[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100635963":3},{"organization":4,"outcomesModule":7,"designInfo":64,"detailedDescription":11,"studyPopulation":11,"armGroups":70,"interventions":82,"overallOfficials":90,"centralContacts":94,"locations":11,"responsibleParty":100,"collaborators":11,"id":102,"slug":11,"hasResults":103,"nctId":104,"briefTitle":105,"officialTitle":106,"acronym":107,"eligibilityCriteria":108,"healthyVolunteers":103,"sex":109,"minAge":110,"maxAge":111,"enrollmentInfo":112,"targetDuration":11,"studyType":115,"phases":116,"briefSummary":118,"conditions":119,"keywords":121,"overallStatus":123,"whyStopped":11,"lastUpdateSubmitDate":124,"lastUpdatePostDateStruct":125,"startDateStruct":128,"completionDateStruct":130,"leadSponsor":132,"locationsCount":11},{"fullName":5,"class":6},"Regeneron Pharmaceuticals","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":22,"otherOutcomes":11},[9,13,16,18,19],{"measure":10,"description":11,"timeFrame":12},"Occurrence of Treatment-Emergent Adverse Event (TEAEs)",null,"Baseline to week 28",{"measure":14,"description":11,"timeFrame":15},"Occurrence of TEAEs","Baseline to week 56",{"measure":17,"description":11,"timeFrame":12},"Severity of TEAEs",{"measure":17,"description":11,"timeFrame":15},{"measure":20,"description":11,"timeFrame":21},"Concentrations of functional garetosmab in serum","Through week 56",[23,26,28,30,33,35,38,40,43,46,49,51,53,55,57,60],{"measure":24,"description":11,"timeFrame":25},"Total volume of new Heterotopic Ossification (HO) lesion","At week 28 and week 56",{"measure":27,"description":11,"timeFrame":25},"Number of new HO lesions",{"measure":29,"description":11,"timeFrame":25},"Occurrence of new HO lesions",{"measure":31,"description":11,"timeFrame":32},"Number of clinician-assessed flare-ups","Through week 28 and week 56",{"measure":34,"description":11,"timeFrame":32},"Occurrence of clinician-assessed flare-ups",{"measure":36,"description":11,"timeFrame":37},"Number of patient\u002Fcaregiver-reported flare-ups","Through Week 28 and week 56",{"measure":39,"description":11,"timeFrame":32},"Occurrence of patient\u002Fcaregiver-reported flare-ups",{"measure":41,"description":42,"timeFrame":25},"Change from baseline in Tanner puberty scale","Tanner puberty scale or stages: Staging of sexual development is graded on a 5-point ordinal scale ranging from 1 (prepubertal) to 5 (adultlike) for female breast development, male external genitals, and pubic hair",{"measure":44,"description":11,"timeFrame":45},"Characteristics of menstrual cycles for female participants who reached menarche","Over 28 weeks and 56 weeks",{"measure":47,"description":48,"timeFrame":21},"Height-for-age Z-Scores according to the World Health Organization (WHO) Growth Reference Data for Children","Participants 5-19 years of age Z-score represents standardized measure of how far an individual deviated from study cohort average at baseline. A higher Z-score reflects better performance.",{"measure":50,"description":11,"timeFrame":21},"Concentrations of total activin A in serum",{"measure":52,"description":11,"timeFrame":21},"Occurence of Anti-Drug Antibody (ADA) to garetosmab",{"measure":54,"description":11,"timeFrame":21},"Magnitude of ADA to garetosmab",{"measure":56,"description":11,"timeFrame":25},"Change from baseline in hearing function as assessed by audiometry",{"measure":58,"description":59,"timeFrame":25},"Change from baseline in Pediatric Quality of Life inventory (PedsQL) scores","Age-appropriate PedsQL Generic Core Scales will be used to measure HRQoL in children and adolescents. Response options include 5-point Likert scale (or 3-point Likert scale for the young children self-report) for each item asking about experience within the past week. Global scores are transformed to a 0 to 100 scale with higher scores indicating better quality of life.",{"measure":61,"description":62,"timeFrame":63},"Acceptability and tolerability assessment via exit interview","Each interview will be conducted by trained external interviewers following a semi-structured interview guide of questions for the participants about their overall experience in the trial.","Up to week 30",{"allocation":65,"interventionModel":66,"interventionModelDescription":11,"primaryPurpose":67,"observationalModel":11,"timePerspective":11,"maskingInfo":68},"NON_RANDOMIZED","SINGLE_GROUP","TREATMENT",{"masking":69,"maskingDescription":11,"whoMasked":11},"NONE",[71,76,79],{"label":72,"type":73,"description":11,"interventionNames":74},"Cohort 1: Adolescents","EXPERIMENTAL",[75],"Drug: garetosmab",{"label":77,"type":73,"description":11,"interventionNames":78},"Cohort 2: Children",[75],{"label":80,"type":73,"description":11,"interventionNames":81},"Cohort 3: Children and Adolescents",[75],[83],{"type":84,"name":85,"description":86,"armGroupLabels":87,"otherNames":88},"DRUG","garetosmab","Administered per the protocol",[72,77,80],[89],"REGN2477",[91],{"name":92,"affiliation":5,"role":93},"Clinical Trial Management","STUDY_DIRECTOR",[95],{"name":96,"role":97,"phone":98,"phoneExt":11,"email":99},"Clinical Trials Administrator","CONTACT","844-734-6643","clinicaltrials@regeneron.com",{"type":101,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100635963",false,"NCT07559513","A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)","Phase 3 Evaluation of the Safety, Pharmacokinetics, and Efficacy of Garetosmab (Anti-Activin A Monoclonal Antibody) in Children and Adolescents With Fibrodysplasia Ossificans Progressiva","OPTIMA-2","Key Inclusion Criteria:\n\n1. For USA participants, age criteria are 4 to \\\u003C 18 years old, at the time of the administration of the first dose of study intervention. Non-USA participants age criteria are 2 to \\\u003C 18 years old\n2. Must have a confirmation of FOP diagnosis, as described in the protocol\n3. At the time of enrollment, participants must weight:\n\n   1. Cohort 1 \\> 30 kg\n   2. Cohort 2 \\> 30 kg\n   3. Cohort 3 ≤ 30 kg\n\nKey Exclusion Criteria:\n\n1. Cumulative Analog Joint Involvement Scale (CAJIS) score \\> 19 at the time of screening\n2. Participant has significant concomitant illness or history of significant illness, as described in the protocol\n3. Previous history or diagnosis of cancer\n4. Ongoing significant viral or bacterial illness, within 2 weeks of the first study drug administration\n5. History of severe respiratory compromise requiring oxygen, respiratory support\n6. Known history of cerebral vascular malformation\n7. Participants with a history of severe, non-traumatic bleeding requiring transfusion or hospitalization for hemodynamic compromise\n8. Participants with a known pre-existing medical history of a bleeding diathesis, as described in the protocol\n\nNOTE: Other Protocol-defined Inclusion\u002FExclusion Criteria Apply","ALL","2 Years","18 Years",{"count":113,"type":114},18,"ESTIMATED","INTERVENTIONAL",[117],"PHASE3","This study is researching an experimental drug called garetosmab, referred to as \"study drug\". The study is focused on children and adolescent participants with FOP.\n\nThe aim of the study is to see how safe, tolerable, and effective the study drug is.\n\nThe study is looking at several other research questions, including:\n\n* What side effects may happen from taking the study drug\n* How much study drug is in the blood at different times\n* Whether the body makes antibodies against the study drug (which could make the study drug less effective or could lead to side effects)",[120],"Fibrodysplasia Ossificans Progressiva (FOP)",[122],"Type I Activin A Receptor (ACVR1) FOP-causing mutation","NOT_YET_RECRUITING","2026-04-22",{"date":126,"type":127},"2026-04-30","ACTUAL",{"date":129,"type":114},"2026-07-30",{"date":131,"type":114},"2029-12-21",{"name":5,"class":6}]