[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100658326":3},{"organization":4,"outcomesModule":7,"designInfo":38,"detailedDescription":11,"studyPopulation":11,"armGroups":44,"interventions":55,"overallOfficials":61,"centralContacts":66,"locations":72,"responsibleParty":84,"collaborators":11,"id":86,"slug":87,"hasResults":88,"nctId":89,"briefTitle":90,"officialTitle":91,"acronym":11,"eligibilityCriteria":92,"healthyVolunteers":88,"sex":93,"minAge":94,"maxAge":95,"enrollmentInfo":96,"targetDuration":11,"studyType":99,"phases":100,"briefSummary":102,"conditions":103,"keywords":105,"overallStatus":107,"whyStopped":11,"lastUpdateSubmitDate":108,"lastUpdatePostDateStruct":109,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":117},{"fullName":5,"class":6},"Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":24,"otherOutcomes":11},[9,13,15,17,20,22],{"measure":10,"description":11,"timeFrame":12},"Number of Participants With Treatment-emergent Adverse Events (TEAEs)",null,"Up to approximately 6 years",{"measure":14,"description":11,"timeFrame":12},"Number of Participants With Clinically Significant Changes in Laboratory Parameters, Vital Signs, Physical Examination Findings and Electrocardiograms (ECGs), Echocardiogram (ECHO), and Ophthalmic Exam Findings",{"measure":16,"description":11,"timeFrame":12},"Number of Participants With Severity of Adverse Events (AEs) According to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0",{"measure":18,"description":11,"timeFrame":19},"Minimum Observed Concentration (Cmin) of Mirdametinib","Cycle 1 Days 1 and 15: pre-dose and post-dose at 1 and 4 hours; Cycles 2, 4 and 8 Day 15: Pre-dose and post-dose at 1 hour (each cycle = 28 days)",{"measure":21,"description":11,"timeFrame":19},"Maximum Observed Concentration (Cmax) of Mirdametinib",{"measure":23,"description":11,"timeFrame":19},"Area Under the Concentration-time Curve (AUC) at Steady State for Mirdametinib",[25,28,31,35],{"measure":26,"description":27,"timeFrame":12},"Confirmed Objective Response Rate (ORR)","Confirmed ORR is defined as plexiform neurofibroma (PN) decrease greater than or equal to (\\>=) 20 percent (%) compared to baseline in consecutive scans within 2 to 6 months) using centrally read magnetic resonance imaging (MRI) volumetric analyses by a Blinded Independent Central Review (BICR).",{"measure":29,"description":30,"timeFrame":12},"Duration of Response (DoR)","DoR is defined as the time from onset of confirmed complete response (CR) + partial response (PR) using centrally read MRI volumetric analysis by a BICR to the earliest event of progressive disease (PD) or death.",{"measure":32,"description":33,"timeFrame":34},"Change From Baseline in Quality of Life (QoL) Assessed by the age-specific Pediatric Quality of Life Inventory (PedsQL) Infant Scales","The PedsQL Infant Scales are multidimensional parent-proxy-report scales designed to assess health-related quality of life in infants from 1 to 24 months of age over the past 7 days. The Infant Scales consists of 36 items in the 1- to 12-month form and 45 items in the 13- to 24-month form, with 5 dimensions: physical functioning, physical symptoms, emotional functioning, social functioning, and cognitive functioning.","Baseline up to approximately 6 years",{"measure":36,"description":37,"timeFrame":34},"Change From Baseline Pain Assessment by Age-specific Pain Scale: Faces, Legs, Activity, Cry, Consolability (FLACC)","FLACC is a behavioural pain assessment scale used by the investigator for nonverbal or preverbal patients who are unable to self-report their level of pain. Pain is assessed in infants and children from 2 months to 7 years of age through observation of 5 categories, including face, legs, activity, cry, and consolability. The total score for FLACC scale ranges from 0 to 10, with each category scored on a scale of 0 to 2.",{"allocation":39,"interventionModel":40,"interventionModelDescription":11,"primaryPurpose":41,"observationalModel":11,"timePerspective":11,"maskingInfo":42},"NA","SINGLE_GROUP","TREATMENT",{"masking":43,"maskingDescription":11,"whoMasked":11},"NONE",[45,51],{"label":46,"type":47,"description":48,"interventionNames":49},"Cohort 1: 12 to 21 months of age","EXPERIMENTAL","Participant receives 0.5 milligram (mg) twice daily.",[50],"Drug: Mirdametinib",{"label":52,"type":47,"description":53,"interventionNames":54},"Cohort 2: 0 to 9 months of age","Participants receives 0.25 mg twice daily.",[50],[56],{"type":57,"name":58,"description":59,"armGroupLabels":60,"otherNames":11},"DRUG","Mirdametinib","Mirdametinib dispersible tablet administered orally.",[46,52],[62],{"name":63,"affiliation":64,"role":65},"Medical Responsible","SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany","STUDY_DIRECTOR",[67],{"name":68,"role":69,"phone":70,"phoneExt":11,"email":71},"Communication Center","CONTACT","+49 6151 72 5200","service@emdgroup.com",[73],{"facility":74,"status":11,"city":75,"state":11,"zip":11,"country":76,"countryCode":77,"cosmosGeoPoint":78,"geoPoint":83,"contacts":11},"Research Site","Darmstadt","Germany","DE",{"type":79,"coordinates":80},"Point",[81,82],8.65027,49.87167,{"lat":82,"lon":81},{"type":85,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100658326","phase-1-a-study-of-mirdametinib-in-infants-and-toddlers-with-neurofibromatosis-type-1-associated-plexiform-neurofibromas-100658326",false,"NCT07850635","A Study of Mirdametinib in Infants and Toddlers With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas","A Phase 1b, Open-Label Study of Mirdametinib in Infants and Toddlers With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas","Inclusion Criteria:\n\n1. Age 1.1 Cohort 1: greater than (\\>) 12 to less than or equal to (\\\u003C=) 21 months of age at first dose 1.2 Cohort 2: 0 to \\\u003C=9 months of age at first dose For both cohorts, corrected age must be used to determine eligibility for age at first dose. Note: Corrected age is the age of the infant from the expected date of delivery (40 weeks gestation), rather than from the actual date of birth. For example, a 3-month-old infant who was born 2 months early would have a corrected age of 1 month.\n2. Participants must have had the clinical diagnosis of NF1 according to the 2021 International Consensus Guidelines with presence of a PN and at least 1 additional diagnostic criterion for NF1.\n3. Participants must have a target PN, defined as the clinically most relevant PN amenable to volumetric MRI analysis. For the purpose of this study, the target PN must be seen on at least 3 consecutive MRI slices and the field of view must contain the entire tumour of interest. As determined by central radiologic review, a target PN must be analysable by volumetrics using Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS), at least 3 milliliter (mL) in volume.\n4. Participants must have 1 of the following presentations of PN:\n\n4.1. Asymptomatic: Participant has a measurable NF1 PN in a high risk location with a high risk of morbidity.\n\nA high-risk location with a high risk of morbidity is defined as:\n\n* In the head or neck (except for isolated scalp lesions) OR\n* Within the brachial or lumbosacral plexus OR\n* Adjacent to high-risk structure(s), defined as:\n\n  * Major (\"named\") blood vessel OR\n  * Major (\"named\") airway OR\n  * Hollow viscus OR\n  * Spinal cord and foramina OR\n  * Vital organs (including heart, lungs, liver, spleen, etc) OR 4.2. Symptomatic: Participant has a PN that is causing clinical symptoms at the discretion of the investigator (e.g., head and neck lesions compromising the airway or great vessels, brachial or lumbar plexus lesions causing nerve compression and loss of function, lesions causing major deformity or significant disfigurement, lesions of the extremity causing limb hypertrophy or loss of function, and painful lesions).\n\nExclusion Criteria:\n\n1. Participant has family history of sudden cardiac death.\n2. Participant has a history of, or evidence of, retinal pathology on ophthalmologic examination that is considered a risk factor for central serous retinopathy, retinal vein occlusion (RVO), retinal pigment epithelial detachment, or neovascular macular degeneration.\n3. Participant has a history of congenital glaucoma.\n4. Participant has received NF1 PN-targeted therapy (e.g., farnesyltransferase inhibitors, kinase inhibitors, etc) within 28 days of first dose of study treatment (or 5 half-lives, whichever is longer). All toxicities from prior therapy must resolve to Grade \\\u003C=1 or baseline.\n5. Participant is receiving inhibitors or inducers of P-glycoprotein or breast cancer resistance protein.\n6. Participant is currently enrolled or has past participation in any other clinical study (excluding observational studies) within 28 days of signing of informed consent.","ALL","0 Months","21 Months",{"count":97,"type":98},6,"ESTIMATED","INTERVENTIONAL",[101],"PHASE1","The purpose of this study is to determine the safety, pharmacokinetic (PK), and tolerability of multiple treatment cycles of oral mirdametinib in infants and toddlers (0 to less than \\[\\\u003C\\] 24 months of age) with measurable neurofibromatosis type 1 (NF1) associated plexiform neurofibromas (PNs) that is symptomatic or asymptomatic but in a high-risk location with a high risk of morbidity.",[104],"Neurofibromatosis Type 1-Associated Plexiform Neurofibromas",[58,106],"Single arm study","NOT_YET_RECRUITING","2026-09-24",{"date":110,"type":111},"2026-09-30","ACTUAL",{"date":113,"type":98},"2026-12-10",{"date":115,"type":98},"2032-10-21",{"name":64,"class":6},1]