[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100596567":3},{"organization":4,"armGroups":7,"interventions":19,"overallOfficials":39,"centralContacts":40,"locations":46,"responsibleParty":383,"collaborators":39,"id":385,"slug":386,"hasResults":387,"nctId":388,"briefTitle":389,"officialTitle":390,"acronym":391,"eligibilityCriteria":392,"healthyVolunteers":387,"sex":393,"minAge":39,"maxAge":394,"enrollmentInfo":395,"targetDuration":39,"studyType":398,"phases":399,"briefSummary":401,"conditions":402,"keywords":39,"overallStatus":49,"whyStopped":39,"lastUpdateSubmitDate":409,"lastUpdatePostDateStruct":410,"startDateStruct":413,"completionDateStruct":415,"leadSponsor":417,"locationsCount":418},{"fullName":5,"class":6},"Scholar Rock, Inc.","INDUSTRY",[8,16],{"label":9,"type":10,"description":11,"interventionNames":12},"Apitegromab low dose + SMN Therapy","EXPERIMENTAL","Patients who are less than 2 years of age with SMA will receive apitegromab every 4 weeks by intravenous (IV) infusion during the 48-week Treatment Period.\n\nPatients must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam).",[13,14,15],"Drug: Apitegromab","Drug: Nusinersen","Drug: Risdiplam",{"label":17,"type":10,"description":11,"interventionNames":18},"Apitegromab high dose + SMN Therapy",[13,14,15],[20,27,33],{"type":21,"name":22,"description":23,"armGroupLabels":24,"otherNames":25},"DRUG","Apitegromab","Apitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)\u002Flambda isotype that specifically binds to human pro\u002Flatent myostatin with high affinity inhibiting myostatin activation. SRK-015 will be administered every 4 weeks by intravenous (IV) infusion.",[17,9],[26],"SRK-015",{"type":21,"name":28,"description":29,"armGroupLabels":30,"otherNames":31},"Nusinersen","Nusinersen is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered intrathecally per the prescribing information.",[17,9],[32],"Spinraza",{"type":21,"name":34,"description":35,"armGroupLabels":36,"otherNames":37},"Risdiplam","Risdiplam is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered orally per the prescribing information.",[17,9],[38],"Evrysdi",null,[41],{"name":42,"role":43,"phone":44,"phoneExt":39,"email":45},"Scholar Rock","CONTACT","857-259-3860","MedicalInformation@scholarrock.com",[47,66,81,95,110,125,140,155,170,185,201,216,230,244,258,275,284,293,309,320,326,335,346,357,366],{"facility":48,"status":49,"city":50,"state":51,"zip":52,"country":53,"countryCode":54,"cosmosGeoPoint":55,"geoPoint":60,"contacts":61},"Phoenix Children's Hospital","RECRUITING","Phoenix","Arizona","85016","United States","US",{"type":56,"coordinates":57},"Point",[58,59],-112.07404,33.44838,{"lat":59,"lon":58},[62],{"name":63,"role":43,"phone":64,"phoneExt":39,"email":65},"Melony Valdez","602-933-2017","mvaldez8@phoenixchildrens.com",{"facility":67,"status":49,"city":68,"state":69,"zip":70,"country":53,"countryCode":54,"cosmosGeoPoint":71,"geoPoint":75,"contacts":76},"Children's Hospital of Orange County (CHOC)","Orange","California","92868",{"type":56,"coordinates":72},[73,74],-117.85311,33.78779,{"lat":74,"lon":73},[77],{"name":78,"role":43,"phone":79,"phoneExt":39,"email":80},"Sabrine Serrano","714-509-7926","Sabrina.serrano@choc.org",{"facility":82,"status":49,"city":83,"state":69,"zip":84,"country":53,"countryCode":54,"cosmosGeoPoint":85,"geoPoint":89,"contacts":90},"Stanford Neuroscience Health Center (SNHC)","Palo Alto","94304",{"type":56,"coordinates":86},[87,88],-122.14302,37.44188,{"lat":88,"lon":87},[91],{"name":92,"role":43,"phone":93,"phoneExt":39,"email":94},"Wendy Ao","650-725-4341","NeuromuscularResearch@stanford.edu",{"facility":96,"status":49,"city":97,"state":98,"zip":99,"country":53,"countryCode":54,"cosmosGeoPoint":100,"geoPoint":104,"contacts":105},"Children's Hospital Colorado","Aurora","Colorado","80045",{"type":56,"coordinates":101},[102,103],-104.83192,39.72943,{"lat":103,"lon":102},[106],{"name":107,"role":43,"phone":108,"phoneExt":39,"email":109},"Hannah 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Stephan","319-356-2673","carrie-stephan@iowa.edu",{"facility":141,"status":49,"city":142,"state":143,"zip":144,"country":53,"countryCode":54,"cosmosGeoPoint":145,"geoPoint":149,"contacts":150},"Helen DeVos Children's Hospital at Spectrum Health","Grand Rapids","Michigan","49503",{"type":56,"coordinates":146},[147,148],-85.66809,42.96336,{"lat":148,"lon":147},[151],{"name":152,"role":43,"phone":153,"phoneExt":39,"email":154},"Rosa Sifontes-Escamilla","616-486-6342","rosa.sifontes-escamilla@helendevoschildrens.org",{"facility":156,"status":49,"city":157,"state":158,"zip":159,"country":53,"countryCode":54,"cosmosGeoPoint":160,"geoPoint":164,"contacts":165},"Atrium Health Wake Forest Baptist","Winston-Salem","North Carolina","27157",{"type":56,"coordinates":161},[162,163],-80.24422,36.09986,{"lat":163,"lon":162},[166],{"name":167,"role":43,"phone":168,"phoneExt":39,"email":169},"Donna Gallimore","336-716-3919","Donna.Gallimore@Advocatehealth.org",{"facility":171,"status":49,"city":172,"state":173,"zip":174,"country":53,"countryCode":54,"cosmosGeoPoint":175,"geoPoint":179,"contacts":180},"The Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"type":56,"coordinates":176},[177,178],-75.16362,39.95238,{"lat":178,"lon":177},[181],{"name":182,"role":43,"phone":183,"phoneExt":39,"email":184},"Karen Monono","267-425-0158","mononok@chop.edu",{"facility":186,"status":187,"city":188,"state":189,"zip":190,"country":53,"countryCode":54,"cosmosGeoPoint":191,"geoPoint":195,"contacts":196},"Le Bonheur Children's Hospital","NOT_YET_RECRUITING","Memphis","Tennessee","38103",{"type":56,"coordinates":192},[193,194],-90.04898,35.14953,{"lat":194,"lon":193},[197],{"name":198,"role":43,"phone":199,"phoneExt":39,"email":200},"Daniel Guillen (Mendoza)","901-287-7337","dguillen@uthsc.edu",{"facility":202,"status":49,"city":203,"state":204,"zip":205,"country":53,"countryCode":54,"cosmosGeoPoint":206,"geoPoint":210,"contacts":211},"University of Texas Southwestern Medical Center","Dallas","Texas","75207",{"type":56,"coordinates":207},[208,209],-96.80667,32.78306,{"lat":209,"lon":208},[212],{"name":213,"role":43,"phone":214,"phoneExt":39,"email":215},"Holly Lawrence","214-456-9561","Holly.Lawrence@utsouthwestern.edu",{"facility":217,"status":49,"city":218,"state":204,"zip":219,"country":53,"countryCode":54,"cosmosGeoPoint":220,"geoPoint":224,"contacts":225},"Neurology Rare Disease Center","Flower Mound","75028",{"type":56,"coordinates":221},[222,223],-97.09696,33.01457,{"lat":223,"lon":222},[226],{"name":227,"role":43,"phone":228,"phoneExt":39,"email":229},"Jennifer Avelar","972-999-1011","jennifer.avelar@neuromdcenter.com",{"facility":231,"status":49,"city":232,"state":204,"zip":233,"country":53,"countryCode":54,"cosmosGeoPoint":234,"geoPoint":238,"contacts":239},"Cook Children's Medical Center","Fort Worth","76104",{"type":56,"coordinates":235},[236,237],-97.32085,32.72541,{"lat":237,"lon":236},[240],{"name":241,"role":43,"phone":242,"phoneExt":39,"email":243},"Kayla Blough","682-303-1489","kayla.blough@cookchildrens.org",{"facility":245,"status":49,"city":246,"state":204,"zip":247,"country":53,"countryCode":54,"cosmosGeoPoint":248,"geoPoint":252,"contacts":253},"Texas Children's Hospital (TCH)-Clinical Care Center (CCC)","Houston","77030",{"type":56,"coordinates":249},[250,251],-95.36327,29.76328,{"lat":251,"lon":250},[254],{"name":255,"role":43,"phone":256,"phoneExt":39,"email":257},"Kim 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NeMO Milano - Fondazione Serena Onlus","20162",{"type":56,"coordinates":324},[317,318],{"lat":318,"lon":317},{"facility":327,"status":49,"city":328,"state":39,"zip":329,"country":313,"countryCode":314,"cosmosGeoPoint":330,"geoPoint":334,"contacts":39},"Fondazione Policlinico Universitario Agostino Gemelli IRCCS","Rome","00168",{"type":56,"coordinates":331},[332,333],12.51133,41.89193,{"lat":333,"lon":332},{"facility":336,"status":49,"city":337,"state":39,"zip":338,"country":339,"countryCode":340,"cosmosGeoPoint":341,"geoPoint":345,"contacts":39},"Universitair Medisch Centrum Utrecht","Utrecht","3584 CX","Netherlands","NL",{"type":56,"coordinates":342},[343,344],5.12222,52.09083,{"lat":344,"lon":343},{"facility":347,"status":49,"city":348,"state":39,"zip":349,"country":350,"countryCode":351,"cosmosGeoPoint":352,"geoPoint":356,"contacts":39},"Hospital Sant Joan de Déu Barcelona","Barcelona","08950","Spain","ES",{"type":56,"coordinates":353},[354,355],2.15899,41.38879,{"lat":355,"lon":354},{"facility":358,"status":49,"city":359,"state":39,"zip":360,"country":350,"countryCode":351,"cosmosGeoPoint":361,"geoPoint":365,"contacts":39},"Hospital Universitari i Politecnico La Fecnic La Fe","Valencia","46026",{"type":56,"coordinates":362},[363,364],-0.37966,39.47391,{"lat":364,"lon":363},{"facility":367,"status":49,"city":368,"state":369,"zip":370,"country":371,"countryCode":372,"cosmosGeoPoint":373,"geoPoint":377,"contacts":378},"Leeds General Infirmary Children's Research Department","Leeds","West Yorkshire","LS1 3EX","United Kingdom","UK",{"type":56,"coordinates":374},[375,376],-1.54785,53.79648,{"lat":376,"lon":375},[379],{"name":380,"role":43,"phone":381,"phoneExt":39,"email":382},"Anne-Marie Childs","+44 11 3392 2160","paedonc.research@nhs.net",{"type":384,"investigatorFullName":39,"investigatorTitle":39,"investigatorAffiliation":39,"oldNameTitle":39,"oldOrganization":39},"SPONSOR","100596567","phase-2-a-study-to-evaluate-how-apitegromab-works-in-subjects-who-are-less-than-2-years-old-and-have-spinal-muscular-atrophy-100596567",false,"NCT07047144","A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy","A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects \u003C2 Years Old With Spinal Muscular Atrophy (SMA)","OPAL","Inclusion Criteria:\n\n1. Is \\\u003C2 years old at the time of the informed consent\n2. Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth\n3. Has confirmed diagnosis of 5q autosomal recessive SMA\n4. Has confirmed presence of SMN2 gene copy(ies)\n5. Must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam)\n6. Body weight for age is no less than 1st percentile based on the WHO Child Growth Standards at the Screening Visit\n7. Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score \\\u003C55\n\nExclusion Criteria:\n\n1. Nutritional status that is not anticipated to be stable throughout the study or medical necessity for a gastric feeding tube, where most feeds are administered by this route\n2. Major orthopedic issues such as severe scoliosis or severe contractures or interventional procedure, including spine or hip surgery, which is considered to have the potential to substantially limit the ability of the subject to be evaluated on any motor function outcome measures, within 6 months before Screening or anticipated during the study\n3. Any other physical limitations (eg, the subject requires cast for contractures) that would prevent the subject from undergoing motor function outcome measures throughout the study.","ALL","2 Years",{"count":396,"type":397},52,"ESTIMATED","INTERVENTIONAL",[400],"PHASE2","This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK\u002FPD, efficacy, safety, and tolerability of apitegromab in subjects \\\u003C2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score \\\u003C55.",[403,404,405,406,407,408],"Spinal Muscular Atrophy","SMA","Spinal Muscular Atrophy Type 2","Spinal Muscular Atrophy Type 3","Neuromuscular Manifestations","Anti-myostatin","2026-04-29",{"date":411,"type":412},"2026-05-01","ACTUAL",{"date":414,"type":412},"2025-09-15",{"date":416,"type":397},"2029-03",{"name":5,"class":6},25]