[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100530263":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":10,"centralContacts":22,"locations":28,"responsibleParty":63,"collaborators":10,"id":65,"slug":66,"hasResults":67,"nctId":68,"briefTitle":69,"officialTitle":70,"acronym":71,"eligibilityCriteria":72,"healthyVolunteers":67,"sex":73,"minAge":10,"maxAge":74,"enrollmentInfo":75,"targetDuration":10,"studyType":78,"phases":10,"briefSummary":79,"conditions":80,"keywords":10,"overallStatus":31,"whyStopped":10,"lastUpdateSubmitDate":82,"lastUpdatePostDateStruct":83,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":91},{"fullName":5,"class":6},"Chiesi Farmaceutici S.p.A.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Paediatric patients with alpha-mannosidosis treated with Lamzede before 3 years of age",null,"Paediatric patients with a confirmed diagnosis of alpha-mannosidosis with data for at least one pre- and one post-Lamzede treatment sample obtained when \\\u003C 3 YOA.",[13],"Drug: Velmanase Alfa",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"DRUG","Velmanase Alfa","Lamzede® (velmanase alfa, henceforth referred to as Lamzede) is a recombinant human lysosomal alpha-mannosidase product developed as an intravenous enzyme replacement therapy (ERT) for the treatment of alpha-mannosidosis.",[9],[21],"Lamzede",[23],{"name":24,"role":25,"phone":26,"phoneExt":10,"email":27},"Chiesi Clinical trials","CONTACT","+39.0521 2791","clinicaltrials_info@chiesi.com",[29,48],{"facility":30,"status":31,"city":32,"state":33,"zip":34,"country":35,"countryCode":36,"cosmosGeoPoint":37,"geoPoint":42,"contacts":43},"Nicklaus Children's Hospital","RECRUITING","Miami","Florida","33155","United States","US",{"type":38,"coordinates":39},"Point",[40,41],-80.19366,25.77427,{"lat":41,"lon":40},[44],{"name":45,"role":25,"phone":46,"phoneExt":10,"email":47},"Parul Jayakar","786-624-4717","Parul.Jayakar@Nicklaushealth.org",{"facility":49,"status":31,"city":50,"state":51,"zip":52,"country":35,"countryCode":36,"cosmosGeoPoint":53,"geoPoint":57,"contacts":58},"Greenwood Genetic Center","Greenwood","South Carolina","29605",{"type":38,"coordinates":54},[55,56],-82.16179,34.1954,{"lat":56,"lon":55},[59],{"name":60,"role":25,"phone":61,"phoneExt":10,"email":62},"Laura Buch","864-250-7944","lbuch@ggc.org",{"type":64,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR","100530263","analysis-of-velmanase-alfa-lamzedes-effects-in-the-body-of-children-with-alpha-mannosidosis-under-the-age-3-100530263",false,"NCT06184503","Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3","A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age","LAMPO(0-3)","Inclusion Criteria:\n\n* Subjects with the provision of informed consent from their legal guardians (LAR)\n* Have a confirmed diagnosis of alpha mannosidosis\n* Have initiated treatment with velmanase alfa between birth to at least six weeks before turning 3 years of age\n* Have information on the disease marker GlcNAc(Man)2 obtained:\n\nbefore velmanase treatment initiation (ideally max 6 month before), and at least one post-treatment sample, collected following at least six weeks of treatment.\n\n\\- Participants treated with Lamzede, 1 mg\u002Fkg body weight, via weekly intravenous infusions.\n\nExclusion Criteria:\n\nParticipants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).","ALL","3 Years",{"count":76,"type":77},5,"ESTIMATED","OBSERVATIONAL","The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis.\n\nThe main questions it aims to answer are:\n\n* study the effect of velmanase alfa on a marker of the disease called GlcNAc(Man)2 after one year of therapy\n* explore how the child's body reacts to velmanase alfa during the therapy The parents or legal guardians of participants will be asked to provide the results of analyses performed in the routine clinical setting related to the participant's general health and the administration of velmanase alfa.\n\nAdditional data will be extracted from other observational sponsored studies\u002Fregistries, compassionate use programs, investigator-initiated studies (IIS), and published case reports (presented in the literature) if existing.",[81],"Alpha-Mannosidosis","2025-05-15",{"date":84,"type":85},"2025-05-16","ACTUAL",{"date":87,"type":85},"2025-02-19",{"date":89,"type":77},"2029-09",{"name":5,"class":6},2]