[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100606525":3},{"organization":4,"outcomesModule":7,"designInfo":57,"detailedDescription":63,"studyPopulation":22,"armGroups":64,"interventions":71,"overallOfficials":79,"centralContacts":84,"locations":94,"responsibleParty":391,"collaborators":22,"id":393,"slug":394,"hasResults":395,"nctId":396,"briefTitle":397,"officialTitle":398,"acronym":22,"eligibilityCriteria":399,"healthyVolunteers":395,"sex":400,"minAge":401,"maxAge":402,"enrollmentInfo":403,"targetDuration":22,"studyType":406,"phases":407,"briefSummary":409,"conditions":410,"keywords":413,"overallStatus":97,"whyStopped":22,"lastUpdateSubmitDate":417,"lastUpdatePostDateStruct":418,"startDateStruct":421,"completionDateStruct":423,"leadSponsor":425,"locationsCount":426},{"fullName":5,"class":6},"Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":23},[9],{"measure":10,"description":11,"timeFrame":12},"Ovarian function recovery rate of ovarian toxicity (OT) treatment-emergent adverse events (TEAEs)","Ovarian function recovery is defined as achieving the resumption of ≥2 consecutive menstrual periods and an FSH level \\\u003C30 mIU\u002FmL with concomitant estradiol \\\u003C80 pg\u002FmL OR resumption of ≥2 consecutive menstrual periods and AMH level within normal range adjusted for age and pretreatment baseline OR a positive serum β-HCG pregnancy test.","Up to 24 cycles (each cycle is 28 days) of treatment and up to 2 years in the Clinical Follow-up Period",[14,17,20],{"measure":15,"description":16,"timeFrame":12},"Incidence of OT TEAEs","OT is any new onset amenorrhea lasting ≥3 consecutive menstrual periods, FSH level ≥30 mIU\u002FmL and a negative β-HCG pregnancy test.",{"measure":18,"description":19,"timeFrame":12},"Time to ovarian function recovery in participants with a TEAE of OT","Time to ovarian function recovery is evaluated in participants who had a TEAE of OT and is defined as the time it takes to resolve.",{"measure":21,"description":22,"timeFrame":12},"The incidence of adverse events (AEs) according to toxicities graded by National Cancer Institute (NCI) Common Technology Criteria for Adverse Events (CTCAE) Version 5",null,[24,28,32,35,38,41,44,46,49,53],{"measure":25,"description":26,"timeFrame":27},"Objective Response Rate (ORR)","Proportion of participants with Complete Response (CR) and Partial Response (PR) using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria","First day of every 3 cycles (each cycle is 28 days) for up to 24 cycles of treatment and up to 1 year in the Clinical Follow-up Period",{"measure":29,"description":30,"timeFrame":31},"Duration of Response (DoR) for participants whose best response is CR or PR","DoR is defined as the duration from the time of first assessment of CR or PR response (per RECIST v1.1) to the first date of disease progression or death (whichever comes first).","First day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, whichever comes first, up to 24 cycles of treatment and up to 1 year in the Clinical Follow-up Period",{"measure":33,"description":34,"timeFrame":27},"Disease Control Rate (DCR) for participants whose best response is CR, PR or Stable Disease (SD)","DCR is defined as the proportion of participants who have a best response of CR, PR, or SD.",{"measure":36,"description":37,"timeFrame":31},"Duration of Disease Control (DoDC) for participants whose best response is CR, PR or SD","DoDC is defined as the duration from the start of study treatment until first date of disease progression or death (whichever comes first).",{"measure":39,"description":40,"timeFrame":31},"Progression Free Survival (PFS)","PFS is defined at the start of study treatment until the date of assessment of progression or death by any cause. Progression will be determined radiographically using RESIST v1.1 performed by a local radiologist.",{"measure":42,"description":22,"timeFrame":43},"Changes in tumor volume from baseline assessed by MRI volumetric analysis performed locally","First day of every 3 cycles (each cycle is 28 days) up to 24 cycles and up to 1 year in the Clinical Follow-up Period",{"measure":45,"description":22,"timeFrame":43},"Change in T2 hyperintensity baseline assessed by MRI analysis performed locally",{"measure":47,"description":22,"timeFrame":48},"Characterization of serum nirogacestat concentrations assessed in sparse pharmacokinetic (PK) sampling (pre-dose and 1-hour post dose at Cycle 1 Day 1 only) and trough PK sampling (pre-dose at treatment period visits after Cycle 1 Day 1","First day of every 3 cycles (each cycle is 28 days) up to 24 cycles of treatment",{"measure":50,"description":51,"timeFrame":52},"Pain severity using Brief Pain Index - Short Form (BPI-SF), question #3","BPI question #3 will measure worst pain in the last 24 hours using an 11-point numeric scale from 0 (no pain) to 10 (pain as bad as you can imagine)","From 7 days prior to date of first dose of treatment through end of Cycle 2 (each cycle is 28 days), assessed up to 9 weeks",{"measure":54,"description":55,"timeFrame":56},"Patient experience of desmoid tumors and nirogacestat","Two (2) optional 60-minute qualitative interviews using open-ended exploratory questions to elicit spontaneous responses will be transcribed and analyzed in an inductive\u002Fdeductive method to identify themes in the data","One interview will occur following treatment completion (on average approximately 2 years after study entry) and a second interview will occur at study completion (on average approximately 3 years after study entry)",{"allocation":58,"interventionModel":59,"interventionModelDescription":22,"primaryPurpose":60,"observationalModel":22,"timePerspective":22,"maskingInfo":61},"NA","SINGLE_GROUP","TREATMENT",{"masking":62,"maskingDescription":22,"whoMasked":22},"NONE","Desmoid tumors, also referred to as aggressive fibromatosis, are rare, locally invasive, slow growing soft tissue tumors. Although considered benign because of their inability to metastasize, desmoid tumors can cause significant morbidity and occasionally mortality in patients.\n\nNirogacestat is a tumor inhibitor that works by slowing or stopping the growth of tumor cells. Nirogacestat is a tablet taken by mouth and has been approved in the USA for adult patients with progressing desmoid tumors who require systemic treatment.\n\nThis is an open-label study to characterize the incidence and ovarian function recovery rates of ovarian toxicity (OT) events and to evaluate the efficacy, safety, and tolerability of nirogacestat in postpubertal and premenopausal females with desmoid tumors (DT).",[65],{"label":66,"type":67,"description":68,"interventionNames":69},"Nirogacestat","EXPERIMENTAL","Nirogacestat 150 mg by mouth, twice daily",[70],"Drug: Nirogacestat",[72],{"type":73,"name":66,"description":74,"armGroupLabels":75,"otherNames":76},"DRUG","Nirogacestat oral tablet",[66],[77,78],"PF-03084014","Ogsiveo",[80],{"name":81,"affiliation":82,"role":83},"Medical Responsible","SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany","STUDY_DIRECTOR",[85,90],{"name":86,"role":87,"phone":88,"phoneExt":22,"email":89},"US Medical Information","CONTACT","888-275-7376","eMediUSA@emdserono.com",{"name":91,"role":87,"phone":92,"phoneExt":22,"email":93},"Communication Center","+49 6151 72 5200","service@emdgroup.com",[95,112,127,143,157,170,179,192,205,218,233,246,261,271,284,294,304,314,327,340,355,368,378],{"facility":96,"status":97,"city":98,"state":22,"zip":99,"country":100,"countryCode":101,"cosmosGeoPoint":102,"geoPoint":107,"contacts":108},"Cliniques Universitaires Saint-Luc (CUSL)","RECRUITING","Brussels","1200","Belgium","BE",{"type":103,"coordinates":104},"Point",[105,106],4.34878,50.85045,{"lat":106,"lon":105},[109],{"name":110,"role":87,"phone":22,"phoneExt":22,"email":111},"Filomena Mazzeo, MD","Filomena.mazzeo@saintluc.uclouvain.be",{"facility":113,"status":97,"city":114,"state":22,"zip":115,"country":116,"countryCode":117,"cosmosGeoPoint":118,"geoPoint":122,"contacts":123},"Universitaetsmedizin Mannheim","Mannheim","68167","Germany","DE",{"type":103,"coordinates":119},[120,121],8.46694,49.4891,{"lat":121,"lon":120},[124],{"name":125,"role":87,"phone":22,"phoneExt":22,"email":126},"Bernd Kasper, MD","bernd.kasper@umm.de",{"facility":128,"status":97,"city":129,"state":130,"zip":131,"country":132,"countryCode":133,"cosmosGeoPoint":134,"geoPoint":138,"contacts":139},"IRCCS Istituto Romagnolo per lo Studio dei Tumori \"Dino Amadori\" - IRST S.r.l.","Meldola","Forli-Cesena","47014","Italy","IT",{"type":103,"coordinates":135},[136,137],12.0626,44.12775,{"lat":137,"lon":136},[140],{"name":141,"role":87,"phone":22,"phoneExt":22,"email":142},"Lorena Gurrieri, MD","Lorena.gurrieri@irst.emr.it",{"facility":144,"status":97,"city":145,"state":146,"zip":147,"country":132,"countryCode":133,"cosmosGeoPoint":148,"geoPoint":152,"contacts":153},"La Fondazione e l'Istituto di Candiolo","Candiolo","Torino","10060",{"type":103,"coordinates":149},[150,151],7.59812,44.95858,{"lat":151,"lon":150},[154],{"name":155,"role":87,"phone":22,"phoneExt":22,"email":156},"Giovanni Grignani, MD","giovanni.grignani@ircc.it",{"facility":158,"status":97,"city":159,"state":22,"zip":160,"country":132,"countryCode":133,"cosmosGeoPoint":161,"geoPoint":165,"contacts":166},"IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant Orsola","Bologna","40138",{"type":103,"coordinates":162},[163,164],11.33875,44.49381,{"lat":164,"lon":163},[167],{"name":168,"role":87,"phone":22,"phoneExt":22,"email":169},"Maria Abbondanza Pantaleo, MD","maria.pantaleo@unibo.it",{"facility":171,"status":97,"city":159,"state":22,"zip":160,"country":132,"countryCode":133,"cosmosGeoPoint":172,"geoPoint":174,"contacts":175},"IRCCS Istituto Ortopedico Rizzoli di Bologna",{"type":103,"coordinates":173},[163,164],{"lat":164,"lon":163},[176],{"name":177,"role":87,"phone":22,"phoneExt":22,"email":178},"Giorgio Frega, MD","giorgio.frega@ior.it",{"facility":180,"status":97,"city":181,"state":22,"zip":182,"country":132,"countryCode":133,"cosmosGeoPoint":183,"geoPoint":187,"contacts":188},"Fondazione IRCCS Istituto Nazionale dei Tumori Milano","Milan","20133",{"type":103,"coordinates":184},[185,186],12.59836,42.78235,{"lat":186,"lon":185},[189],{"name":190,"role":87,"phone":22,"phoneExt":22,"email":191},"Silvia Stacchiotti","Silvia.Stacchiotti@istitutotumori.mi.it",{"facility":193,"status":97,"city":194,"state":22,"zip":195,"country":132,"countryCode":133,"cosmosGeoPoint":196,"geoPoint":200,"contacts":201},"Istituto Nazionale Tumori I.R.C.C.S- Fondazione G. Pascale","Naples","80131",{"type":103,"coordinates":197},[198,199],14.26811,40.85216,{"lat":199,"lon":198},[202],{"name":203,"role":87,"phone":22,"phoneExt":22,"email":204},"Salvatore Tafuto, MD","s.tafuto@istitutotumori.na.it",{"facility":206,"status":97,"city":207,"state":22,"zip":208,"country":132,"countryCode":133,"cosmosGeoPoint":209,"geoPoint":213,"contacts":214},"Policlinico Universitario Campus Bio-Medico","Roma","00128",{"type":103,"coordinates":210},[211,212],11.10642,44.99364,{"lat":212,"lon":211},[215],{"name":216,"role":87,"phone":22,"phoneExt":22,"email":217},"Bruno Vincenzi, MD","b.vincenzi@policlinicocampus.it",{"facility":219,"status":97,"city":220,"state":22,"zip":221,"country":222,"countryCode":223,"cosmosGeoPoint":224,"geoPoint":228,"contacts":229},"Nederlands Kanker Instituut - Antoni van Leeuwenhoek Ziekenhuis (NKI-AVL)","Amsterdam","1066 CX","Netherlands","NL",{"type":103,"coordinates":225},[226,227],4.88969,52.37403,{"lat":227,"lon":226},[230],{"name":231,"role":87,"phone":22,"phoneExt":22,"email":232},"Winette van der Graaf, MD","w.vd.graaf@nki.nl",{"facility":234,"status":97,"city":235,"state":22,"zip":236,"country":222,"countryCode":223,"cosmosGeoPoint":237,"geoPoint":241,"contacts":242},"Academisch Ziekenhuis Leiden","Leiden","2333 ZA",{"type":103,"coordinates":238},[239,240],4.49306,52.15833,{"lat":240,"lon":239},[243],{"name":244,"role":87,"phone":22,"phoneExt":22,"email":245},"André Johan Gelderblom, MD","A.J.Gelderblom@lumc.nl",{"facility":247,"status":97,"city":248,"state":22,"zip":249,"country":250,"countryCode":251,"cosmosGeoPoint":252,"geoPoint":256,"contacts":257},"Hospital de la Santa Creu i Sant Pau","Barcelona","08041","Spain","ES",{"type":103,"coordinates":253},[254,255],2.15899,41.38879,{"lat":255,"lon":254},[258],{"name":259,"role":87,"phone":22,"phoneExt":22,"email":260},"Ana Sebio Garcia, MD","asebio@santpau.cat",{"facility":262,"status":97,"city":248,"state":22,"zip":263,"country":250,"countryCode":251,"cosmosGeoPoint":264,"geoPoint":266,"contacts":267},"Institut Catala d'Oncologia","08907",{"type":103,"coordinates":265},[254,255],{"lat":255,"lon":254},[268],{"name":269,"role":87,"phone":22,"phoneExt":22,"email":270},"Laura Jimenez Colomo","ljcolomo@iconcologia.net",{"facility":272,"status":97,"city":273,"state":22,"zip":274,"country":250,"countryCode":251,"cosmosGeoPoint":275,"geoPoint":279,"contacts":280},"Hospital General Universitario Gregorio Maranon","Madrid","28007",{"type":103,"coordinates":276},[277,278],-3.70256,40.4165,{"lat":278,"lon":277},[281],{"name":282,"role":87,"phone":22,"phoneExt":22,"email":283},"Rosa Alvarez, MD","rosa.alvarez.al@gmail.com",{"facility":285,"status":97,"city":273,"state":22,"zip":286,"country":250,"countryCode":251,"cosmosGeoPoint":287,"geoPoint":289,"contacts":290},"Hospital Fundacion Jimenez Diaz","28040",{"type":103,"coordinates":288},[277,278],{"lat":278,"lon":277},[291],{"name":292,"role":87,"phone":22,"phoneExt":22,"email":293},"Nadia Hindi Muniz, MD","nhindi@atbsarc.org",{"facility":295,"status":97,"city":273,"state":22,"zip":296,"country":250,"countryCode":251,"cosmosGeoPoint":297,"geoPoint":299,"contacts":300},"Hospital Universitario 12 de Octubre","28041",{"type":103,"coordinates":298},[277,278],{"lat":278,"lon":277},[301],{"name":302,"role":87,"phone":22,"phoneExt":22,"email":303},"Aranzazu Manzano Fernández, MD","aranzazu.manzano@salud.madrid.org",{"facility":305,"status":97,"city":273,"state":22,"zip":306,"country":250,"countryCode":251,"cosmosGeoPoint":307,"geoPoint":309,"contacts":310},"Hospital Universitario La Paz","28046",{"type":103,"coordinates":308},[277,278],{"lat":278,"lon":277},[311],{"name":312,"role":87,"phone":22,"phoneExt":22,"email":313},"Andres Redondo Sanchez, MD","aredondo12@gmail.com",{"facility":315,"status":97,"city":316,"state":22,"zip":317,"country":250,"countryCode":251,"cosmosGeoPoint":318,"geoPoint":322,"contacts":323},"Hospital Universitari i Politecnic La Fe de Valencia","Valencia","46026",{"type":103,"coordinates":319},[320,321],-0.37966,39.47391,{"lat":321,"lon":320},[324],{"name":325,"role":87,"phone":22,"phoneExt":22,"email":326},"Roberto Diaz, MD","diaz_rob@gva.es",{"facility":328,"status":97,"city":329,"state":22,"zip":330,"country":250,"countryCode":251,"cosmosGeoPoint":331,"geoPoint":335,"contacts":336},"Hospital Universitario Miguel Servet","Zaragoza","50009",{"type":103,"coordinates":332},[333,334],-0.87734,41.65606,{"lat":334,"lon":333},[337],{"name":338,"role":87,"phone":22,"phoneExt":22,"email":339},"Javier Martinez Trufero, MD","jmtrufero@seom.org",{"facility":341,"status":97,"city":342,"state":22,"zip":343,"country":344,"countryCode":345,"cosmosGeoPoint":346,"geoPoint":350,"contacts":351},"Cambridge University - Addenbrooke's Hospital","Cambridge","CB2 0QQ","United Kingdom","UK",{"type":103,"coordinates":347},[348,349],0.11667,52.2,{"lat":349,"lon":348},[352],{"name":353,"role":87,"phone":22,"phoneExt":22,"email":354},"Han Hsi Wong, MD","han.wong@nhs.net",{"facility":356,"status":97,"city":357,"state":22,"zip":358,"country":344,"countryCode":345,"cosmosGeoPoint":359,"geoPoint":363,"contacts":364},"University College London Hospital","London","NW1 2PG",{"type":103,"coordinates":360},[361,362],-0.12574,51.50853,{"lat":362,"lon":361},[365],{"name":366,"role":87,"phone":22,"phoneExt":22,"email":367},"Vasilios Karavasilis, MD","vasilios.karavasilis@nhs.net",{"facility":369,"status":97,"city":357,"state":22,"zip":370,"country":344,"countryCode":345,"cosmosGeoPoint":371,"geoPoint":373,"contacts":374},"The Royal Marsden NHS Foundation Trust","SW3 6JJ",{"type":103,"coordinates":372},[361,362],{"lat":362,"lon":361},[375],{"name":376,"role":87,"phone":22,"phoneExt":22,"email":377},"Charlotte Benson, MD","charlotte.benson@rmh.nhs.uk",{"facility":379,"status":97,"city":380,"state":22,"zip":381,"country":344,"countryCode":345,"cosmosGeoPoint":382,"geoPoint":386,"contacts":387},"The Christie NHS Foundation Trust","Manchester","M20 4BX",{"type":103,"coordinates":383},[384,385],-2.23743,53.48095,{"lat":385,"lon":384},[388],{"name":389,"role":87,"phone":22,"phoneExt":22,"email":390},"Alexander Lee, MD","alexander.lee1@nhs.net",{"type":392,"investigatorFullName":22,"investigatorTitle":22,"investigatorAffiliation":22,"oldNameTitle":22,"oldOrganization":22},"SPONSOR","100606525","phase-4-nirogacestat-in-premenopausal-females-with-desmoid-tumoraggressive-fibromatosis-dtaf-100606525",false,"NCT07176689","Nirogacestat in Premenopausal Females With Desmoid Tumor\u002FAggressive Fibromatosis (DT\u002FAF)","A Single-Arm, Open-Label Phase 4 Study of Nirogacestat in Adult Premenopausal Females With Desmoid Tumors\u002FAggressive Fibromatosis (DT\u002FAF)","Inclusion Criteria:\n\n* Participant is female, postpubertal aged ≥18 and ≤40 years of age at the time of signing the informed consent and premenopausal at baseline. Premenopausal is defined as meeting all of the following: Estradiol \\>30 pg\u002FmL. Follicle-stimulating hormone (FSH) \\\u003C40 IU\u002FL. Regular menses (e.g., menstrual cycle length of 21 to 35 days) for at least 3 menstrual cycles prior to signing informed consent\n* Participant uses 1 highly effective non-hormonal contraceptive method, has a negative pregnancy test prior to first dose of study treatment), is not breastfeeding, agrees to not harvest or donate eggs for at least 90 days prior to and during the study\n* Participant has histologically confirmed DT\u002FAF with symptomatic or progressive disease requiring systemic treatment\n* Participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤2 at screening\n* Participant has adequate organ and bone marrow function.\n\nExclusion Criteria:\n\n* Participant has known malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of nirogacestat\n* Participant has experienced any of the following within 6 months of signing informed consent: clinically significant cardiac disease (New York Heart Association Class III or IV), myocardial infarction, severe\u002Funstable angina, coronary\u002Fperipheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident, transient ischemic attack, or symptomatic pulmonary embolism.\n* Participant has had lymphoma, leukemia, or any malignancy within the past 5 years at the time of informed consent, except for any locally recurring cancer that has been treated curatively (e.g., resected basal or squamous cell skin cancer, superficial bladder cancer, carcinoma in situ of the cervix or breast), with no evidence of metastatic disease for 3 years at the time of informed consent.\n* Participant has known hepatic impairment\n* Participant previously received or is currently receiving gamma secretase inhibitors or anti-Notch antibody therapy\n* Participant is currently using any treatment for DT\u002FAF including tyrosine kinase inhibitors (TKIs) or any investigational treatment 28 days (or 5 half-lives, whichever is longer) prior to the first dose of study treatment\n* Participant is currently using or anticipates using food or drugs that are known strong\u002Fmoderate cytochrome P450 (CYP) 3A4 inhibitors, or strong CYP3A inducers within 14 days prior to the first dose of study treatment.\n* Participant has a history of polycystic ovary syndrome, hypothalamic amenorrhea, severe endometriosis involving ovaries, family history of primary ovarian insufficiency, any chromosomal abnormality, mutation, gene variant or medical condition associated with early\u002Fpremature menopause, including a history of OT while on a TKI\n* Participant is currently using or has used hormonal contraception or ovarian suppression within 90 days prior to first dose of study treatment\n* Participant has a history of heavy tobacco smoking (≥20 pack years) or is a current smoker (\\>1 pack per day)\n* Participant has experienced other severe acute or chronic medical or psychiatric conditions within 1 year of signing informed consent.\n* Participant is unable to comply with study related procedures (including, but not limited to, the completion of a menstrual diary and electronic patient-reported outcomes and ability to return to clinic for hormone level blood draws timed to the menstrual cycle (days 1-5)","FEMALE","18 Years","40 Years",{"count":404,"type":405},50,"ESTIMATED","INTERVENTIONAL",[408],"PHASE4","This study is being conducted to study how nirogacestat may affect the ovarian function of adult premenopausal women with progressing desmoid tumors\u002Faggressive fibromatosis.",[411,412],"Desmoid Tumor","Aggressive Fibromatosis",[66,77,414,415,416,78],"GSI","gamma secretase inhibitor","notch pathway","2026-10-12",{"date":419,"type":420},"2026-10-13","ACTUAL",{"date":422,"type":420},"2025-09-17",{"date":424,"type":405},"2031-02-26",{"name":82,"class":6},23]