[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100599288":3},{"organization":4,"outcomesModule":7,"designInfo":71,"detailedDescription":82,"studyPopulation":46,"armGroups":83,"interventions":95,"overallOfficials":46,"centralContacts":102,"locations":111,"responsibleParty":582,"collaborators":46,"id":584,"slug":46,"hasResults":585,"nctId":586,"briefTitle":587,"officialTitle":588,"acronym":46,"eligibilityCriteria":589,"healthyVolunteers":585,"sex":590,"minAge":591,"maxAge":46,"enrollmentInfo":592,"targetDuration":46,"studyType":595,"phases":596,"briefSummary":598,"conditions":599,"keywords":602,"overallStatus":114,"whyStopped":46,"lastUpdateSubmitDate":603,"lastUpdatePostDateStruct":604,"startDateStruct":607,"completionDateStruct":609,"leadSponsor":611,"locationsCount":612},{"fullName":5,"class":6},"Azafaros B.V.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":16,"otherOutcomes":46},[9,13],{"measure":10,"description":11,"timeFrame":12},"Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score","Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia)","Baseline to month 18",{"measure":14,"description":15,"timeFrame":12},"Change from baseline in functional SARA score","Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment",[17,21,24,27,30,33,36,39,42,44,47,49,51,53,55,58,60,63,66,68],{"measure":18,"description":19,"timeFrame":20},"Change from baseline in SARA score for gait\u002Fposture","Assessed on a 9-point scale with higher scores indicating inability to complete the task","Baseline to months 6, 12, and 18",{"measure":22,"description":23,"timeFrame":20},"Change from baseline in SARA score for speech","Assessed on a 7-point scale with higher scores indicating unintelligible speech\u002Fanarthria",{"measure":25,"description":26,"timeFrame":20},"Change from baseline in SARA score for kinetics","Assessed on a 5-point scale with higher scores indicating more severe impairment",{"measure":28,"description":29,"timeFrame":20},"Change from baseline in Vineland Adaptive Behavior Scale (VABS)","Assesses four domains of function: communication, daily living skills, socialization, and motor skills",{"measure":31,"description":32,"timeFrame":20},"Change from baseline in Penetration-Aspiration Scale (PAS)","Assessed on an 8-point ordinal scale, with 1 representing the lowest and 8 the highest\u002Fmost severe score",{"measure":34,"description":35,"timeFrame":20},"Change from baseline in 9-Hole Peg Test (9-HPT-D)","Assessed by the number of pegs per second placed in a 50-second, 9-HPT using the dominant hand",{"measure":37,"description":38,"timeFrame":20},"Change from baseline in Goal Attainment Scale (GAS)","Assesses individual goals achieved during the study",{"measure":40,"description":41,"timeFrame":20},"Change from baseline in Clinician Global Impression of Change (CGI-C)","Assessed on a 7-point scale with higher scores indicating lower improvement",{"measure":43,"description":41,"timeFrame":20},"Change from baseline in Participant\u002FCaregiver Global Impression of Change (PGI-C)",{"measure":45,"description":46,"timeFrame":20},"Change from baseline in Seizure frequency and duration, as per the seizure diary",null,{"measure":48,"description":46,"timeFrame":12},"Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of an increase in total SARA score of ≥2 points",{"measure":50,"description":46,"timeFrame":12},"Time-to-event comparison between nizubaglustat and placebo over the course of the study for pre-defined detrimental events of an increase in functional SARA of ≥1.5 points",{"measure":52,"description":46,"timeFrame":12},"Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of a decrease in PAS of ≥1 point",{"measure":54,"description":46,"timeFrame":12},"Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of participants leaving the study due to participant\u002Fcaregiver perception of disease progression\u002Flack of efficacy",{"measure":56,"description":46,"timeFrame":57},"Maximum observed plasma concentration (Cmax)","Baseline and months 1,18, and 21",{"measure":59,"description":46,"timeFrame":57},"Time to Cmax (Tmax)",{"measure":61,"description":46,"timeFrame":62},"Plasma trough concentration (Ctrough)","Baseline and month 1",{"measure":64,"description":46,"timeFrame":65},"Area under the plasma concentration-time curve from time of dosing (zero) to 24 hours post-dose (AUC0-24) at baseline","Baseline (Day 1)",{"measure":67,"description":46,"timeFrame":57},"Accumulation ratio for Cmax",{"measure":69,"description":46,"timeFrame":70},"Change from baseline in plasma concentration of glucosylceramide (GlcCer) C16:0; 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To assess additional efficacy in ataxic and non-ataxic manifestations comparing nizubaglustat dosing with placebo when administered over 18 months in participants with late-infantile and juvenile forms of GM1\u002FGM2 gangliosidosis\n\nII. To assess the pharmacokinetic (PK) properties of nizubaglustat after administration of the first dose (Visit 1) and at steady state after multiple once daily doses\n\nIII. To assess the pharmacodynamic (PD) effects of nizubaglustat\n\nIV. To assess the safety and tolerability of daily oral nizubaglustat dosing compared with placebo, when administered over 18 months in participants with late-infantile and juvenile forms of GM1\u002FGM2 gangliosidosis",[84,90],{"label":85,"type":86,"description":87,"interventionNames":88},"Nizubaglustat","EXPERIMENTAL","Once daily oral dispersible tablets",[89],"Drug: AZ-3102",{"label":91,"type":92,"description":46,"interventionNames":93},"Placebo","PLACEBO_COMPARATOR",[94],"Drug: Placebo",[96,100],{"type":97,"name":98,"description":85,"armGroupLabels":99,"otherNames":46},"DRUG","AZ-3102",[85],{"type":97,"name":91,"description":91,"armGroupLabels":101,"otherNames":46},[91],[103,108],{"name":104,"role":105,"phone":106,"phoneExt":46,"email":107},"Patient Advocacy Representative","CONTACT","Please reach out by email","info@azafaros.com",{"name":109,"role":105,"phone":106,"phoneExt":46,"email":110},"Contact for Healthcare Professionals","medinfo@azafaros.com",[112,135,146,155,165,180,187,197,214,229,245,262,275,287,301,311,327,338,355,370,385,395,411,423,432,449,463,479,492,509,526,536,546,556,568,573],{"facility":113,"status":114,"city":115,"state":116,"zip":117,"country":118,"countryCode":119,"cosmosGeoPoint":120,"geoPoint":125,"contacts":126},"UCSF Children's Hospital and Research Center at Oakland","RECRUITING","Oakland","California","94609","United States","US",{"type":121,"coordinates":122},"Point",[123,124],-122.2708,37.80437,{"lat":124,"lon":123},[127,132],{"name":128,"role":105,"phone":129,"phoneExt":130,"email":131},"Veronica Cheung, CRC","510-428-3885","2752","Veronica.Cheung@ucsf.edu",{"name":133,"role":134,"phone":46,"phoneExt":46,"email":46},"Caroline Hastings, MD","PRINCIPAL_INVESTIGATOR",{"facility":136,"status":137,"city":138,"state":139,"zip":140,"country":118,"countryCode":119,"cosmosGeoPoint":141,"geoPoint":145,"contacts":46},"University of Minnesota Medical School","NOT_YET_RECRUITING","Minneapolis","Minnesota","55455",{"type":121,"coordinates":142},[143,144],-93.26384,44.97997,{"lat":144,"lon":143},{"facility":147,"status":114,"city":148,"state":139,"zip":149,"country":118,"countryCode":119,"cosmosGeoPoint":150,"geoPoint":154,"contacts":46},"Mayo Clinic Children's Center - PIN","Rochester","55905",{"type":121,"coordinates":151},[152,153],-92.4699,44.02163,{"lat":153,"lon":152},{"facility":156,"status":114,"city":157,"state":158,"zip":159,"country":118,"countryCode":119,"cosmosGeoPoint":160,"geoPoint":164,"contacts":46},"Children's Medical Center Dallas","Dallas","Texas","75235",{"type":121,"coordinates":161},[162,163],-96.80667,32.78306,{"lat":163,"lon":162},{"facility":166,"status":114,"city":167,"state":168,"zip":169,"country":118,"countryCode":119,"cosmosGeoPoint":170,"geoPoint":174,"contacts":175},"Lysosomal Rare Disorders Research and Treatment Center","Fairfax","Virginia","22030-7404",{"type":121,"coordinates":171},[172,173],-77.30637,38.84622,{"lat":173,"lon":172},[176],{"name":177,"role":105,"phone":178,"phoneExt":46,"email":179},"Lauren Noll","571-732-4655","lnoll@ldrtc.org",{"facility":181,"status":114,"city":182,"state":183,"zip":184,"country":185,"countryCode":186,"cosmosGeoPoint":46,"geoPoint":46,"contacts":46},"Hospital Universitario Austral","Ciudad Autónoma Buenos Aires","Buenos Aires","B1629AHJ","Argentina","AR",{"facility":188,"status":114,"city":189,"state":190,"zip":191,"country":185,"countryCode":186,"cosmosGeoPoint":192,"geoPoint":196,"contacts":46},"Hospital de Niños de La Santisima Trinidad","Córdoba","Córdoba Province","X5004 ASL",{"type":121,"coordinates":193},[194,195],-64.18853,-31.40648,{"lat":195,"lon":194},{"facility":198,"status":114,"city":199,"state":200,"zip":201,"country":202,"countryCode":203,"cosmosGeoPoint":204,"geoPoint":208,"contacts":209},"Women's and Children's Hospital","North Adelaide","South Australia","5006","Australia","AU",{"type":121,"coordinates":205},[206,207],138.59141,-34.90733,{"lat":207,"lon":206},[210],{"name":211,"role":105,"phone":212,"phoneExt":46,"email":213},"Louise Jaensch","+61 881 616 610","louise.jaensch@sa.gov.au",{"facility":215,"status":137,"city":216,"state":217,"zip":218,"country":202,"countryCode":203,"cosmosGeoPoint":219,"geoPoint":223,"contacts":224},"Royal Children's Hospital Melbourne - PIN","Parkville","Victoria","3052",{"type":121,"coordinates":220},[221,222],144.95,-37.78333,{"lat":222,"lon":221},[225],{"name":226,"role":105,"phone":227,"phoneExt":46,"email":228},"Heidi Peters","+61 03 9345 6205","metabolictrials@mcri.edu.au",{"facility":230,"status":114,"city":231,"state":231,"zip":232,"country":233,"countryCode":234,"cosmosGeoPoint":235,"geoPoint":239,"contacts":240},"Instituto Fernandes Figueira","Rio de Janeiro","22250-020","Brazil","BR",{"type":121,"coordinates":236},[237,238],-43.18223,-22.90642,{"lat":238,"lon":237},[241],{"name":242,"role":105,"phone":243,"phoneExt":46,"email":244},"DAFNE D. 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Clinic Ltd. Metabolics and Genetics in Calgary","Calgary","Alberta","T3B 6A8","Canada","CA",{"type":121,"coordinates":283},[284,285],-114.08529,51.05011,{"lat":285,"lon":284},{"facility":288,"status":137,"city":289,"state":278,"zip":290,"country":280,"countryCode":281,"cosmosGeoPoint":291,"geoPoint":295,"contacts":296},"University of Alberta Medical Genetics Clinic","Edmonton","T6G 2B7",{"type":121,"coordinates":292},[293,294],-113.46871,53.55014,{"lat":294,"lon":293},[297],{"name":298,"role":105,"phone":299,"phoneExt":46,"email":300},"Casey Brodie","+1 780-492-9277","casey.brodey@albertahealthservices.ca",{"facility":302,"status":137,"city":303,"state":304,"zip":305,"country":280,"countryCode":281,"cosmosGeoPoint":306,"geoPoint":310,"contacts":46},"Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis","Montreal","Quebec","H2X 0C1",{"type":121,"coordinates":307},[308,309],-73.58781,45.50884,{"lat":309,"lon":308},{"facility":312,"status":114,"city":313,"state":46,"zip":314,"country":315,"countryCode":316,"cosmosGeoPoint":317,"geoPoint":321,"contacts":322},"AP-HP - Hôpital Armand Trousseau","Paris","75012","France","FR",{"type":121,"coordinates":318},[319,320],2.3488,48.85341,{"lat":320,"lon":319},[323],{"name":324,"role":105,"phone":325,"phoneExt":46,"email":326},"Aurelie Phelep","+33 01-71-73-82-16","a.phelep@institut-myologie.org",{"facility":328,"status":114,"city":329,"state":46,"zip":330,"country":331,"countryCode":332,"cosmosGeoPoint":333,"geoPoint":337,"contacts":46},"SphinCS GmbH","Höchheim","65239","Germany","DE",{"type":121,"coordinates":334},[335,336],10.45,50.36667,{"lat":336,"lon":335},{"facility":339,"status":114,"city":340,"state":341,"zip":342,"country":343,"countryCode":344,"cosmosGeoPoint":345,"geoPoint":349,"contacts":350},"Amrita Institute of Medical Sciences and Research Centre","Ernākulam","Kerala","682041","India","IN",{"type":121,"coordinates":346},[347,348],76.29036,9.96714,{"lat":348,"lon":347},[351],{"name":352,"role":105,"phone":353,"phoneExt":46,"email":354},"Sheela Nampoothiri","+91 9447978222","sheelanampoothiri@aims.amrita.edu",{"facility":356,"status":114,"city":357,"state":358,"zip":359,"country":343,"countryCode":344,"cosmosGeoPoint":360,"geoPoint":364,"contacts":365},"All India Institute of Medical Sciences (AIIMS) - New Delhi","New Delhi","National Capital Territory of Delhi","110029",{"type":121,"coordinates":361},[362,363],77.2148,28.62137,{"lat":363,"lon":362},[366],{"name":367,"role":105,"phone":368,"phoneExt":46,"email":369},"Faijan Beg","+91 78283 96432","faijan.crds@outlook.com",{"facility":371,"status":114,"city":372,"state":373,"zip":374,"country":343,"countryCode":344,"cosmosGeoPoint":375,"geoPoint":379,"contacts":380},"JK Lone Hospital","Jaipur","Rajasthan","302004",{"type":121,"coordinates":376},[377,378],75.78781,26.91962,{"lat":378,"lon":377},[381],{"name":382,"role":105,"phone":383,"phoneExt":46,"email":384},"Priyanshu Mathur","+91 9982451490","Priyanshujkl@gmail.com",{"facility":386,"status":137,"city":387,"state":388,"zip":389,"country":343,"countryCode":344,"cosmosGeoPoint":390,"geoPoint":394,"contacts":46},"Christian Medical College and Hospital","Vellore","Tamil Nadu","632004",{"type":121,"coordinates":391},[392,393],79.13255,12.9184,{"lat":393,"lon":392},{"facility":396,"status":114,"city":397,"state":46,"zip":398,"country":399,"countryCode":400,"cosmosGeoPoint":401,"geoPoint":405,"contacts":406},"Fondazione IRCCS Istituto Neurologico Carlo Besta","Milan","20133","Italy","IT",{"type":121,"coordinates":402},[403,404],12.59836,42.78235,{"lat":404,"lon":403},[407],{"name":408,"role":105,"phone":409,"phoneExt":46,"email":410},"Anna Ardissone","+39 0223942210","anna.ardissone@istituto-besta.it",{"facility":412,"status":137,"city":413,"state":414,"zip":415,"country":416,"countryCode":417,"cosmosGeoPoint":418,"geoPoint":422,"contacts":46},"Hospital Universitario Dr. Jose Eleuterio González","Monterrey","Nuevo León","64460","Mexico","MX",{"type":121,"coordinates":419},[420,421],-100.31721,25.68435,{"lat":421,"lon":420},{"facility":424,"status":137,"city":425,"state":46,"zip":426,"country":416,"countryCode":417,"cosmosGeoPoint":427,"geoPoint":431,"contacts":46},"Centenario Hospital Miguel Hidalgo","Aguascalientes","20000",{"type":121,"coordinates":428},[429,430],-102.2843,21.88262,{"lat":430,"lon":429},{"facility":433,"status":114,"city":434,"state":435,"zip":436,"country":437,"countryCode":438,"cosmosGeoPoint":439,"geoPoint":443,"contacts":444},"ULS de Santo António, EPE - Centro Materno Infantil Norte","Porto","Porto District","4050-651","Portugal","PT",{"type":121,"coordinates":440},[441,442],-8.61097,41.1485,{"lat":442,"lon":441},[445],{"name":446,"role":105,"phone":447,"phoneExt":46,"email":448},"Anabela Bandeira","+35 915676520","anabelabandeira.dca@chporto.min-saude.pt",{"facility":450,"status":114,"city":451,"state":46,"zip":452,"country":437,"countryCode":438,"cosmosGeoPoint":453,"geoPoint":457,"contacts":458},"ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS","Lisbon","1649-035",{"type":121,"coordinates":454},[455,456],-9.1498,38.72509,{"lat":456,"lon":455},[459],{"name":460,"role":105,"phone":461,"phoneExt":46,"email":462},"Tiago Proenca dos Santos","+351 210405814","cic@ulssm.min.saude.pt",{"facility":464,"status":114,"city":465,"state":465,"zip":466,"country":467,"countryCode":468,"cosmosGeoPoint":469,"geoPoint":473,"contacts":474},"Hospital Universitario Vall d'Hebron - PPDS","Barcelona","8035","Spain","ES",{"type":121,"coordinates":470},[471,472],2.15899,41.38879,{"lat":472,"lon":471},[475],{"name":476,"role":105,"phone":477,"phoneExt":46,"email":478},"Nuria Leal","+34677171367","nuria.leal@vhir.org",{"facility":480,"status":114,"city":481,"state":481,"zip":482,"country":467,"countryCode":468,"cosmosGeoPoint":483,"geoPoint":487,"contacts":488},"Hospital Infantil Universitario Niño Jesus - PIN","Madrid","28009",{"type":121,"coordinates":484},[485,486],-3.70256,40.4165,{"lat":486,"lon":485},[489],{"name":490,"role":105,"phone":46,"phoneExt":46,"email":491},"Beatriz Bernardino Cuesta","neurologia.hnjs@salud.madrid.org",{"facility":493,"status":114,"city":494,"state":495,"zip":496,"country":497,"countryCode":498,"cosmosGeoPoint":499,"geoPoint":503,"contacts":504},"Sahlgrenska universitetssjukhuset Östra","Gothenburg","Västra Götaland County","416 50","Sweden","SE",{"type":121,"coordinates":500},[501,502],11.96679,57.70716,{"lat":502,"lon":501},[505],{"name":506,"role":105,"phone":507,"phoneExt":46,"email":508},"Niklas Darin","0046 313438213","niklas.darin@vgregim.se",{"facility":510,"status":114,"city":511,"state":512,"zip":513,"country":514,"countryCode":515,"cosmosGeoPoint":516,"geoPoint":520,"contacts":521},"Inselspital - Universitätsspital Bern","Bern","Canton of Bern","3010","Switzerland","CH",{"type":121,"coordinates":517},[518,519],7.44744,46.94809,{"lat":519,"lon":518},[522],{"name":523,"role":105,"phone":524,"phoneExt":46,"email":525},"Sandrina Schneider","+41 31 63 2 07 41","sandrina.schneider@insel.ch",{"facility":527,"status":137,"city":528,"state":528,"zip":529,"country":530,"countryCode":46,"cosmosGeoPoint":531,"geoPoint":535,"contacts":46},"Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi","Adana","1250","Turkey (Türkiye)",{"type":121,"coordinates":532},[533,534],35.32531,36.98615,{"lat":534,"lon":533},{"facility":537,"status":114,"city":538,"state":539,"zip":540,"country":530,"countryCode":46,"cosmosGeoPoint":541,"geoPoint":545,"contacts":46},"Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi","Çankaya","Ankara","6500",{"type":121,"coordinates":542},[543,544],32.86268,39.9179,{"lat":544,"lon":543},{"facility":547,"status":114,"city":548,"state":549,"zip":550,"country":530,"countryCode":46,"cosmosGeoPoint":551,"geoPoint":555,"contacts":46},"Ege Universitesi Tip Fakultesi","Bornova","İzmir","35100",{"type":121,"coordinates":552},[553,554],27.2399,38.47921,{"lat":554,"lon":553},{"facility":557,"status":137,"city":558,"state":559,"zip":560,"country":561,"countryCode":562,"cosmosGeoPoint":563,"geoPoint":567,"contacts":46},"University College London Hospitals (UCLH)","London","Middlesex","WC1N 3BG","United Kingdom","UK",{"type":121,"coordinates":564},[565,566],-0.12574,51.50853,{"lat":566,"lon":565},{"facility":569,"status":137,"city":558,"state":46,"zip":46,"country":561,"countryCode":562,"cosmosGeoPoint":570,"geoPoint":572,"contacts":46},"Great Ormond Street Hospital NHSFT",{"type":121,"coordinates":571},[565,566],{"lat":566,"lon":565},{"facility":574,"status":137,"city":575,"state":46,"zip":576,"country":561,"countryCode":562,"cosmosGeoPoint":577,"geoPoint":581,"contacts":46},"Royal Manchester Children's Hospital","Manchester","M13 9WL",{"type":121,"coordinates":578},[579,580],-2.23743,53.48095,{"lat":580,"lon":579},{"type":583,"investigatorFullName":46,"investigatorTitle":46,"investigatorAffiliation":46,"oldNameTitle":46,"oldOrganization":46},"SPONSOR","100599288",false,"NCT07082543","A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis","18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis","Inclusion Criteria:\n\n* Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant\n* Male and female participants aged 4 years and older at the time of informed consent\n* Onset of neurological symptoms from 1 to 10 years\n* Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline\n* Females of childbearing potential who are sexually active willing to follow the contraceptive guidance\n* Male participants with a female partner of childbearing potential willing to follow the contraceptive guidance\n\nExclusion Criteria:\n\n* A history of medical conditions other than GM1 or GM2 gangliosidosis that, in the opinion of the Principal Investigator, would confound scientific rigor or the interpretation of results\n* Body weight of \\\u003C10 kg\n* The presence of another neurologic disease\n* The presence of moderate or severe hepatic impairment\n* The presence of moderate or severe renal impairment\n* Platelet count of \\\u003C100x10\\^9\u002FL\n* The dose of any anti-epileptic treatment(s) was not stable (required a change in dose within the previous 3 months) and\u002For a new anti-epileptic treatment (drug or procedure) was prescribed in the month before Baseline\n* Prior use of an investigational drug within the 3 months before Screening; or prior participation in a clinical study involving gene therapy or stem cell transplantation within 2 years prior to Screening\n* A positive serum pregnancy test (for women of childbearing potential)","ALL","4 Years",{"count":593,"type":594},75,"ESTIMATED","INTERVENTIONAL",[597],"PHASE3","An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2 gangliosidosis",[600,601],"Gangliosidoses, GM2","Gangliosidosis, GM1",[85],"2026-06-17",{"date":605,"type":606},"2026-06-18","ACTUAL",{"date":608,"type":606},"2025-06-30",{"date":610,"type":594},"2027-11-04",{"name":5,"class":6},36]