[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07703462":3,"trial-entities:NCT07703462":88,"trial-summary:NCT07703462":93},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":21,"primary_purpose":22,"phases":23,"enrollment_info":26,"interventions":29,"primary_outcomes":36,"secondary_outcomes":57,"sex":58,"minimum_age":59,"maximum_age":59,"healthy_volunteers":60,"eligibility_criteria":61,"std_ages":70,"locations":74,"central_contacts":84,"overall_officials":85,"references":86,"see_also_links":87},"NCT07703462","NLF-TARDB-002-A","Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS","An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation","ENROLLING_BY_INVITATION","2028-12","2026-07","2026-07-14","2026-12","n-Lorem Foundation","OTHER",true,"This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.","This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP",[19],"Amyotrophic Lateral Sclerosis (ALS)",[],"INTERVENTIONAL","TREATMENT",[24,25],"PHASE1","PHASE2",{"count":27,"type":28},1,"ESTIMATED",[30],{"type":31,"name":32,"description":33,"armGroupLabels":34},"DRUG","nL-TARDB-002","Personalized antisense oligonucleotide",[35],"Open Label",[37,41,43,45,47,49,51,54],{"measure":38,"description":39,"timeFrame":40},"Clinical Functioning","Change from baseline at 12-months post nL-TARDB-002 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).","Baseline to 12 months",{"measure":38,"description":42,"timeFrame":40},"Change from baseline at 12-months post nL-TARDB-002 administration in Slow Vital Capacity (SVC)",{"measure":38,"description":44,"timeFrame":40},"Change from baseline at 12-months post nL-TARDB-002 administration in Handheld Dynamometry (HHD)",{"measure":38,"description":46,"timeFrame":40},"Change from baseline at 12-months post nL-TARDB-002 administration in scores on Edinburgh Cognitive and Behavioral ALS Screen (ECAS).",{"measure":38,"description":48,"timeFrame":40},"Change from baseline at 12-months post nL-TARDB-002 administration in scores on ALS Cognitive Behavioral Screen (ALS-CBS).",{"measure":38,"description":50,"timeFrame":40},"Change from baseline at 12-months post nL-TARDB-002 administration in scores on Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5).",{"measure":52,"description":53,"timeFrame":40},"Survival","Change from baseline at 12-months post nL-TARDB-002 administration in survival status",{"measure":55,"description":56,"timeFrame":40},"Disease Biomarkers","Change from baseline at 12-months post nL-TARDB-002 administration in serum\u002Fplasma and CSF neurofilament light chain levels",[],"ALL",null,false,{"inclusion":62,"exclusion":66,"raw_text":69},[63,64,65],"Informed consent\u002Fassent provided by the participant (when appropriate), and\u002For participants parent(s) or legally authorized representative(s)","Ability to travel to the study site and adhere to study-related follow-up examinations and\u002For procedures and provide access to participant's medical records","Genetically confirmed neurological disorder",[67,68],"Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures","Use of an investigational medication within less than 5 half-lives of the drug at enrollment","Inclusion Criteria:\n\n* Informed consent\u002Fassent provided by the participant (when appropriate), and\u002For participants parent(s) or legally authorized representative(s)\n* Ability to travel to the study site and adhere to study-related follow-up examinations and\u002For procedures and provide access to participant's medical records\n* Genetically confirmed neurological disorder\n\nExclusion Criteria:\n\n* Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures\n* Use of an investigational medication within less than 5 half-lives of the drug at enrollment",[71,72,73],"CHILD","ADULT","OLDER_ADULT",[75],{"facility":76,"city":77,"state":78,"zip":79,"country":80,"geoPoint":81},"Jefferson Health","Philadelphia","Pennsylvania","19107","United States",{"lat":82,"lon":83},39.95238,-75.16362,[],[],[],[],{"nct_id":4,"conditions":89,"biomarkers":91},[90],"Amyotrophic Lateral Sclerosis",[92],"TARDBP Gene",{"nct_id":4,"found":15,"summary":94,"prompt_version":104},{"design":95,"status":96,"heading":97,"summary":98,"follow_up":99,"word_count":100,"commitments":101,"compensation":102,"drugs_mentioned":103},"This is an interventional study designed for a single participant. It is not specified if it is randomized, blinded, or what phase it is in.","completed","Personalized nL-TARDB-002 for TARDBP ALS","This study is testing a personalized medicine called nL-TARDB-002 for a single participant with Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease. This specific type of ALS is caused by a change in the TARDBP gene. The goal is to see if nL-TARDB-002 is safe and how well it works to improve clinical functioning over 12 months. To join, you would need to have genetically confirmed ALS related to the TARDBP gene, provide informed consent, and be able to travel to the study site for follow-up appointments. The study is currently unclear on its recruitment status.","Your clinical functioning will be measured for up to 12 months after starting treatment.",98,"You would need to travel to the study site for follow-up examinations and procedures, and allow access to your medical records.","Not stated in the trial record.",[32],"v2"]