chronic Kidney Disease","Fabry Disease","Lupus or SLE","Tuberous Sclerosis Complex (TSC)","Systemic Lupus Erythematosus (SLE)",[54,55,56,63,64,65,60,57],"Rare Kidney Diseases","Alport syndrome","Systemic Lupus Erythematosus","2026-06-24",{"date":68,"type":33},"2026-06-26",{"date":70,"type":33},"2026-05-04",{"date":72,"type":22},"2027-12-31",{"name":74,"class":75},"Stefan Lujinschi","OTHER",1,{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":82,"acronym":83,"eligibilityCriteria":84,"healthyVolunteers":11,"sex":17,"minAge":85,"maxAge":86,"enrollmentInfo":87,"targetDuration":4,"studyType":23,"phases":89,"briefSummary":91,"conditions":92,"keywords":101,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":106,"lastUpdatePostDateStruct":107,"startDateStruct":109,"completionDateStruct":111,"leadSponsor":113,"locationsCount":115},"100406380","neptune-match-study-100406380","NCT04571658","NEPTUNE Match Study","Implementing Precision Medicine for Glomerular Diseases in the Nephrotic Syndrome Study Network (NEPTUNE)","NEPTUNE Match","Inclusion Criteria:\n\n1. Consented and eligible participants in the biopsied or non-biopsied cohorts of the NEPTUNE observational study\n2. Must be potentially eligible for the NEPTUNE Match partnering trials (e.g. if no trial is enrolling a participant under age 6, those under 6 are not eligible).\n\n   Note: NEPTUNE Match partnering trials and associated eligibility criteria are expected to be dynamic and change as trial protocols are developed, activated, and amended.\n3. Regular nephrology healthcare provided at a NEPTUNE study site.\n4. Willing and able to consent, and as appropriate assent, to participate in NEPTUNE Match\n\nExclusion Criteria:\n\nCurrently non-NEPTUNE observational study participants are not eligible to be matched to a clinical trial using these biomarker assessments.\n\nExclusion Criteria:\n\n1\\. Non-English or non-Spanish speaking","1 Year","80 Years",{"count":88,"type":22},375,[90],"NA","NEPTUNE Match is an additional opportunity offered to NEPTUNE study participants to prospectively recruit and communicate patient-specific clinical trial matching with kidney patients and their physician investigators.",[93,94,95,96,97,98,99,100,28],"Nephrotic Syndrome in Children","Focal Segmental Glomerulosclerosis","Minimal Change Disease","Minimal Change Nephrotic Syndrome","Membranous Nephropathy","FSGS","MCD","MCD - Minimal Change Disease",[102,103,104,105],"Nephrotic Syndrome","NEPTUNE","Match","Clinical Trial Match","2026-06-08",{"date":108,"type":33},"2026-06-10",{"date":110,"type":33},"2022-05-02",{"date":112,"type":22},"2029-12-30",{"name":114,"class":75},"University of Michigan",16,{"id":117,"slug":118,"hasResults":11,"nctId":119,"briefTitle":120,"officialTitle":121,"acronym":122,"eligibilityCriteria":123,"healthyVolunteers":11,"sex":17,"minAge":85,"maxAge":124,"enrollmentInfo":125,"targetDuration":4,"studyType":23,"phases":127,"briefSummary":128,"conditions":129,"keywords":132,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":134,"lastUpdatePostDateStruct":135,"startDateStruct":137,"completionDateStruct":139,"leadSponsor":141,"locationsCount":143},"100439568","phase-2-study-of-sparsentan-treatment-in-pediatrics-with-proteinuric-glomerular-diseases-100439568","NCT05003986","Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases","A Phase 2, Open-Label, Single-Arm, Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Sparsentan Treatment in Pediatric Subjects With Selected Proteinuric Glomerular Diseases","EPPIK","Inclusion Criteria for All Subjects (All Three Populations):\n\nA subject must meet all of the following criteria to be eligible for participation in this study:\n\n* The subject or parent\u002Flegal guardian (as appropriate) is willing and able to provide signed informed consent\u002Fassent, and where required, the subject is willing to provide assent before any screening procedures per local requirements.\n* The subject has an estimated glomerular filtration rate (eGFR) ≥30 mL\u002Fmin\u002F1.73 m2 at screening.\n* The subject has a mean seated blood pressure between the 5th and 95th percentile for sex and height.\n\nInclusion Criteria for Population 1:\n\n* The subject is male or female ≥1 year at screening and \\\u003C18 years of age at Day 1 (Baseline).\n* The subject has a UP\u002FC ≥1.5 g\u002Fg (170 mg\u002Fmmol) at screening AND one of the following:\n* Kidney biopsy-proven FSGS or MCD histological patterns and clinical presentation consistent with primary FSGS or MCD and qualifying proteinuria at screening despite history or ongoing treatment with corticosteroids and\u002For other immunosuppressive disease-modifying agents.\n* Documentation of a genetic mutation in a podocyte protein associated with FSGS or MCD. Subjects with a documented podocytic mutation do not require kidney biopsy.\n* Kidney biopsy-proven FSGS histological pattern with medical history and clinical presentation consistent with maladaptive cause of the lesion.\n\nNote: The kidney biopsy may have been performed at any time in the past but must include light microscopy and electron microscopy characteristics and\u002For immunofluorescence findings consistent with FSGS or MCD.\n\nInclusion Criteria for Population 2:\n\n* The subject is male or female ≥2 years at screening and \\\u003C18 years of age at Day 1 (Baseline).\n* The subject has UP\u002FC ≥0.6 g\u002Fg (68 mg\u002Fmmol) at screening AND one of the following diagnoses:\n* Kidney biopsy-confirmed IgAN, IgAV, or AS\n* Diagnosis of AS by genetic testing (pathogenic X-linked Collagen, Type IV, Alpha-5 (COL4A5) mutation OR autosomal-recessive mutations in both alleles of Collagen, Type IV, Alpha-3 (COL4A3) and\u002For Collagen, Type IV, Alpha-4 (COL4A4) OR autosomal-dominant COL4A3 and\u002For COL4A4 and digenic mutations \\[ie, simultaneous mutations in 2 of the COL4A3, COL4A4, and COL4A5 genes\\])\n\nInclusion Criteria for Population 3:\n\n* The subject is male or female ≥8 years at screening and \\\u003C18 years of age at Day 1 (Baseline).\n* The subject has UP\u002FC ≥1.0 g\u002Fg (113 mg\u002Fmmol) at screening AND has kidney biopsy-confirmed IgAN\n* Subject weighs ≥40 kg\n* The subject has been on ACEI and\u002For ARB therapy for at least 12 weeks prior to screening\n\nExclusion Criteria for All Subjects (All Three Populations):\n\nA subject who meets any of the following will be excluded from this study:\n\n* The subject weighs \\\u003C7.3 kg at screening.\n* The subject has FSGS or MCD histological pattern secondary to viral infections, drug toxicities, or malignancies.\n* The subject has immunoglobulin A (IgA) glomerular deposits not in the context of primary IgAN or IgAV (ie, secondary to another condition; eg, systemic lupus erythematosus and liver cirrhosis).\n* The subject has had an acute onset or presentation of glomerular disease or a diagnostic biopsy or a relapse of glomerular disease requiring new or different class of immunosuppressive treatment (including, but not limited to, systemic corticosteroids, calcineurin inhibitors and mycophenolate mofetil, abatacept, cyclophosphamide, rituximab, ofatumumab, and ocrelizumab) within 6 months before screening.\n* Subjects taking chronic immunosuppressive medications (including systemic steroids) not on a stable dose for ≥1 month before screening.\n* The subject requires any of the prohibited concomitant medications as defined in the study protocol.\n* The subject has undergone any organ transplantation, with the exception of corneal transplants.\n* The subject has a documented history of congenital or acquired heart failure (modified Ross heart failure classification for children Class II to Class IV) and\u002For previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and\u002For peripheral edema.\n* The subject has hemodynamically significant cardiac valvular disease.\n* The subject has clinically significant congenital vascular disease.\n* The subject has jaundice, hepatitis, or known hepatobiliary disease, or alanine aminotransferase and\u002For aspartate aminotransferase \\>2 times the upper limit of the normal range at screening.\n* The subject has a history of malignancy within the past 2 years.\n* The subject has a screening hematocrit \\\u003C27% (0.27 L\u002FL) or a hemoglobin value \\\u003C9 g\u002FdL (90 g\u002FL).\n* The subject has a screening potassium value \\>5.5 milliequivalent (mEq)\u002FL (5.5 mmol\u002FL).\n* The subject has any abnormal clinical laboratory screening values that are considered by the Investigator to be clinically significant.\n* The subject has a history of allergic response to any angiotensin II antagonist or endothelin receptor antagonist, including sparsentan, or has a hypersensitivity to any of the excipients in the study medication.\n* The female subject is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.\n* Female subjects of childbearing potential, beginning at menarche, who do not agree to use 1 highly reliable (ie, can achieve a failure rate of \\\u003C1% per year) method of contraception from 7 days before the first dose of the study medication until 28 days after the last dose of study medication. Examples of highly reliable contraception methods include stable oral, implanted, transdermal, or injected contraceptive hormones associated with the inhibition of ovulation or an intrauterine device. One additional barrier method must also be used during vaginal sexual activity, such as a diaphragm, diaphragm with spermicide (preferred), or male partner's use of male condom or male condom with spermicide (preferred), from Day 1\u002FRandomization until 28 days after the last dose of study medication. Female subjects of childbearing potential are defined as those who are fertile after menarche, unless permanently sterile; permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy. All female subjects of childbearing potential must have a negative serum pregnancy test result at screening (Visit 1) and a negative urine pregnancy test result, with positive results confirmed by serum, at every study visit from Day 1 (Visit 3) and after.\n\nNote: Before menarche, pregnancy testing and contraceptive use are not required. However, subjects and their parents\u002Flegal guardians must be advised that, immediately upon menarche, subjects will be required to begin pregnancy testing and initiate contraceptive use. This requirement cannot be waived.\n\n* The subject has participated in a study of another study medication within 28 days before screening or plans to participate in such a study during the course of this study.\n* The subject has had prior exposure to sparsentan.\n* The subject or parent\u002Flegal guardian (as appropriate), in the opinion of the Investigator, are unable to adhere to the requirements of the study including but not limited to, a history of noncompliance and\u002For any other reason that causes the Investigator to believe the subject would not be a good candidate for the study.\n* For Population 3 - the subject is unable to swallow the study medication tablets whole.","17 Years",{"count":126,"type":22},67,[25],"To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.",[94,95,130,131,28],"Immunoglobulin A Nephropathy","IgA Vasculitis",[133],"Alport, AS, FSGS, IgAN, IgAV, MCD, pediatrics, peds","2026-05-08",{"date":136,"type":33},"2026-05-12",{"date":138,"type":33},"2021-08-12",{"date":140,"type":22},"2027-04-12",{"name":142,"class":40},"Travere Therapeutics, Inc.",47,{"id":145,"slug":146,"hasResults":11,"nctId":147,"briefTitle":148,"officialTitle":149,"acronym":4,"eligibilityCriteria":150,"healthyVolunteers":11,"sex":17,"minAge":151,"maxAge":4,"enrollmentInfo":152,"targetDuration":154,"studyType":51,"phases":4,"briefSummary":155,"conditions":156,"keywords":159,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":168,"lastUpdatePostDateStruct":169,"startDateStruct":171,"completionDateStruct":173,"leadSponsor":175,"locationsCount":76},"100556564","asf-alport-patient-registry-100556564","NCT06526741","ASF Alport Patient Registry","Alport Syndrome Foundation Alport Patient Registry","Inclusion Criteria:\n\n1. Confirmed diagnosis of Alport syndrome by a certified genetic counselor, treating physician or nephrologist.\n2. Signed informed consent\u002Fassent must be provided by the subject and\u002For caregiver (parent\u002Flegal guardian) including compliance with the restrictions listed in the informed consent\u002Fassent form and in the study protocol. (Separate age-appropriate assent forms are provided for ages 7-12 years and ages 13-17 years.)\n3. Must reside in the USA or US territories and outlying islands. (This criterium may change at an as-yet undetermined future date.)\n\nExclusion Criteria:\n\n\\[none\\]","0 Years",{"count":153,"type":22},2500,"90 Days","Alport Syndrome Foundation's (ASF's) Alport Patient Registry (the Registry) is open to individuals living with Alport syndrome in the United States (US) and US territories and outlying islands. The Registry welcomes participants of all ages who have a confirmed clinical diagnosis of Alport syndrome. A confirmed diagnosis could be obtained via genetic testing, biopsy, and\u002For from a medical professional's clinical assessment of the individual's symptoms and\u002For family history. Participants can have any form and stage of this disease to be eligible for inclusion in the Registry.\n\nPatient participation in the Registry is crucial to helping attract and advance research, understanding understudied aspects of the disease, and informing clinical trials that may lead to Alport syndrome therapies and\u002For a cure.\n\nThe Registry is accessed through a secure, online application. Participants report their own health history in the Registry and are encouraged to update any changes, at most, every three months.\n\nThe security of each participant's information is a top priority. Any detail that could identify an individual participant is kept confidential in the Registry and such data are de-identified to protect the participant's privacy. No electronic health records or social security numbers are requested by or connected to the Registry.\n\nA parent or legal guardian may consent to enroll a child\u002Fdren Alport patient(s) under the age of 18 years. An additional assent form is used for individuals ages 7-17. At age 18, participants will be required to re-consent as an adult if they choose to continue to participate in the Registry.",[28,157,158],"Thin Basement Membrane Disease","Hereditary Nephritis",[160,161,162,163,164,165,166,167],"Alport","COL4A3","COL4A4","COL4A5","Collagen Type-IV","Glomerulonephropathy","Glomerulosclerosis","Genetic Kidney Disease","2026-04-09",{"date":170,"type":33},"2026-04-14",{"date":172,"type":33},"2023-08-24",{"date":174,"type":22},"2048-08-23",{"name":176,"class":75},"Alport Syndrome Foundation",{"id":178,"slug":179,"hasResults":11,"nctId":180,"briefTitle":181,"officialTitle":182,"acronym":183,"eligibilityCriteria":184,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":185,"targetDuration":4,"studyType":51,"phases":4,"briefSummary":187,"conditions":188,"keywords":189,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":196,"lastUpdatePostDateStruct":197,"startDateStruct":199,"completionDateStruct":201,"leadSponsor":203,"locationsCount":76},"100510517","eurbio-alport-radico-cohort-radico-eurbio-alport-100510517","NCT05927467","Eurbio-Alport (RaDiCo Cohort) (RaDiCo Eurbio-Alport)","Study of the Natural History of Alport Syndrome by Establishment of an International Database","Eurbio-Alport","Inclusion Criteria:\n\n* Diagnosis of AS based on electron microscopic examination of the renal biopsy and\u002For molecular studies and\u002For abnormal expression of type IV collagen chains on skin and\u002For glomerular basement membranes.\n* Signed informed consent\n\nExclusion Criteria:\n\n\\- No exclusion criteria",{"count":186,"type":22},700,"Alport syndrome is a rare, inherited condition characterized by a combination of glomerular nephropathy progressing to kidney failure, deafness, and eye involvement. This disease is associated with mutations in the genes encoding one of the three IV collagen chains expressed in the glomerular basement membrane. Significant progress has been made in understanding the molecular mechanisms responsible for the disease, but relatively little in understanding the progression of renal failure and in the area of therapeutics. We have shown in a retrospective European study that blockers of the renin angiotensin system may slow disease progression, but no controlled studies have been performed. Finally, innovative therapies (anti-micro-RNA, stem cells) have recently shown their effectiveness in animal models of the disease, and industrials are planning to quickly carry out phase 1 trials to test molecules. Carrying out therapeutic trials in humans will require full knowledge of the natural history of the disease (isolated hematuria, microalbuminuria, macroalbuminuria, renal failure and its progression) and gathering a sufficient number of patients, especially in the early stages. These trials and the indications for treatments would be greatly facilitated by the discovery of biomarkers that make it possible to predict the progression to renal failure earlier than the onset of proteinuria.\n\nThe study aims to:\n\n* Establish a European database on Alport syndrome to assess the natural history of the disease.\n* To investigate the impact of the disease on the educational and professional life of patients and their families, and on the adherence and tolerance to renin-angiotensin system blockers prescribed to proteinuric patients.\n* Investigate access to molecular diagnostics and genetic counseling, as well as identify biomarkers that can predict progression of kidney disease.\n\nThis project will be carried out at a French level with the support and participation of the very active renal rare disease sector, in collaboration with various countries wishing to participate.",[28],[28,190,191,192,193,194,195],"Rare disease","European database","Quality of life","Kidney disease","Molecular diagnostics","Predictive biomarkers","2025-09-17",{"date":198,"type":33},"2025-09-18",{"date":200,"type":33},"2017-05-09",{"date":202,"type":22},"2026-06-30",{"name":204,"class":205},"Institut National de la Santé Et de la Recherche Médicale, France","OTHER_GOV",{"id":207,"slug":208,"hasResults":11,"nctId":209,"briefTitle":210,"officialTitle":211,"acronym":212,"eligibilityCriteria":213,"healthyVolunteers":214,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":215,"targetDuration":217,"studyType":51,"phases":4,"briefSummary":218,"conditions":219,"keywords":223,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":226,"lastUpdatePostDateStruct":227,"startDateStruct":229,"completionDateStruct":231,"leadSponsor":233,"locationsCount":76},"100238186","alport-therapy-registry---european-initiative-towards-delaying-renal-failure-in-alport-syndrome-100238186","NCT02378805","Alport Therapy Registry - European Initiative Towards Delaying Renal Failure in Alport Syndrome","European Alport Therapy Registry - European Initiative Towards Delaying Renal Failure in Alport Syndrome: Current and Novel Therapies","Alport-XXL","Inclusion Criteria:\n\nDiagnosis of Alport syndrome (AS) by kidney biopsy or mutation analysis (or both).\n\nAny type of genetic variant is accepted for X-linked, autosomal or digenic Alport syndrome (COL4A3, 4 or 5 genes).\n\nExclusion criteria:\n\nPatients not willing to give informed consent. Patient with suspected diagnosis, whcih cannot be confirmed.",true,{"count":216,"type":22},800,"30 Years","The hereditary type IV collagen disease Alport syndrome leads to kidney failure early in life. Currently there are no specific medications approved for treatment, however, several therapies have been evaluated preclinically and could improve outcome. For that reason, this non-interventional, observational study investigates, if medications (1) delay disease progression; (2) delay time to kidney failure; (3) improve life-expectancy compared to untreated patients (relatives). This observational study started in 2006 as an European registry. Since 2019, this registry has been expanded to \"Alport XXL\" via the International Alport Alliance as a global effort across all continents. From 2020 on to present, \"Alport XXL\" has a special focus on the outcomes of early therapy in young patients on ACE-inhibitors vs. Angiotensin-receptor blockers vs. their combination.",[28,220,221,157,222],"Hereditary Kidney Disease","Pediatric Kidney Disease","Familial Benign Hematuria",[64,224,225],"thin basement membrane disease","familial benign hematuria","2025-03-03",{"date":228,"type":33},"2025-03-06",{"date":230,"type":4},"1995-07",{"date":232,"type":22},"2036-03-01",{"name":234,"class":75},"University Hospital Goettingen",{"id":236,"slug":237,"hasResults":11,"nctId":238,"briefTitle":239,"officialTitle":240,"acronym":241,"eligibilityCriteria":242,"healthyVolunteers":11,"sex":17,"minAge":243,"maxAge":244,"enrollmentInfo":245,"targetDuration":4,"studyType":23,"phases":247,"briefSummary":249,"conditions":250,"keywords":251,"overallStatus":254,"whyStopped":4,"lastUpdateSubmitDate":255,"lastUpdatePostDateStruct":256,"startDateStruct":258,"completionDateStruct":260,"leadSponsor":262,"locationsCount":4},"100572278","phase-2-human-umbilical-cord-mesenchymal-stem-cells-for-alport-syndrome-100572278","NCT06731192","Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome","Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome: Α Prospective Randomized, Single-blind, Placebo-controlled, Single-center Clinical Trial","HUCMSC","Inclusion Criteria:\n\n* 3 years old ≤Age ≤ 12 years old;\n* Meet the diagnostic criteria of Alport syndrome;\n* Positive proteinuria or combined hematuria;\n* Chronic kidney disease (CKD) stage: I-III stage children, that is, glomerular filtration rate greater than 60 ml\u002Fmin.1.73m2;\n* No history of infectious diseases within 1 week before treatment;\n* Negative infectious disease screening;\n* No allergic state and related clinical manifestations;\n* Signed informed consent (children or their families).\n\nExclusion Criteria:\n\n* Age \\\u003C3 years or \\>12 years old;\n* Alport syndrome patients with only microscopic hematuria and normal glomerular filtration rate;\n* Patients with significantly reduced renal function, chronic kidney disease stage IV or V, Alport syndrome;\n* Patients with other renal diseases;\n* Have a history of severe allergic reactions or be allergic to 2 or more foods or drugs;\n* Known allergy to stem cells or stem cell-derived products or ingredients in stem cell preparations;\n* Have severe heart, liver, lung and other organ dysfunction or have tumors;\n* Those with developmental malformations of the urinary system;\n* Those with autoimmune diseases and regular use of immunosuppressants;\n* Those with serious infectious diseases that are not under control;\n* Those with a history of infectious diseases such as HBV, HCV, HIV, syphilis;\n* History of surgery or acute trauma or blood loss exceeding 200ml within 3 months;\n* Participated in other clinical studies within 3 months;\n* Have received any cell product or derivative product treatment within 12 months;\n* Other circumstances that the researcher deems inappropriate for inclusion.","3 Years","12 Years",{"count":246,"type":22},40,[25,248],"PHASE3","The goal of this clinical trial is to evaluate the safety and efficacy of human umbilical cord mesenchymal stem cells (hUC-MSC) in the treatment of Alport syndrome (AS) in a randomized, single-blind, placebo-controlled trial, to provide a clinical basis for the development of stem cell products for the treatment of AS, and to further clarify the therapeutic effect of hUC-MSC in the treatment of AS.",[28],[64,252,253],"Human Umbilical Cord Mesenchymal Stem Cells","Randomized Clinical Trial","NOT_YET_RECRUITING","2024-12-29",{"date":257,"type":33},"2024-12-31",{"date":259,"type":22},"2025-01-01",{"date":261,"type":22},"2028-12-31",{"name":263,"class":75},"Guangzhou Women and Children's Medical Center",{"id":265,"slug":266,"hasResults":11,"nctId":267,"briefTitle":268,"officialTitle":269,"acronym":270,"eligibilityCriteria":271,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":272,"targetDuration":217,"studyType":51,"phases":4,"briefSummary":274,"conditions":275,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":356,"lastUpdatePostDateStruct":357,"startDateStruct":359,"completionDateStruct":361,"leadSponsor":363,"locationsCount":76},"100521150","national-registry-of-rare-kidney-diseases-100521150","NCT06065852","National Registry of Rare Kidney Diseases","National Registry of Rare Kidney Diseases (RaDaR)","RaDaR","* Kidney Rare Disease\n* Paeds and adults\n* Eligibility differs for each rare disease group\n* See: https:\u002F\u002Fukkidney.org\u002Frare-renal\u002Frecruitment",{"count":273,"type":22},35000,"The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.\n\nThe purpose of this research is to:\n\n* Develop Clinical Guidelines for specific rare kidney diseases. These are written recommendations on how to diagnose and treat a medical condition.\n* Audit treatments and outcomes. An audit makes checks to see if what should be done is being done and asks if it could be done better.\n* Further the development of future treatments.\n\nParticipants will be invited to participate on clinical trials and other studies. The registry has the capacity to feedback relevant information to patients and in conjunction with Patient Knows Best (Home - Patients Know Best), allows patients to provide information themselves, including their own reported quality of life and outcome measures.",[276,277,278,279,28,280,281,282,283,284,285,286,287,288,289,290,291,292,293,294,295,296,297,298,299,300,301,302,58,303,304,305,306,307,308,309,310,311,94,312,313,314,315,316,317,318,319,320,321,322,323,324,325,326,327,328,329,97,330,331,332,333,334,335,336,337,338,339,340,341,342,343,344,345,346,347,348,349,350,351,352,353,354,355],"Adenine Phosphoribosyltransferase Deficiency","AH Amyloidosis","AHL Amyloidosis","AL Amyloidosis","Atypical Hemolytic Uremic Syndrome","Autoimmune Distal Renal Tubular Acidosis","Autosomal Recessive Proximal Renal Tubular Acidosis","Autosomal Recessive Distal Renal Tubular Acidosis","Autosomal Dominant Polycystic Kidney Disease","Autosomal Recessive Polycystic Kidney Disease","Bartter Syndrome","BK Nephropathy","C3 Glomerulopathy With Monoclonal Gammopathy","C3 Glomerulopathy","Calciphylaxis","Crystalglobulinaemia","Crystal-storing Histiocytosis","Cystinosis","Cystinuria","Dense Deposit Disease","Dent Disease","Denys-Drash Syndrome","Dominant Hypophosphataemia With Nephrolithiasis and\u002For Osteoporosis","Drug Induced Fanconi Syndrome","Drug-Induced Hypomagnesemia","Drug-Induced Nephrogenic Diabetes Insipidus","Epilepsy, Ataxia, Sensorineural Deafness and Tubulopathy","Familial Hypomagnesemia With Hypercalciuria and Nephrocalcinosis","Familial Primary Hypomagnesemia With Hypocalcuria","Familial Primary Hypomagnesaemia With Normocalciuria","Familial Renal Glucosuria","Fanconi Renotubular Syndrome 1","Fanconi Renotubular Syndrome 2","Fanconi Renotubular Syndrome 3","Fibrillary Glomerulonephritis","Fibromuscular Dysplasia","Generalised Pseudohypoaldosteronism Type 1","Gitelman Syndrome","Heavy-Metal-Induced Fanconi Syndrome","Hepatocyte Nuclear Factor 1-Beta-Associated Monogenic Diabetes","Hereditary Renal Hypouricemia","Hereditary Hypophosphatemic Rickets With Hypercalciuria","Hyperuricaemic Nephropathy","IgA Nephropathy","Immunotactoid Glomerulonephritis With Organised Microtubular Mononoclonal Immunoglobulin Deposits","Inherited Renal Cancer Syndromes","Intracapillary Monoclonal IgM Without Cryoglobulin","Intraglomerular\u002FCapillary Lymphoma\u002FLeukaemia","Isolated Autosomal Dominant Hypomagnesaemia Glaudemans Type","Liddle Syndrome","Light Chain Cast Nephropathy","Light Chain Proximal Tubulopathy Without Crystals","Light Chain Proximal Tubulopathy With Crystals","Lowe Syndrome","Membranoproliferative Glomerulonephritis","Medullary Cystic Kidney Disease","Minimal Change Nephropathy","Mitochondrial Disease Of The Kidney","Monoclonal Immunoglobulin Deposition Disease","Nail Patella Syndrome","Nephrogenic Diabetes Insipidus","Nephrogenic Syndrome of Inappropriate Antidiuresis","Nephronophthisis","Primary Hypomagnesemia With Secondary Hypocalcemia","Primary Hyperoxaluria","Proliferative Glomerulonephritis With Monoclonal IgG Deposits","Proximal Tubulopathy Without Crystals","Pseudohypoaldosteronism Type 1, 2A-2E","Pure Red Cell Aplasia","Retroperitoneal Fibrosis","Sickle Cell Nephropathy","Shiga Toxin Associated Haemolytic Uraemic Syndrome","Steroid Resistant Nephrotic Syndrome","Steroid-Sensitive Nephrotic Syndrome","Thin Basement Membrane Nephropathy","Thrombotic Microangiopathy With Monoclonal Gammopathy","Type 1 Cryoglobulinaemic Glomerulonephritis","Tuberous Sclerosis","Unclassified Monoclonal Gammopathy Of Renal Significance","Vasculitis","2023-09-26",{"date":358,"type":33},"2023-10-04",{"date":360,"type":33},"2009-11-06",{"date":362,"type":22},"2039-12-31",{"name":364,"class":75},"UK Kidney Association",{"id":366,"slug":367,"hasResults":11,"nctId":368,"briefTitle":369,"officialTitle":370,"acronym":4,"eligibilityCriteria":371,"healthyVolunteers":11,"sex":17,"minAge":217,"maxAge":372,"enrollmentInfo":373,"targetDuration":4,"studyType":23,"phases":375,"briefSummary":376,"conditions":377,"keywords":4,"overallStatus":254,"whyStopped":4,"lastUpdateSubmitDate":378,"lastUpdatePostDateStruct":379,"startDateStruct":381,"completionDateStruct":383,"leadSponsor":385,"locationsCount":76},"100449482","safety-and-efficacy-of-acei-in-alport-syndrome-patients-with-col4a3col4a4col4a5-variants-100449482","NCT05133050","Safety and Efficacy of ACEI in Alport Syndrome Patients With COL4A3\u002FCOL4A4\u002FCOL4A5 Variants","Safety and Efficacy of Early Angiotensin-converting Enzyme Inhibition in Patients With Alport Syndrome Carrying Pathogenic Heterozygous COL4A3,COL4A4 or COL4A5 Mutations","Inclusion Criteria:\n\n1. Age: 30-50 Years;\n2. Sex: All;\n3. Alport syndrome patients with variants of COL4A3\u002FCOL4A4\u002FCOL4A5; hematuria or microalbuminuria; eGFR\\>90 mL\u002Fmin\u002F1.73m2;\n4. Patients with microscopic hematuria only;\n5. Patients with microscopic hematuria and microalbuminuria: 30-300mg\u002F24h or urine albumin\u002Fcreatinine: 30-300mg\u002Fg;\n6. No angiotensin converting enzyme inhibitor (ACEI) and other renin-angiotensin system inhibitors (including angiotensin II receptor antagonists, etc.) treatment.\n\nExclusion Criteria:\n\n1. With primary or secondary kidney disease, including IgA nephropathy, membranous nephropathy, lupus nephropathy, benign renal arterioles, etc.;\n2. Patients with a history of angioedema;\n3. Hypovolemia or hypotension (systolic blood pressure less than 90mmHg and\u002For diastolic blood pressure less than 60mmHg);\n4. Pregnant and lactating women;\n5. Patients with bilateral renal artery stenosis or unilateral renal artery stenosis with solitary kidney;\n6. Hyperkalemia, blood potassium\\>5.5mmol\u002FL;\n7. Severe aortic stenosis, severe mitral stenosis;\n8. Treatment of drug allergy;\n9. Hypertension or other diseases that may require treatment with angiotensin-converting enzyme inhibitors;\n10. Disagree to participate in this research.","50 Years",{"count":374,"type":22},510,[90],"Alport syndrome (AS) is the second most common monogenic cause of end-stage renal failure (ESRF). AS is caused by variants in the COL4A3, COL4A4, and COL4A5 genes, which encode for the a3, a4, and a5 chains of type IV collagen. This trial is a prospective, randomized, controlled and multicenter trial. Mainly to assess the safety and efficacy of ramipril in Alport syndrome patients with variants of COL4A3\u002FCOL4A4\u002FCOL4A5.",[28],"2021-11-23",{"date":380,"type":33},"2021-11-24",{"date":382,"type":22},"2022-01-01",{"date":384,"type":22},"2026-12-31",{"name":386,"class":75},"Xinhua Hospital, Shanghai Jiao Tong University School of Medicine",{"id":388,"slug":389,"hasResults":11,"nctId":390,"briefTitle":391,"officialTitle":392,"acronym":4,"eligibilityCriteria":393,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":394,"targetDuration":4,"studyType":51,"phases":4,"briefSummary":396,"conditions":397,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":398,"lastUpdatePostDateStruct":399,"startDateStruct":401,"completionDateStruct":403,"leadSponsor":405,"locationsCount":76},"100435254","genotype-phenotype-correlations-in-patients-with-alport-syndrome-100435254","NCT04947813","Genotype-Phenotype Correlations in Patients With Alport Syndrome","Association Analysis Between Variants of COL4A3\u002FCOL4A4\u002FCOL4A5 and Alport Syndrome in the Han Chinese Population","Inclusion Criteria:\n\n1. Age: up to 99 Years (Child, Adult, Older Adult)\n2. Sex: All;\n3. Families and patients with a history of renal hematuria;\n4. Those who signed the informed consent.\n\nExclusion Criteria:\n\n1. Polycystic kidney disease, hypertensive nephropathy, etc.;\n2. Kidney biopsy is diagnosed as other primary\u002Fsecondary kidney disease without type IV collagen-related kidney disease, including IgA nephropathy, membranous nephropathy, lupus nephritis, etc.\n3. Incomplete medical history or clinical data.",{"count":395,"type":22},8165,"Alport syndrome (AS) is caused by pathogenic variants in the type IV collagen genes COL4A3, COL4A4, and COL4A5. This study aims to enroll families and patients with a history of renal hematuria in 27 hospitals and detect these three genes for AS screening. This study also aims to analysis the effect of COL4A3\u002FCOL4A4\u002FCOL4A5 genotype on the development of kidney disease.",[28],"2021-06-24",{"date":400,"type":33},"2021-07-01",{"date":402,"type":33},"2021-01-01",{"date":404,"type":22},"2030-12-31",{"name":386,"class":75}]