[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"battens-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:battens-disease":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,50],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":16,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":24,"conditions":25,"keywords":31,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"100439876","caregiving-networks-across-disease-context-and-the-life-course-100439876",false,"NCT05007990","Caregiving Networks Across Disease Context and the Life Course","Caregiving Networks Across Disease Context and the Life Course: A Comparative Longitudinal Study","* INCLUSION CRITERIA:\n\nTo be eligible to participate in this study, an individual must meet all of the following criteria:\n\n* Adults aged 18 years and older\n* If the Care Recipient is living, they must self-identify as a primary caregiver to the Care Recipient (individual with a chronic medical condition), OR if the Care Recipient is deceased, they must self-identify as having been a primary caregiver to the now-deceased Care Recipient, OR they must otherwise be identified (i.e., referred) by a participant as a part of the caregiving network\n* Ability to consent to research\n* Fluency in English will be needed to complete interview as well as to read, comprehend surveys and consent forms, as appropriate validated measures in other languages are not readily available.\n* Physically capable of participating in applicable assessments\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from this study:\n\n* Care Recipients (as defined in this protocol)\n* Staff of NHGRI\n\nStaff of NHGRI are unable to participate in this study as a safeguard against the risk of ethical concerns. As per OHSRP SOP 404, NIH staff may be a vulnerable class of study subjects. Excluding staff of the Institute conducting the study assures there will not be any perceived or actual conflict of interest, pressure\u002Fcoercion to participate among co-workers, subordinates, work unit-members, etc. As further noted in OHSRP SOP 404, exclusion further protects this class of subjects privacy and confidentiality; and protects the study s scientific integrity.\n\nPersons with impaired neuro-sensory or decision-making ability (adults unable to provide consent) will not be enrolled in the study. Persons with impaired neuro-sensory or decision making ability would not be able to participate with independent responses to the various social behavioral measures we use in the study interview and survey. Learning information about these individuals through other people instead of themselves would introduce bias to this study.",true,"ALL","18 Years","100 Years",{"count":21,"type":22},2800,"ESTIMATED","OBSERVATIONAL","Background:\n\nIn the U.S., about 53 million informal, unpaid caregivers provide care to a person who is ill, is disabled, or has age-related loss of function. These caregivers may be adult children, spouses, parents, or others. The stress of providing long-term care affects caregivers health and well-being. Researchers want to learn more about this stress and its effects.\n\nObjective:\n\nTo learn how the caregiving process affects the health and well-being of caregivers over time.\n\nEligibility:\n\nAdults aged 18 years and older who are caregivers for a person with a chronic medical condition and who have already given consent to take part in other study activities.\n\nDesign:\n\nParticipants will be put in different groups. They will complete some or all of the following tasks over 1 year. They may repeat these tasks once a year for up to 5 years.\n\nParticipants will fill out 2 online surveys. One will ask about their health and their caregiving experience. The other will ask them to list people in their social network and their care recipient s social network who give them support.\n\nParticipants will have a 2-part phone interview. It will be audio recorded. In part 1, they will be asked about the people they listed in the survey. In part 2, they will be asked about their caregiving experience and events in the care recipient s life.\n\nParticipants may fill out a weeklong diary every 3 months. It will ask about their daily social activities, well-being, and stress levels. It will also ask about their thoughts and feelings about caregiving.\n\nParticipants may give a blood sample each year they are in the study.\n\n...",[26,27,28,29,30],"Inherited Metabolic Disorders","Undiagnosed Diseases","Batten's Disease","Tay Sachs","Diabetes",[32,33,34,35,36],"Social Support","Rare Diseases","Natural History","Genetic Conditions","Family Network","RECRUITING","2026-06-25",{"date":40,"type":41},"2026-06-26","ACTUAL",{"date":43,"type":41},"2022-09-08",{"date":45,"type":22},"2030-12-31",{"name":47,"class":48},"National Human Genome Research Institute (NHGRI)","NIH",1,{"id":51,"slug":52,"hasResults":11,"nctId":53,"briefTitle":54,"officialTitle":55,"acronym":4,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":17,"minAge":57,"maxAge":4,"enrollmentInfo":58,"targetDuration":4,"studyType":60,"phases":61,"briefSummary":64,"conditions":65,"keywords":70,"overallStatus":74,"whyStopped":4,"lastUpdateSubmitDate":75,"lastUpdatePostDateStruct":76,"startDateStruct":78,"completionDateStruct":80,"leadSponsor":82,"locationsCount":49},"100640347","phase-1-gene-therapy-trial-for-cln6-batten-disease-100640347","NCT07582484","Gene Therapy Trial for CLN6 Batten Disease","Phase 1\u002F2b Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease), Delivering the CLN6 Gene by Self-Complementary AAV9","Inclusion Criteria:\n\n* Diagnosis of CLN6\n* At least 4 months old\n\nExclusion Criteria:\n\n* Presence of another inherited neurologic disease\n* Prior stem cell transplantation\n* Prior gene transfer, gene editing, or viral vector therapy","4 Months",{"count":59,"type":22},12,"INTERVENTIONAL",[62,63],"PHASE1","PHASE2","The goal of this clinical trial is to learn if a gene therapy called scAAV9.CB.CLN6 can treat children with CLN6 Batten disease (variant late infantile neuronal ceroid lipofuscinosis). The main questions it aims to answer are if he gene therapy safe and well tolerated, and if the gene therapy help slow disease progression or improve symptoms.\n\nParticipants will:\n\nReceive a single dose of the gene therapy through an injection into the fluid around the spinal cord (intrathecal administration) Have regular study visits over 2 years for safety checks and assessments of disease progression Be followed for an additional 3 years in a long-term follow-up study",[66,67,28,68,69],"CLN6","Batten Disease","Neuronal Ceroid Lipofuscinosis CLN6","Neuronal Ceroid Lipofuscinosis",[66,71,72,73,69],"CLN6 Batten","Batten","Batten's","NOT_YET_RECRUITING","2026-05-12",{"date":77,"type":41},"2026-05-14",{"date":79,"type":22},"2026-08",{"date":81,"type":22},"2028-08",{"name":83,"class":84},"The Charlotte and Gwenyth Gray Foundation","OTHER"]