[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05146830":3,"trial-entities:NCT05146830":118,"trial-summary:NCT05146830":121},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":6,"overall_status":7,"completion_date":8,"status_verified_date":9,"last_update_date":10,"start_date":11,"sponsor_name":12,"lead_sponsor_class":13,"has_dmc":14,"brief_summary":15,"detailed_description":16,"conditions":17,"keywords":19,"study_type":24,"primary_purpose":25,"phases":26,"enrollment_info":27,"interventions":30,"primary_outcomes":38,"secondary_outcomes":48,"sex":83,"minimum_age":84,"maximum_age":85,"healthy_volunteers":14,"eligibility_criteria":86,"std_ages":90,"locations":93,"central_contacts":103,"overall_officials":104,"references":109,"see_also_links":117},"NCT05146830","CTNS-RD-04-LTFU01","A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04","ENROLLING_BY_INVITATION","2036-11-30","2024-07","2024-07-03","2022-11-14","Stephanie Cherqui","OTHER",false,"This is a multinational, long-term follow-up study to assess the long-term safety and durability of CTNS-RD-04 treatment in participants who received a single dose administration of lentiviral gene therapy. No investigational product will be administered in this study. Participants will continue periodic safety and efficacy assessments in this long-term follow-up study up to 15 years from the initial date of CTNS-RD-04 infusion.","Participants enrolled in a study where the individual received CTNS-RD-04 will be offered participation in the CTNS-RD-04-LTF01 study. The Baseline visit for the CTNS-RD-04-LTF01 study will likely coincide with the final visit in the parent study. Participants confirmed eligible for the CTNS-RD-04-LTF01 study will be asked to return for study visits at approximately 6-month intervals for the first 4 years and annually thereafter for up to 11 years until a total of 15 years have elapsed during which time continued safety, engraftment, and efficacy of CTNS-RD-04 treatment will be assessed.",[18],"Cystinosis",[20,21,22,23],"Cystinosis Disease","Cell therapy","Gene therapy","Long-term follow-up","OBSERVATIONAL",null,[],{"count":28,"type":29},50,"ESTIMATED",[31],{"type":13,"name":32,"description":33,"armGroupLabels":34,"otherNames":36},"Safety and Efficacy Assessments","Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.",[35],"Participants with Cystinosis Disease",[37],"Gene Therapy Intervention",[39,42,44,46],{"measure":40,"timeFrame":41},"Incidence of clinically significant Adverse Events (AEs) and Serious Adverse Events (SAEs)","Baseline to Year 15 post gene therapy",{"measure":43,"timeFrame":41},"Number of participants with clinically relevant abnormalities, as assessed by vital sign (heart rate, pulse rate, and temperature)",{"measure":45,"timeFrame":41},"Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests (chemistry and hematology)",{"measure":47,"timeFrame":41},"Number of participants with clinically relevant abnormalities, as assessed by by electrocardiograms (ECGs) (rate, rhythm, intervals)",[49,51,53,55,57,59,61,63,65,67,69,71,73,75,77,79,81],{"measure":50,"timeFrame":41},"Change from baseline in Corneal cystine crystal score (CCCS) as assessed by in vivo confocal microscopy (IVCM)",{"measure":52,"timeFrame":41},"Change from baseline in Renal glomerular and tubular functions measured by glomerular filtration rate (GFR)",{"measure":54,"timeFrame":41},"Change from baseline in vision function as assessed by ophthalmology exams",{"measure":56,"timeFrame":41},"Change from baseline in grip strength measured by dynamometry",{"measure":58,"timeFrame":41},"Change from baseline in respiratory function measured by spirometry",{"measure":60,"timeFrame":41},"Change from baseline in bone density assessed by dual-energy X-ray absorptiometry (DEXA)",{"measure":62,"timeFrame":41},"Change from baseline in muscle mass assessed by dual-energy X-ray absorptiometry (DEXA)",{"measure":64,"timeFrame":41},"Change from baseline in Endocrine function measured by fasting glucose, thyroid function, and gonadotropin levels",{"measure":66,"timeFrame":41},"Change from baseline in neurological function assessed by neurological exam (mental status, coordination, sensory, reflexes, and visual motor integration)",{"measure":68,"timeFrame":41},"Change from baseline in psychometric function assessed by neurological exam (memory, oromotor function, intelligence quotient (IQ))",{"measure":70,"timeFrame":41},"Change from baseline in pill\u002Finjection count related to cystinosis treatment",{"measure":72,"timeFrame":41},"Change from baseline in Cystinosin (CTNS) as assessed by quantitative Polymerase Chain Reaction (qPCR)",{"measure":74,"timeFrame":41},"Change from baseline in mean Vector Copy Number (VCN) as assessed by quantitative Polymerase Chain Reaction (qPCR)",{"measure":76,"timeFrame":41},"Change from baseline in Cystine levels in leukocytes measured by mass spectrometry",{"measure":78,"timeFrame":41},"Change from baseline in ovarian reserve and menstrual cycle as assessed by anti-Müllerian hormone (AMH) and gynecology exams",{"measure":80,"timeFrame":41},"Change in male reproductive potential as assessed by urology exams (sperm count, motility, and morphology)",{"measure":82,"timeFrame":41},"Absence of Replication Competent Lentivirus (RCL) as assessed by Elisa assay","ALL","14 Years","50 Years",{"inclusion":87,"exclusion":88,"raw_text":89},[],[],"Inclusion Criteria:\n\n• Participant must have received CTNS-RD-04 in a preceding study\n\nExclusion Criteria:\n\n• Participant is currently enrolled in an CTNS-RD-04 treatment study. Participants who have either completed, withdrawn, or prematurely discontinued participation for any reason at any time after receiving CTNS-RD-04 are eligible for CTNS RD 04 LTF01 study participation.",[91,92],"CHILD","ADULT",[94],{"facility":95,"city":96,"state":97,"zip":98,"country":99,"geoPoint":100},"University of California San Diego","La Jolla","California","92037","United States",{"lat":101,"lon":102},32.84727,-117.2742,[],[105],{"name":106,"affiliation":107,"role":108},"Stephanie Cherqui, PhD","University of California, San Diego","PRINCIPAL_INVESTIGATOR",[110,114],{"pmid":111,"type":112,"citation":113},"23089735","RESULT","Harrison F, Yeagy BA, Rocca CJ, Kohn DB, Salomon DR, Cherqui S. Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis. Mol Ther. 2013 Feb;21(2):433-44. doi: 10.1038\u002Fmt.2012.214. Epub 2012 Oct 23.",{"pmid":115,"type":112,"citation":116},"25186209","Naphade S, Sharma J, Gaide Chevronnay HP, Shook MA, Yeagy BA, Rocca CJ, Ur SN, Lau AJ, Courtoy PJ, Cherqui S. Brief reports: Lysosomal cross-correction by hematopoietic stem cell-derived macrophages via tunneling nanotubes. Stem Cells. 2015 Jan;33(1):301-9. doi: 10.1002\u002Fstem.1835.",[],{"nct_id":4,"conditions":119,"biomarkers":120},[18],[],{"nct_id":4,"found":14,"summary":25,"prompt_version":25}]