[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Genetix Biotherapeutics Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":64},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,42],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":18,"targetDuration":21,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":26,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100533330","a-study-of-participants-with-cerebral-adrenoleukodystrophy-cald-treated-with-elivaldogene-autotemcel-100533330",false,"NCT06224413","A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel","A Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)","Stargazer","Inclusion Criteria:\n\nParticipants who fulfill the following criteria will be eligible for inclusion in this Registry Study.\n\n* Participant must be treated with eli-cel in the post marketing setting at a center in the United States (US) that participates in the Registry Study.\n* Participant must have provided an informed consent and\u002For assent to participate in Center for International Blood and Marrow Transplant Research (CIBMTR) registry.\n* Participant must have provided an informed consent and\u002For assent to participate in the Registry Study.\n* Participant must receive follow up care by a US-based physician with the ability to submit REG-502 data.\n\nRegistry Study Subpopulation inclusion:\n\nTwenty-four of the 120 patients in the Registry Study must meet the following inclusion criteria which will be used to create the more advanced early active CALD subpopulation:\n\n• Participant must meet the above inclusion criteria and have a Loes score of 4.5 through 9.0 with GdE+ (gadolinium enhancement positivity) from an MRI performed before treatment with eli-cel and with NFS of 0 or 1 at baseline.\n\nExclusion Criteria:\n\nThere are no exclusion criteria for this Registry Study.","MALE",{"count":19,"type":20},120,"ESTIMATED","15 Years","OBSERVATIONAL","The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major functional disability (MFD)-free survival over time in participants with more advanced early active CALD. All enrolled participants with CALD treated with eli-cel in the post-marketing setting will be followed in this study for 15 years. No investigational drug product will be administered in this study. This study will enroll 120 participants with CALD treated with eli-cel in the post-marketing setting. A subpopulation of 24 participants with more advanced early active CALD will be specifically enrolled as required by the US FDA as a condition of accelerated approval and will be considered as a separate cohort for effectiveness outcomes.",[25],"Cerebral Adrenoleukodystrophy (CALD)",[27,28],"Adrenoleukodystrophy","Adrenoleukodystrophy protein","RECRUITING","2025-06-30",{"date":32,"type":33},"2025-07-02","ACTUAL",{"date":35,"type":33},"2024-03-27",{"date":37,"type":20},"2047-12-30",{"name":39,"class":40},"Genetix Biotherapeutics Inc.","INDUSTRY",3,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":4,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":49,"minAge":4,"maxAge":4,"enrollmentInfo":50,"targetDuration":21,"studyType":22,"phases":4,"briefSummary":52,"conditions":53,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":55,"lastUpdatePostDateStruct":56,"startDateStruct":58,"completionDateStruct":60,"leadSponsor":62,"locationsCount":63},"100536952","a-study-of-participants-with--thalassemia-treated-with-betibeglogene-autotemcel-100536952","NCT06271512","A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel","A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)","Inclusion Criteria:\n\n* Participant must be treated with beti-cel in the post marketing setting at a center in the US that participates in the Registry.\n* Participant must sign an informed consent and\u002For assent prior to enrollment as required under applicable laws and regulations.\n* Participant must have signed an informed consent and\u002For assent permitting data to be shared with Center for International Blood and Marrow Transplant Research (CIBMTR).\n* Participant must be followed by a hematologist based in the US.\n\nExclusion Criteria:\n\n\\- There are no exclusion criteria for Registry participation.","ALL",{"count":51,"type":20},150,"The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.",[54],"Beta-Thalassemia","2025-02-10",{"date":57,"type":33},"2025-02-11",{"date":59,"type":33},"2024-01-23",{"date":61,"type":20},"2043-12",{"name":39,"class":40},6,""]