[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100654835":3},{"organization":4,"outcomesModule":7,"designInfo":21,"detailedDescription":24,"studyPopulation":25,"armGroups":20,"interventions":20,"overallOfficials":26,"centralContacts":30,"locations":35,"responsibleParty":52,"collaborators":20,"id":54,"slug":55,"hasResults":56,"nctId":57,"briefTitle":58,"officialTitle":59,"acronym":20,"eligibilityCriteria":60,"healthyVolunteers":56,"sex":61,"minAge":20,"maxAge":20,"enrollmentInfo":62,"targetDuration":65,"studyType":66,"phases":20,"briefSummary":67,"conditions":68,"keywords":70,"overallStatus":37,"whyStopped":20,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":84,"startDateStruct":87,"completionDateStruct":89,"leadSponsor":91,"locationsCount":92},{"fullName":5,"class":6},"St. Jude Children's Research Hospital","OTHER",{"primaryOutcomes":8,"secondaryOutcomes":20,"otherOutcomes":20},[9,13,16],{"measure":10,"description":11,"timeFrame":12},"Maternal-fetal monitoring outcomes from instances of healthy women dosed with risdiplam to treat their fetus diagnosed with Spinal Muscular Atrophy (SMA)","Maternal-fetal monitoring outcome endpoints will be obtained from questionnaires and medical chart review. Analyses will be conducted using SAS software and will be summarized using descriptive statistics.","At enrollment and shortly after delivery",{"measure":14,"description":15,"timeFrame":12},"Pregnancy outcomes from instances of healthy women dosed with risdiplam to treat their fetus diagnosed with SMA","Pregnancy outcome endpoints will be obtained from questionnaires and medical chart review. Analyses will be conducted using SAS software and will be summarized using descriptive statistics.",{"measure":17,"description":18,"timeFrame":19},"Delivery outcomes associated with prenatally treated SMA","Delivery endpoints will be obtained from questionnaires and medical chart review. Analyses will be conducted using SAS software and will be summarized using descriptive statistics.","At delivery",null,{"allocation":20,"interventionModel":20,"interventionModelDescription":20,"primaryPurpose":20,"observationalModel":22,"timePerspective":23,"maskingInfo":20},"COHORT","PROSPECTIVE","Primary Objectives\n\n* Describe maternal-fetal monitoring and pregnancy outcomes from instances of healthy women dosed with risdiplam to treat their fetus diagnosed with Spinal Muscular Atrophy (SMA).\n* Describe delivery outcomes associated with prenatally treated SMA.\n\nSecondary Objectives\n\n* Describe postnatal clinical outcomes of women that were dosed with risdiplam during pregnancy to treat an affected fetus.\n* Describe clinical outcomes of children with SMA treated prenatally.\n\nThis is a longitudinal retrospective\u002Fprospective registry study with the expected sample size of 30 mother-child dyads. The study will collect data elements from cases of fetuses diagnosed with SMA in utero and where their mothers self-administered a disease-modifying therapy (DMT) to treat the affected fetus.\n\nInformation will be collected from the participant medical records, interviews, and surveys or questionnaires. Survey information will be collected at enrollment, shortly after delivery, and then on 6-month intervals starting 6 months after delivery. The questionnaires will be sent via email through an application called REDCap and may be completed by the participant (or someone helping them) directly or by interview with the support of a research staff member (remotely).\n\nThe total study duration is 7 years, consisting of a 3-year accrual period and minimum 4-year follow-up period.","Participants who meet the eligibility criteria and consent to participate.",[27],{"name":28,"affiliation":5,"role":29},"Richard Finkel, MD","PRINCIPAL_INVESTIGATOR",[31],{"name":28,"role":32,"phone":33,"phoneExt":20,"email":34},"CONTACT","888-226-4343","referralinfo@stjude.org",[36],{"facility":5,"status":37,"city":38,"state":39,"zip":40,"country":41,"countryCode":42,"cosmosGeoPoint":43,"geoPoint":48,"contacts":49},"RECRUITING","Memphis","Tennessee","38105","United States","US",{"type":44,"coordinates":45},"Point",[46,47],-90.04898,35.14953,{"lat":47,"lon":46},[50,51],{"name":28,"role":32,"phone":33,"phoneExt":20,"email":34},{"name":28,"role":29,"phone":20,"phoneExt":20,"email":20},{"type":53,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100654835","a-registry-of-cases-of-spinal-muscular-atrophy-100654835",false,"NCT07805278","A Registry of Cases of Spinal Muscular Atrophy","A Registry of Cases of Spinal Muscular Atrophy Diagnosed in the Prenatal Period That Received Disease-Modifying Therapy","Inclusion Criteria:\n\n* Current or past pregnancy during which the fetus received a genetic diagnosis of SMA.\n* Current or past treatment with SMA-directed therapy intended to treat the fetus during gestation (such as risdiplam, nusinersen, onasemnogene abeparvovec, or another product intended to treat SMA).\n\nExclusion Criteria:\n\n* Inability or unwillingness of research participant or legal guardian\u002Frepresentative to give written informed consent.","ALL",{"count":63,"type":64},30,"ESTIMATED","4 Years","OBSERVATIONAL","Because no long-term record of prenatal Spinal Muscular Atrophy (SMA) treatment exists, this study will address an important gap in the understanding of the safety and benefit of treatment while in the womb. The registry will help guide best practices for monitoring pregnant women and their fetus with SMA, if treated with a medication while in the womb, and to follow the baby after birth for signs of safety and added benefit birth and support future research and study development.",[69],"Spinal Muscular Atrophy",[71,72,73,74,75,76,77,78,79,80,81,82],"Spinal Muscular Atrophy (SMA)","Registry Study","Fetuses Diagnosed with SMA","Current or Past Pregnancy","SMA Treatment During Gestation","Risdiplam","Nusinersen","Onasemnogene Abeparvovec","Prenatal SMA","Fetal SMA","Prenatal risdiplam","Fetal risdiplam","2026-09-29",{"date":85,"type":86},"2026-09-30","ACTUAL",{"date":88,"type":64},"2026-10",{"date":90,"type":64},"2033-08",{"name":5,"class":6},1]