[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100654346":3},{"organization":4,"outcomesModule":7,"designInfo":40,"detailedDescription":11,"studyPopulation":11,"armGroups":51,"interventions":64,"overallOfficials":11,"centralContacts":73,"locations":78,"responsibleParty":167,"collaborators":11,"id":169,"slug":170,"hasResults":171,"nctId":172,"briefTitle":173,"officialTitle":174,"acronym":11,"eligibilityCriteria":175,"healthyVolunteers":171,"sex":176,"minAge":177,"maxAge":178,"enrollmentInfo":179,"targetDuration":11,"studyType":182,"phases":183,"briefSummary":186,"conditions":187,"keywords":11,"overallStatus":126,"whyStopped":11,"lastUpdateSubmitDate":189,"lastUpdatePostDateStruct":190,"startDateStruct":193,"completionDateStruct":195,"leadSponsor":197,"locationsCount":198},{"fullName":5,"class":6},"Soufflé Therapeutics, Inc.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":11},[9],{"measure":10,"description":11,"timeFrame":12},"Number of participants with treatment-emergent adverse events, serious adverse events, and any other dose-limiting toxicities as assessed by CTCAE v6.0.",null,"Part A: Up to Week 24; Part B: Up to Week 48",[14,17,19,21,23,25,28,31,34,37],{"measure":15,"description":11,"timeFrame":16},"Pharmacokinetics of SFL-0821: Maximum Observed Plasma Concentration (Cmax)","Part A and B: through 24 hours post dose (all cohorts)",{"measure":18,"description":11,"timeFrame":16},"Pharmacokinetics of SFL-0821: Elimination half-life (T1\u002F2 [h])",{"measure":20,"description":11,"timeFrame":16},"Pharmacokinetics of SFL-0821: Area under the concentration-time curve (AUC)",{"measure":22,"description":11,"timeFrame":12},"DUX4-regulated gene expression in skeletal muscle",{"measure":24,"description":11,"timeFrame":12},"Incidence of anti-drug antibodies (ADAs), and if positive, of neutralizing ADAs",{"measure":26,"description":11,"timeFrame":27},"Change from Baseline in quantitative skeletal muscle magnetic resonance imaging (MRI) at the cohort specified timepoints after dosing","Part B: Up to Week 48",{"measure":29,"description":30,"timeFrame":27},"Change from Baseline to Week 48 in 10-Meter Walk-Run-Test (10MWRT)","Assessment used to measure gait speed and functional mobility over a short distance (10 meters).",{"measure":32,"description":33,"timeFrame":27},"Change from Baseline to Week 48 in Timed Up-and-Go (TUG) Test","Assessment of functional mobility by measuring the time required to stand up from a chair, walk 3 meters, turn, return, and sit down.",{"measure":35,"description":36,"timeFrame":27},"Change from Baseline to Week 48 in 4-Step Stair Climb Test","Assessment of the time to ascend and descend 4 stair steps.",{"measure":38,"description":39,"timeFrame":27},"Change from Baseline to Week 48 in Sit-to-Stand Test","Assessment of the number of full stands completed in the allotted time.",{"allocation":41,"interventionModel":42,"interventionModelDescription":11,"primaryPurpose":43,"observationalModel":11,"timePerspective":11,"maskingInfo":44},"RANDOMIZED","SEQUENTIAL","TREATMENT",{"masking":45,"maskingDescription":11,"whoMasked":46},"QUADRUPLE",[47,48,49,50],"PARTICIPANT","CARE_PROVIDER","INVESTIGATOR","OUTCOMES_ASSESSOR",[52,58],{"label":53,"type":54,"description":55,"interventionNames":56},"SFL-0821","EXPERIMENTAL","SFL-0821 for Injection",[57],"Drug: SFL-0821 for injection",{"label":59,"type":60,"description":61,"interventionNames":62},"Placebo","PLACEBO_COMPARATOR","Placebo for Injection",[63],"Drug: Placebo",[65,70],{"type":66,"name":67,"description":68,"armGroupLabels":69,"otherNames":11},"DRUG","SFL-0821 for injection","single or multiple doses of SFL-0821 by intravenous (IV) infusion",[53],{"type":66,"name":59,"description":71,"armGroupLabels":72,"otherNames":11},"calculated volume to match active treatment by IV infusion",[59],[74],{"name":5,"role":75,"phone":76,"phoneExt":11,"email":77},"CONTACT","1 617-458-7581","SFL0821clinicaltrials@souffletx.com",[79,97,110,124,138,152],{"facility":80,"status":81,"city":82,"state":83,"zip":84,"country":85,"countryCode":86,"cosmosGeoPoint":87,"geoPoint":92,"contacts":93},"Concord Repatriation General Hospital","NOT_YET_RECRUITING","Concord","New South Wales","2139","Australia","AU",{"type":88,"coordinates":89},"Point",[90,91],151.10381,-33.84722,{"lat":91,"lon":90},[94],{"name":95,"role":75,"phone":11,"phoneExt":11,"email":96},"Katrina Morris, MBBS (Hons), PhD, FRACP","katrina.morris1@health.nsw.gov.au",{"facility":98,"status":81,"city":99,"state":83,"zip":100,"country":85,"countryCode":86,"cosmosGeoPoint":101,"geoPoint":105,"contacts":106},"Liverpool Hospital","Liverpool","2170",{"type":88,"coordinates":102},[103,104],150.92588,-33.91938,{"lat":104,"lon":103},[107],{"name":108,"role":75,"phone":11,"phoneExt":11,"email":109},"Suzanne Hodgkinson, MBBS, PhD, FRACP","s.hodgkinson@unsw.edu.au",{"facility":111,"status":81,"city":112,"state":113,"zip":114,"country":85,"countryCode":86,"cosmosGeoPoint":115,"geoPoint":119,"contacts":120},"Wesley Research Institute","Brisbane","Queensland","4066",{"type":88,"coordinates":116},[117,118],153.02809,-27.46794,{"lat":118,"lon":117},[121],{"name":122,"role":75,"phone":11,"phoneExt":11,"email":123},"Robert Henderson, MBBS (Hons), PhD, FRACP","robert.henderson@health.qld.gov.au",{"facility":125,"status":126,"city":127,"state":113,"zip":128,"country":85,"countryCode":86,"cosmosGeoPoint":129,"geoPoint":133,"contacts":134},"Mater Hospital Brisbane","RECRUITING","South Brisbane","4101",{"type":88,"coordinates":130},[131,132],153.02049,-27.48034,{"lat":132,"lon":131},[135],{"name":136,"role":75,"phone":11,"phoneExt":11,"email":137},"Cullen O'Gorman, M.B.B.S., Ph.D.","Cullen.OGorman@health.qld.gov.au",{"facility":139,"status":126,"city":140,"state":141,"zip":142,"country":85,"countryCode":86,"cosmosGeoPoint":143,"geoPoint":147,"contacts":148},"The Alfred Hospital","Melbourne","Victoria","3004",{"type":88,"coordinates":144},[145,146],144.96332,-37.814,{"lat":146,"lon":145},[149],{"name":150,"role":75,"phone":11,"phoneExt":11,"email":151},"Edrich Rodrigues, MBBS, PhD","E.Rodrigues@alfred.org.au",{"facility":153,"status":81,"city":154,"state":154,"zip":155,"country":156,"countryCode":157,"cosmosGeoPoint":158,"geoPoint":162,"contacts":163},"Pacific Clinic Research Network","Auckland","0622","New Zealand","NZ",{"type":88,"coordinates":159},[160,161],174.76349,-36.84853,{"lat":161,"lon":160},[164],{"name":165,"role":75,"phone":11,"phoneExt":11,"email":166},"Miriam Rodrigues","miriam.rodrigues@tewhatuora.govt.nz",{"type":168,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100654346","phase-1-a-trial-to-assess-safety-tolerability-pharmacokinetics-pharmacodynamics-and-preliminary-efficacy-of-sfl-0821-in-adults-with-fshd-100654346",false,"NCT07798609","A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD","A Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Trial to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)","Inclusion Criteria:\n\n* Genetically confirmed diagnosis of FSHD 1 or 2\n* FSHD Clinical Severity Score ≥1.5 to ≤4 at screening (5-point Ricci scale)\n* Ambulatory and able to walk 10 meters between ≥5 and ≤14 seconds (only assistive devices allowed are single cane or splints for ankle-foot orthoses)\n* Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader\n\nExclusion Criteria:\n\n* History of any illness or any clinical condition that, in the opinion of the Investigator, might confound the results of the study or pose an additional risk in administering investigational product to the participant.\n* History of biopsy of the same muscle within 30 days of the Screening biopsy or planning to undergo any non-study muscle biopsies over the duration of the study\n* Treatment with an oligonucleotide, gene therapy, or other experimental therapies for FSHD.","ALL","18 Years","65 Years",{"count":180,"type":181},90,"ESTIMATED","INTERVENTIONAL",[184,185],"PHASE1","PHASE2","The purpose of this study is to evaluate the safety, tolerability, and preliminary efficacy of SFL-0821 in adult patients with Facioscapulohumeral Muscular Dystrophy (FSHD)",[188],"Facioscapulohumeral Muscular 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