[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"cerebral-adrenoleukodystrophy-cald\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:cerebral-adrenoleukodystrophy-cald":54},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,40],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":29,"startDateStruct":32,"completionDateStruct":34,"leadSponsor":36,"locationsCount":39},"100502254","phase-3-a-clinical-study-to-assess-the-efficacy-and-safety-of-leriglitazone-in-adult-male-subjects-with-cerebral-adrenoleukodystrophy-100502254",false,"NCT05819866","A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy","CALYX","Key Inclusion Criteria:\n\n* Subject is male and aged ≥18 years.\n* Subject has progressive cALD, defined as GdE+ brain lesions.\n* Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT.\n* Subject has a Loes score ≥0.5 and ≤12 at Screening.\n* Subject does not have major functional disability in the Major Functional Disabilities-Neurological Function Score (MFD-NFS), except for \"wheelchair bound\" or \"total incontinence\", which will be allowed as these are considered expected symptoms of AMN in the time course of the disease\n* Subject does not have major cognitive impairment which would impair his ability to take part in the study as determined by the investigator at screening.\n\nKey Exclusion Criteria:\n\n* Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel).\n* Subject has known type 1 or type 2 diabetes.\n* Subject has known hypersensitivity or intolerance to pioglitazone or any other thiazolidinedione.\n* Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to Screening.\n* Subject with current participation in another interventional clinical study or within 1 month prior to Screening.\n* Subject with other medical, neuropsychiatric or social conditions that, in the opinion of the investigator, are likely to adversely affect the risk-benefit of study participation, interfere with study compliance, or confound the study results.","MALE","18 Years",{"count":19,"type":20},40,"ESTIMATED","INTERVENTIONAL",[23],"PHASE3","A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.",[26],"Cerebral Adrenoleukodystrophy (cALD)","RECRUITING","2025-11-14",{"date":30,"type":31},"2025-11-17","ACTUAL",{"date":33,"type":31},"2023-07-12",{"date":35,"type":20},"2027-05",{"name":37,"class":38},"Minoryx Therapeutics, S.L.","INDUSTRY",13,{"id":41,"slug":42,"hasResults":11,"nctId":43,"briefTitle":44,"officialTitle":45,"acronym":46,"eligibilityCriteria":47,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":48,"targetDuration":50,"studyType":51,"phases":4,"briefSummary":52,"conditions":53,"keywords":55,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":67},"100533330","a-study-of-participants-with-cerebral-adrenoleukodystrophy-cald-treated-with-elivaldogene-autotemcel-100533330","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.",{"count":49,"type":20},120,"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.",[54],"Cerebral Adrenoleukodystrophy (CALD)",[56,57],"Adrenoleukodystrophy","Adrenoleukodystrophy protein","2025-06-30",{"date":60,"type":31},"2025-07-02",{"date":62,"type":31},"2024-03-27",{"date":64,"type":20},"2047-12-30",{"name":66,"class":38},"Genetix Biotherapeutics Inc.",3]