[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100231593":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":25,"locations":26,"responsibleParty":94,"collaborators":25,"id":96,"slug":97,"hasResults":98,"nctId":99,"briefTitle":100,"officialTitle":101,"acronym":102,"eligibilityCriteria":103,"healthyVolunteers":104,"sex":105,"minAge":25,"maxAge":106,"enrollmentInfo":107,"targetDuration":25,"studyType":110,"phases":111,"briefSummary":114,"conditions":115,"keywords":25,"overallStatus":117,"whyStopped":25,"lastUpdateSubmitDate":118,"lastUpdatePostDateStruct":119,"startDateStruct":121,"completionDateStruct":123,"leadSponsor":125,"locationsCount":127},{"fullName":5,"class":6},"Sanofi","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Olipudase alfa","EXPERIMENTAL","Participants received intravenous (IV) infusion of olipudase alfa once every 2 weeks (Q2W) for 64 weeks. Each participant underwent a dose escalation according to the following paradigm: 0.03, 0.1, 0.3, 0.3, 0.6, 0.6, 1.0, 2.0, 3.0 milligram per kilogram (mg\u002Fkg). Three (3) mg\u002Fkg was the target maintenance dose, which was maintained for the remaining duration of 64 treatment weeks.",[13],"Drug: Olipudase alfa",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous infusion",[9],[20],"GZ402665",[22],{"name":23,"affiliation":5,"role":24},"Clinical Sciences & Operations","STUDY_DIRECTOR",null,[27,39,50,61,72,83],{"facility":28,"status":25,"city":29,"state":29,"zip":30,"country":31,"countryCode":32,"cosmosGeoPoint":33,"geoPoint":38,"contacts":25},"Investigational Site Number 840001","New York","10029","United States","US",{"type":34,"coordinates":35},"Point",[36,37],-74.00597,40.71427,{"lat":37,"lon":36},{"facility":40,"status":25,"city":41,"state":25,"zip":42,"country":43,"countryCode":44,"cosmosGeoPoint":45,"geoPoint":49,"contacts":25},"Investigational Site Number 076001","Porto Alegre","90035 003","Brazil","BR",{"type":34,"coordinates":46},[47,48],-51.23019,-30.03283,{"lat":48,"lon":47},{"facility":51,"status":25,"city":52,"state":25,"zip":53,"country":54,"countryCode":55,"cosmosGeoPoint":56,"geoPoint":60,"contacts":25},"Investigational Site Number 250002","Bron","69677","France","FR",{"type":34,"coordinates":57},[58,59],4.91303,45.73865,{"lat":59,"lon":58},{"facility":62,"status":25,"city":63,"state":25,"zip":64,"country":65,"countryCode":66,"cosmosGeoPoint":67,"geoPoint":71,"contacts":25},"Investigational Site Number 276001","Mainz","55131","Germany","DE",{"type":34,"coordinates":68},[69,70],8.28008,49.98185,{"lat":70,"lon":69},{"facility":73,"status":25,"city":74,"state":25,"zip":75,"country":76,"countryCode":77,"cosmosGeoPoint":78,"geoPoint":82,"contacts":25},"Investigational Site Number 380001","Udine","33100","Italy","IT",{"type":34,"coordinates":79},[80,81],13.23715,46.0693,{"lat":81,"lon":80},{"facility":84,"status":25,"city":85,"state":25,"zip":86,"country":87,"countryCode":88,"cosmosGeoPoint":89,"geoPoint":93,"contacts":25},"Investigational Site Number 826001","Manchester","M13 9WL","United Kingdom","UK",{"type":34,"coordinates":90},[91,92],-2.23743,53.48095,{"lat":92,"lon":91},{"type":95,"investigatorFullName":25,"investigatorTitle":25,"investigatorAffiliation":25,"oldNameTitle":25,"oldOrganization":25},"SPONSOR","100231593","phase-1-safety-tolerability-pk-and-efficacy-evaluation-of-repeat-ascending-doses-of-olipudase-alfa-in-pediatric-patients-18-years-of-age-with-acid-sphingomyelinase-deficiency-100231593",true,"NCT02292654","Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients \u003C18 Years of Age With Acid Sphingomyelinase Deficiency","A Phase 1\u002F2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Aged \u003C18 Years With Acid Sphingomyelinase Deficiency","ASCEND-Peds","Inclusion criteria :\n\n* The participant and\u002For participant's parent(s)\u002Flegal guardian(s) must provide written informed assent\u002Fconsent prior to any protocol-related procedures being performed.\n* The participant was \\\u003C18 years of age on the date of informed assent\u002Fconsent.\n* The participant had documented deficiency of acid sphingomyelinase as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes.\n* The participant had a spleen volume greater than or equal to (\\>=) 5 multiples of normal (MN) measured by magnetic resonance imaging (MRI); participants who had partial splenectomy were allowed if the procedure was performed \\>=1 year before screening and the residual spleen volume was \\>=5 MN.\n* The participant's height was -1 Z-score or lower.\n* A negative serum pregnancy test in female participants of childbearing potential.\n* Female participants of childbearing potential and male participants must be willing to practice true abstinence in line with their preferred and usual lifestyle or use 2 acceptable effective methods of contraception.\n\nExclusion criteria:\n\n* The participant had received an investigational drug within 30 days before study enrollment.\n* The participant had any of the following medical conditions:\n* An active, serious, intercurrent illness.\n* Active hepatitis B or hepatitis C infection.\n* Infection with human immunodeficiency virus (HIV).\n* Cirrhosis (determined by clinical evaluation).\n* Significant cardiac disease (eg, clinically significant arrhythmia, moderate or severe pulmonary hypertension or valvular dysfunction, or \\\u003C40 percent (%) left ventricular ejection fraction by echocardiogram).\n* Malignancy diagnosed within the previous 5 years (except basal cell carcinoma).\n* Any other extenuating circumstance that can significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.\n* The participant had acute or rapidly progressive neurological abnormalities.\n* The participant was homozygous for SMPD1 gene mutations R496L, L302P, and fs330 or any combination of these 3 mutations.\n* The participant had a delay of gross motor skills.\n* The participant had a major organ transplant (eg, bone marrow, liver).\n* The participant required use of invasive ventilatory support.\n* The participant required use of noninvasive ventilatory support while awake and for greater than (\\>)12 hours a day.\n* The participant in the investigator's opinion, was unable to adhere to the requirements of the study.\n* The participant had a platelet count \\\u003C60 × 10\\^3\u002FµL (based on the average of 2 screening samples obtained up to 24 hours apart).\n* The participant had alanine aminotransferase or aspartate aminotransferase \\>250 IU\u002FL or total bilirubin \\>1.5 mg\u002FdL.\n* The participant had an international normalized ratio (INR) \\>1.5.\n* The participant was unwilling or unable to abstain from ingesting alcohol the day before through 3 days after each infusion of olipudase alfa during the treatment period. Measuring alcohol concentration in blood was not required.\n* The participant was scheduled during the study for in-patient hospitalization including elective surgery.\n* The participant required medication(s) that may can decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants \\[eg, imipramine, or desipramine\\]).\n* The participant was breast-feeding.\n\nThe above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.",false,"ALL","17 Years",{"count":108,"type":109},20,"ACTUAL","INTERVENTIONAL",[112,113],"PHASE1","PHASE2","Primary Objective:\n\nTo evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.\n\nSecondary Objective:\n\nTo characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.",[116],"Sphingomyelin Lipidosis","COMPLETED","2022-03-15",{"date":120,"type":109},"2022-03-23",{"date":122,"type":109},"2015-05-01",{"date":124,"type":109},"2019-12-09",{"name":126,"class":6},"Genzyme, a Sanofi Company",6]