[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"genetic-diseases-x-linked\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:genetic-diseases-x-linked":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,58],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":18,"phases":4,"briefSummary":19,"conditions":20,"keywords":30,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":50,"lastUpdatePostDateStruct":51,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":54,"locationsCount":57},"100612219","managed-access-program-for-del-zota-in-participants-with-dmd-mutations-amenable-to-exon-44-skipping-100612219",false,"NCT07250737","Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping","Managed Access to Investigational Use of AOC 1044 in Participants With DMD Mutations Amenable to Exon 44 Skipping","Key Inclusion Criteria\n\nRollover Participants\n\n* Completed Study EXPLORE44-OLE Treatment Period (through W102)\n* No significant tolerability issues with AOC 1044\n\nNew (Non-Rollover) Participants\n\n* Permanently residing in the US and have a US primary health care provider\n* Documented dystrophin gene mutation that is amenable to exon 44 skipping\n* Age 2 or older at the time of consent\n* If previously treated with gene therapy for DMD, treatment and associated immunosuppressive regimen was more than 12 months before consent and in the opinion of the prescriber, participant has had an unsatisfactory treatment response\n\nKey Exclusion Criteria\n\nRollover Participants\n\n• Prescence of any new condition or worsening of existing condition that could affect participant's safety or ability to comply with the program requirements\n\nNew (Non-Rollover) Participants\n\n* Recently treated with or on a clinical study for another investigation drug\n* Serious respiratory or cardiac dysfunction, or nearing end of life\n* Screening laboratory parameters do not meet protocol requirements\n* History of multiple drug allergies or to any component of AOC 1044\n* Participants who discontinued early from the treatment period of EXPLORE44 or EXPLORE44-OLE","MALE","2 Years","EXPANDED_ACCESS","The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.",[21,22,23,24,25,26,27,28,29],"Muscular Disorders, Atrophic","Muscular Diseases","Musculoskeletal Diseases","Neuromuscular Diseases","Nervous System Diseases","Genetic Diseases, Inborn","Genetic Diseases, X-Linked","Muscular Dystrophies","Muscular Dystrophy, Duchenne",[31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48],"Avidity","Avidity Biosciences","AOC 1044","AOC 1044-CS1","AOC 1044-CS2","EXPLORE44","EXPLORE44-OLE","Del-zota","delpacibart zotadirsen","DMD","exon skipping therapy","dystrophin","managed access","expanded access","pre-approval access","compassionate use","MAP","EAP","AVAILABLE","2026-06-26",{"date":52,"type":53},"2026-06-29","ACTUAL",{"name":55,"class":56},"Avidity Biosciences, Inc.","INDUSTRY",14,{"id":59,"slug":60,"hasResults":11,"nctId":61,"briefTitle":62,"officialTitle":63,"acronym":64,"eligibilityCriteria":65,"healthyVolunteers":11,"sex":66,"minAge":67,"maxAge":68,"enrollmentInfo":69,"targetDuration":72,"studyType":73,"phases":4,"briefSummary":74,"conditions":75,"keywords":84,"overallStatus":90,"whyStopped":4,"lastUpdateSubmitDate":50,"lastUpdatePostDateStruct":91,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":97,"locationsCount":100},"100472474","rett-syndrome-registry-100472474","NCT05432349","Rett Syndrome Registry","Rett Syndrome Real World Data Observational Registry","RSR","Inclusion Criteria:\n\n* Male or female with a pathologic loss of function alteration of MECP2\n\nExclusion Criteria:\n\n* Male or female with a gain of function alteration of MECP2, including those with MEPC2 duplication or triplication","ALL","0 Years","99 Years",{"count":70,"type":71},3000,"ESTIMATED","5 Years","OBSERVATIONAL","The Rett Syndrome Registry is a longitudinal observational study of individuals with MECP2 mutations and a diagnosis of Rett syndrome. Designed together with the IRSF Rett Syndrome Center of Excellence Network medical directors, this study collects data on the signs and symptoms of Rett syndrome as reported by the Rett syndrome experts and by the caregivers of individuals with Rett syndrome. This study will be used to develop consensus based guidelines for the care of your loved ones with Rett syndrome and to facilitate the development of better clinical trials and other aspects of the drug development path for Rett syndrome.",[76,77,78,27,79,80,81,82,83,25],"Rett Syndrome","Rett Syndrome, Atypical","Genetic Disease","Intellectual Disability","Neurobehavioral Manifestations","Neurologic Manifestations","Neurologic Disorder","Neurodevelopmental Disorders",[85,86,87,88,89],"Rett syndrome","MECP2","Neurodevelopmental disorder","Registry","Natural History Study","RECRUITING",{"date":92,"type":53},"2026-06-30",{"date":94,"type":53},"2022-08-02",{"date":96,"type":71},"2028-07",{"name":98,"class":99},"International Rett Syndrome Foundation","OTHER",19]