[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100084645":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":25,"locations":30,"responsibleParty":45,"collaborators":10,"id":47,"slug":48,"hasResults":49,"nctId":50,"briefTitle":51,"officialTitle":51,"acronym":10,"eligibilityCriteria":52,"healthyVolunteers":49,"sex":53,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":10,"studyType":59,"phases":10,"briefSummary":60,"conditions":61,"keywords":62,"overallStatus":33,"whyStopped":10,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":72,"completionDateStruct":10,"leadSponsor":74,"locationsCount":75},{"fullName":5,"class":6},"National Institutes of Health Clinical Center (CC)","NIH",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Cystinosis",null,"Patients with a diagnosis of cystinosis",[13],"Drug: Cysteamine",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":10},"DRUG","Cysteamine","Cystine-depleting agent",[9],[21],{"name":22,"affiliation":23,"role":24},"William A Gahl, M.D.","National Human Genome Research Institute (NHGRI)","PRINCIPAL_INVESTIGATOR",[26],{"name":22,"role":27,"phone":28,"phoneExt":10,"email":29},"CONTACT","(301) 402-2739","gahlw@mail.nih.gov",[31],{"facility":32,"status":33,"city":34,"state":35,"zip":36,"country":37,"countryCode":38,"cosmosGeoPoint":39,"geoPoint":44,"contacts":10},"National Institutes of Health Clinical Center","RECRUITING","Bethesda","Maryland","20892","United States","US",{"type":40,"coordinates":41},"Point",[42,43],-77.10026,38.98067,{"lat":43,"lon":42},{"type":46,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR","100084645","use-of-cysteamine-in-the-treatment-of-cystinosis-100084645",false,"NCT00359684","Use of Cysteamine in the Treatment of Cystinosis","* INCLUSION CRITERIA:\n\nDiagnosis of cystinosis, whether classical or one of the variants with later onset or no renal complications.\n\nPatients will be diagnosed as having cystinosis based upon a leucocyte cystine content greater than 1 nmol half-cystine\u002Fmg protein (normal, less than 0.2) and a typical clinical course.\n\nEXCLUSION CRITERIA:\n\nInability to travel to the NIH.\n\nAge less than one week.\n\nNonviable neonates and neonates of uncertain viability will be excluded.","ALL","1 Week","115 Years",{"count":57,"type":58},330,"ESTIMATED","OBSERVATIONAL","Cystinosis is an inherited disease resulting in poor growth and kidney failure. There is no known cure for cystinosis, although kidney transplantation may help the renal failure and prolong survival. Both the kidney damage and growth failure are thought to be due to the accumulation of the amino acid cystine within the cells of the body. The cystine storage later damages other organs besides the kidneys, including the thyroid gland, pancreas, eyes, and muscle.\n\nThe drug cysteamine (Cystagon; ProCysBi) is an oral medication given to patients with cystinosis prior to kidney transplantation. The drug works by reducing the level of cystine in the white blood cells and muscle tissue. The drug may also decrease levels of cystine in the kidneys and other tissues.\n\nThis study has several goals:\n\n1. Long-term surveillance of cysteamine treated patients.\n2. Detection of new non-kidney complications of cystinosis.\n3. Maintenance of a patient population for genetic testing (mutational analysis) of the cystinosis gene.\\\u003CTAB\\>",[9],[9,63,64,65,66,67],"Cystine","Lysomal Storage Disease","Mutation Analysis","Metabolic Disease","Natural History","2026-06-27",{"date":70,"type":71},"2026-06-30","ACTUAL",{"date":73,"type":71},"1979-01-04",{"name":23,"class":6},1]