[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07159841":3,"trial-entities:NCT07159841":462,"trial-summary:NCT07159841":466},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":21,"study_type":27,"primary_purpose":28,"phases":29,"enrollment_info":32,"interventions":35,"primary_outcomes":49,"secondary_outcomes":59,"sex":78,"minimum_age":79,"maximum_age":80,"healthy_volunteers":81,"eligibility_criteria":82,"std_ages":86,"locations":88,"central_contacts":453,"overall_officials":459,"references":460,"see_also_links":461},"NCT07159841","CRN04894-13","A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)","A Phase 2\u002F3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Atumelnant Treatment in Pediatric Participants With Congenital Adrenal Hyperplasia Including a Long-Term Extension","RECRUITING","2030-03","2026-06","2026-09-02","2026-01-22","Crinetics Pharmaceuticals Inc.","INDUSTRY",true,"The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).","This Phase 2\u002F3 plus open-label extension study is designed to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic CAH. Part A is a Phase 2, open-label, semi-sequential cohorts portion of the study. Part B is the Phase 3, double-blind, randomized, placebo controlled confirmatory portion of the study. Part C is the open-label extension (OLE) portion of the study. Participants in Part A and B are eligible to enroll in Part C (OLE).\n\nA total of approximately 153 participants may be enrolled in the study (planned and optional cohorts) ages 1 to \\\u003C 18 years old.\n\nThe first 3 cohorts in Part A are for ages 12 to \\\u003C18 years and will be semi-sequential, and Safety Review Committee (SRC) review of data and approval to proceed is required prior to enrolling each subsequent cohort. The fourth cohort in Part A is for ages 1 to 11 years old and will begin after Cohorts 1 and 2 have been completed, additional requirements are fulfilled, and following SRC review of Cohorts 1 and 2 data.",[19,20],"Congenital Adrenal Hyperplasia","Classic Congenital Adrenal Hyperplasia",[19,22,23,24,25,26],"CAH","CRN04894","Atumelnant","Pediatric","Balance-CAH","INTERVENTIONAL","TREATMENT",[30,31],"PHASE2","PHASE3",{"count":33,"type":34},153,"ESTIMATED",[36,44],{"type":37,"name":24,"description":38,"armGroupLabels":39,"otherNames":43},"DRUG","Atumelnant, tablets, once daily by mouth, weight-based dosing",[40,41,42],"Active Treatment (Part B)","Open-Label Treatment (Part C)","Treatment (Part A)",[23],{"type":37,"name":45,"description":46,"armGroupLabels":47},"Placebo","Placebo, tablets, once daily by mouth, weight-based dosing",[48],"Placebo (Part B)",[50,53,56],{"measure":51,"timeFrame":52},"Change from baseline in morning serum androstenedione (A4) (Part A)","Week 8",{"measure":54,"timeFrame":55},"Percent change from baseline in glucocorticoid (GC) daily dose while serum early morning A4 ≤Upper Limit of Normal (ULN) (Part B)","Week 28",{"measure":57,"timeFrame":58},"Change from baseline in serum early morning A4 over time (Part C)","Up to Week 260",[60,62,65,68,70,72,74,76],{"measure":61,"timeFrame":52},"Change from baseline in morning serum 17-hydroxyprogesterone (17-OHP) (Part A)",{"measure":63,"timeFrame":64},"Plasma and\u002For blood concentrations of atumelnant (Part A)","Up to Week 8",{"measure":66,"timeFrame":67},"Change from baseline in serum early morning A4 (Part B)","Week 4",{"measure":69,"timeFrame":67},"Change from baseline in serum early morning 17-OHP (Part B)",{"measure":71,"timeFrame":55},"Proportion of participants with physiologic GC dose while serum early morning A4 ≤ULN (Part B)",{"measure":73,"timeFrame":58},"Change from baseline in serum early morning 17-OHP over time (Part C)",{"measure":75,"timeFrame":58},"Percent change from baseline in GC daily dose over time (Part C)",{"measure":77,"timeFrame":58},"Proportion of participants with physiologic GC dose while serum early morning A4 ≤ULN over time (Part C)","ALL","1 Year","17 Years",false,{"inclusion":83,"exclusion":84,"raw_text":85},[],[],"Inclusion Criteria:\n\nPart A and B participants are eligible to be included in the study only if all of the following criteria apply:\n\n1. Male or female at birth, between 1 to \\\u003C18 years of chronological age at the time of signing the Informed Consent Form (ICF).\n2. Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency (21-OHD) based on standard medically accepted criteria such as elevated 17-OHP level, confirmed CYP21A2 genetic testing, positive newborn screening with confirmatory second tier testing, or cosyntropin stimulation.\n3. Participants must have an elevated morning serum A4 level \\>ULN during Screening obtained prior to morning glucocorticoid (GC) administration.\n4. Participants must be on a stable supraphysiologic GC replacement therapy for at least one month prior to Screening.\n5. Compliance, as judged per Investigator discretion, with GC replacement and mineralocorticoid replacement (if applicable) regimen documented during the Screening Period.\n6. Biochemical euthyroidism as determined by the Investigator.\n\nPart C inclusion criteria require participants to complete treatment in either Part A or Part B and in the Investigator's opinion it would benefit the participant to continue in Part C, regardless of age.\n\nExclusion Criteria:\n\nPart A and Part B: Individuals in Part A and Part B who meet any of the following criteria will be excluded from participation in this study:\n\n1. Diagnosis of any form of CAH other than classic 21-OHD.\n2. Participants treated with other GCs within 30 days of Screening.\n3. Stress dose of GC therapy within 2 weeks of start of Screening, defined as any dose above the normal maintenance dose, including but not limited to intravenous (IV) or intramuscular (IM) hydrocortisone.\n4. Use of growth hormones within 1 week of start of Screening for short acting, or within 6 weeks of start of Screening for long acting.\n5. Use of a corticotropin-releasing factor receptor antagonist within 14 days of Screening.\n6. History of cancer excluding cured\u002Ftreated dermal squamous or basal cell carcinoma or cervical carcinoma in situ.\n7. Abnormal sleep\u002Fwake cycles (as determined by the Investigator).\n8. Female participants who are pregnant or lactating.\n9. Participants who have been dosed with an investigational drug (including atumelnant) in any prior clinical study within 60 days or 5 half-lives (whichever is longer) prior to the first dose.\n10. Individuals in Part C who do not meet the Part C Inclusion Criteria.",[87],"CHILD",[89,98,106,114,122,130,137,145,153,161,166,174,180,184,188,198,206,214,222,231,239,247,255,264,272,278,287,294,302,309,316,323,330,334,343,351,358,367,375,383,391,400,409,416,424,432,439,446],{"facility":90,"status":8,"city":91,"state":92,"zip":93,"country":94,"geoPoint":95},"UCSF Ron Conway Gateway Medical Building","San Francisco","California","94158","United States",{"lat":96,"lon":97},37.77493,-122.41942,{"facility":99,"status":8,"city":100,"state":101,"zip":102,"country":94,"geoPoint":103},"Boston's Children's Hospital","Boston","Massachusetts","02115",{"lat":104,"lon":105},42.35843,-71.05977,{"facility":107,"status":8,"city":108,"state":109,"zip":110,"country":94,"geoPoint":111},"University of Michigan","Ann Arbor","Michigan","48109",{"lat":112,"lon":113},42.27756,-83.74088,{"facility":115,"status":8,"city":116,"state":117,"zip":118,"country":94,"geoPoint":119},"University of Minnesota","Minneapolis","Minnesota","55454",{"lat":120,"lon":121},44.97997,-93.26384,{"facility":123,"status":8,"city":124,"state":125,"zip":126,"country":94,"geoPoint":127},"Rutgers Robert Wood Johnson Medical School","New Brunswick","New Jersey","08901",{"lat":128,"lon":129},40.48622,-74.45182,{"facility":131,"status":8,"city":132,"state":132,"zip":133,"country":94,"geoPoint":134},"Icahn School of Medicine at Mount Sinai-Mount Sinai Hospital","New York","10029",{"lat":135,"lon":136},40.71427,-74.00597,{"facility":138,"status":8,"city":139,"state":140,"zip":141,"country":94,"geoPoint":142},"Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"lat":143,"lon":144},39.95238,-75.16362,{"facility":146,"status":8,"city":147,"state":148,"zip":149,"country":94,"geoPoint":150},"Cook Children's Health Care System","Fort Worth","Texas","76104",{"lat":151,"lon":152},32.72541,-97.32085,{"facility":154,"status":8,"city":155,"state":156,"zip":157,"country":94,"geoPoint":158},"University of Virginia Health System","Charlottesville","Virginia","22903",{"lat":159,"lon":160},38.02931,-78.47668,{"facility":162,"status":8,"city":163,"state":163,"zip":164,"country":165},"Instituto de Investigaciones Metabólicas","Buenos Aires","C1012AAR","Argentina",{"facility":167,"status":8,"city":168,"state":169,"zip":170,"country":165,"geoPoint":171},"Hospital de Niños de la Santísima Trinidad","Córdoba","Córdoba Province","X5000",{"lat":172,"lon":173},-31.40648,-64.18853,{"facility":175,"status":8,"city":163,"zip":176,"country":165,"geoPoint":177},"Hospital Italiano de Buenos Aires","C1199ABB",{"lat":178,"lon":179},-34.61315,-58.37723,{"facility":181,"status":8,"city":163,"zip":182,"country":165,"geoPoint":183},"Instituto Médico Especializado (IME)","C1405BCH",{"lat":178,"lon":179},{"facility":185,"status":8,"city":163,"zip":186,"country":165,"geoPoint":187},"CEDIE \"Centro de Investigaciones Endocrinológicas\", CONICET-FEI División de Endocrinología, Hosp de Niños Ricardo Gutiérrez","C1425EFD",{"lat":178,"lon":179},{"facility":189,"status":190,"city":191,"state":192,"zip":193,"country":194,"geoPoint":195},"Institute of Endocrinology of Diabetes, The Children's Hospital at Westmead","NOT_YET_RECRUITING","Westmead","New South Wales","2145","Australia",{"lat":196,"lon":197},-33.80383,150.98768,{"facility":199,"status":190,"city":200,"state":201,"zip":202,"country":194,"geoPoint":203},"Queensland Children's Hospital","South Brisbane","Queensland","4101",{"lat":204,"lon":205},-27.48034,153.02049,{"facility":207,"status":8,"city":208,"state":209,"zip":210,"country":194,"geoPoint":211},"Monash Children's Hospital, Monash Health","Clayton","Victoria","3168",{"lat":212,"lon":213},-37.91667,145.11667,{"facility":215,"status":190,"city":216,"state":217,"zip":218,"country":194,"geoPoint":219},"Perth Children's Hospital","Nedlands","Western Australia","6009",{"lat":220,"lon":221},-31.98184,115.8073,{"facility":223,"status":8,"city":224,"state":225,"zip":226,"country":227,"geoPoint":228},"UZA (Antwerp University Hospital)","Edegem","Antwerp","2650","Belgium",{"lat":229,"lon":230},51.15662,4.44504,{"facility":232,"status":8,"city":233,"state":234,"zip":235,"country":227,"geoPoint":236},"Cliniques Universitaires Saint-Luc","Brussels","Brussels Capital","1200",{"lat":237,"lon":238},50.85045,4.34878,{"facility":240,"status":8,"city":241,"state":242,"zip":243,"country":227,"geoPoint":244},"UZ Gent (University Hospital Ghent)","Ghent","East Flanders","9000",{"lat":245,"lon":246},51.05,3.71667,{"facility":248,"status":8,"city":249,"state":250,"zip":251,"country":227,"geoPoint":252},"UZ Leuven (Universitair Ziekenhuis Leuven)","Leuven","Flemish Brabant","3000",{"lat":253,"lon":254},50.87959,4.70093,{"facility":256,"status":8,"city":257,"state":258,"zip":259,"country":260,"geoPoint":261},"Nucleo de Pesquisa Clinica do Rio Grande do Sul","Porto Alegre","Rio Grande do Sul","90430-001","Brazil",{"lat":262,"lon":263},-30.03283,-51.23019,{"facility":265,"status":8,"city":266,"state":267,"zip":268,"country":260,"geoPoint":269},"Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo","Ribeirão Preto","São Paulo","14051-140",{"lat":270,"lon":271},-21.1775,-47.81028,{"facility":273,"status":8,"city":267,"state":267,"zip":274,"country":260,"geoPoint":275},"Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)","05403-000",{"lat":276,"lon":277},-23.5475,-46.63611,{"facility":279,"status":8,"city":280,"state":281,"zip":282,"country":283,"geoPoint":284},"Instituto de Investigaciones Materno Infantil IDIMI, Facultad de Medicina de la Universidad de Chile","Santiago","Santiago Metropolitan","8360160","Chile",{"lat":285,"lon":286},-33.45694,-70.64827,{"facility":288,"status":8,"city":289,"zip":290,"country":283,"geoPoint":291},"Hospital las Hilgueras de Talcahuano","Talcahuano","4270918",{"lat":292,"lon":293},-36.72494,-73.11684,{"facility":295,"status":8,"city":296,"zip":297,"country":298,"geoPoint":299},"Centre Hospitalier Universitaire (CHU) d'Angers","Angers","49100","France",{"lat":300,"lon":301},47.47156,-0.55202,{"facility":303,"status":8,"city":304,"zip":305,"country":298,"geoPoint":306},"Hopital Kremlin-Bicétre - APHP Paris Saclay","Le Kremlin-Bicêtre","94270",{"lat":307,"lon":308},48.81471,2.36073,{"facility":310,"status":8,"city":311,"zip":312,"country":298,"geoPoint":313},"Hopital Jeanne de Flandre - CHU de Lille","Lille","59037",{"lat":314,"lon":315},50.63391,3.05512,{"facility":317,"status":8,"city":318,"zip":319,"country":298,"geoPoint":320},"APHM -Hopital La Timone Enfants","Marseille","13385 Cedex 5",{"lat":321,"lon":322},43.29695,5.38107,{"facility":324,"status":8,"city":325,"zip":326,"country":298,"geoPoint":327},"Hopital Necker - Enfants Malades","Paris","75015",{"lat":328,"lon":329},48.85341,2.3488,{"facility":331,"status":8,"city":325,"zip":332,"country":298,"geoPoint":333},"Hopital Robert Debre","75019",{"lat":328,"lon":329},{"facility":335,"status":8,"city":336,"state":337,"zip":338,"country":339,"geoPoint":340},"Universitätsklinikum Tübingen Klinik fur Kinder und Jugendmedizin","Tübingen","Baden","72076","Germany",{"lat":341,"lon":342},48.52266,9.05222,{"facility":344,"status":8,"city":345,"state":346,"zip":347,"country":339,"geoPoint":348},"Universitatsklinikum Koln Klinik und Poliklinik fur Kinder- und Jugendmedizin","Cologne","North Rhine-Westphalia","50937",{"lat":349,"lon":350},50.93333,6.95,{"facility":352,"status":8,"city":353,"zip":354,"country":339,"geoPoint":355},"Charité - Universitätsmedizin Berlin Campus Virchow-Klinikum Klinik für pädiatrische Endokrinologie und Diabetologie","Berlin","13353",{"lat":356,"lon":357},52.52437,13.41053,{"facility":359,"status":8,"city":360,"state":361,"zip":362,"country":363,"geoPoint":364},"AOU Federico II","Naples","Campania","80131","Italy",{"lat":365,"lon":366},40.85216,14.26811,{"facility":368,"status":8,"city":369,"state":370,"zip":371,"country":363,"geoPoint":372},"IRCCS Istituto Giannina Gaslini","Genoa","Liguria","16147",{"lat":373,"lon":374},44.40478,8.94439,{"facility":376,"status":8,"city":377,"state":378,"zip":379,"country":363,"geoPoint":380},"IRCCS Ospedale San Raffaele","Milan","Lombardy","20132",{"lat":381,"lon":382},45.46427,9.18951,{"facility":384,"status":8,"city":385,"state":386,"zip":387,"country":363,"geoPoint":388},"Azienda Ospedaliera Universitaria Meyer IRCCS","Florence","Tuscany","50139",{"lat":389,"lon":390},43.77925,11.24626,{"facility":392,"status":8,"city":393,"state":394,"zip":395,"country":396,"geoPoint":397},"Radboudumc Amalia Children's Hospital, Department of Pediatrics","Nijmegen","Gelderland","6525 GA","Netherlands",{"lat":398,"lon":399},51.8425,5.85278,{"facility":401,"status":8,"city":402,"state":403,"zip":404,"country":405,"geoPoint":406},"Uniwersytecki Dziecięcy Szpital Kliniczny im. L. Zamenhofa w Białymstoku, Klinika Pediatrii, Endokrynologii, Diabetologii z Pododdziałem Kardiologii","Bialystok","Podlaskie Voivodeship","15-274","Poland",{"lat":407,"lon":408},53.13333,23.16433,{"facility":410,"status":8,"city":411,"state":405,"zip":412,"country":405,"geoPoint":413},"lnstytut Centrum Zdrowia Matki Polki, Klinika Endokrynologii i Chor6b Metabolicznych","Lodz","93-338",{"lat":414,"lon":415},51.77058,19.47395,{"facility":417,"status":8,"city":418,"state":419,"zip":420,"country":405,"geoPoint":421},"Uniwersytecki Szpital Kliniczny Nr 1 im. Prof. Tadeusza Sokotowskiego PUM w Szczecinie, Centrum Wsparcia Badan Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie","Szczecin","West Pomeranian Voivodeship","71-252",{"lat":422,"lon":423},53.42894,14.55302,{"facility":425,"status":8,"city":426,"zip":427,"country":428,"geoPoint":429},"Alder Hey Children's NHS Foundation Trust, Alder Hey Children's Hospital","Liverpool","L12 2AP","United Kingdom",{"lat":430,"lon":431},53.41058,-2.97794,{"facility":433,"status":8,"city":434,"zip":435,"country":428,"geoPoint":436},"Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street Hospital","London","WC1N 3JH",{"lat":437,"lon":438},51.50853,-0.12574,{"facility":440,"status":8,"city":441,"zip":442,"country":428,"geoPoint":443},"Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital","Manchester","M13 9WL",{"lat":444,"lon":445},53.48095,-2.23743,{"facility":447,"status":8,"city":448,"zip":449,"country":428,"geoPoint":450},"Sheffield Children's Hospital NHS Trust, Sheffield Children's Hospital, Western Bank","Sheffield","S10 2TH",{"lat":451,"lon":452},53.38297,-1.4659,[454],{"name":455,"role":456,"phone":457,"email":458},"Crinetics Clinical Trials","CONTACT","833-827-9741","clinicaltrials@crinetics.com",[],[],[],{"nct_id":4,"conditions":463,"biomarkers":464},[20],[465],"Steroid 21-monooxygenase",{"nct_id":4,"found":15,"summary":467,"prompt_version":477},{"design":468,"status":469,"heading":470,"summary":471,"follow_up":472,"word_count":473,"commitments":474,"compensation":475,"drugs_mentioned":476},"This is a Phase 2\u002F3 study with an open-label extension. It includes a double-blind, randomized, placebo-controlled part, meaning some participants will receive atumelnant and others a placebo, and neither you nor your doctor will know which you are receiving.","completed","A Study of Atumelnant for Children with Congenital Adrenal Hyperplasia","This study is testing a drug called atumelnant in children aged 1 to 17 years old who have classic congenital adrenal hyperplasia (CAH), a condition where the adrenal glands don't make enough of certain hormones. The main goals are to see how safe and effective atumelnant is, and how the body uses and reacts to the drug. Researchers will measure changes in a hormone called androstenedione (A4) and how much steroid medicine (glucocorticoid) participants need. Some participants will receive atumelnant, while others will receive a placebo (an inactive substance that looks like the drug). The study aims to enroll about 153 participants.","Some participants may be followed for up to 260 weeks (about 5 years) to observe changes in serum early morning A4 over time.",102,"Not specified in the trial record.","Not stated in the trial record.",[24,45],"v2"]