[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05754450":3,"trial-entities:NCT05754450":95,"trial-summary:NCT05754450":99},{"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":25,"interventions":28,"primary_outcomes":34,"secondary_outcomes":38,"sex":53,"minimum_age":54,"maximum_age":55,"healthy_volunteers":15,"eligibility_criteria":56,"std_ages":66,"locations":70,"central_contacts":84,"overall_officials":89,"references":93,"see_also_links":94},"NCT05754450","AVTX-803-LAD-302","An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II","A Phase 3, Open-Label, Extension Study to Assess the Long-term Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II)","RECRUITING","2026-12-31","2026-04","2026-05-01","2023-04-10","AUG Therapeutics","INDUSTRY",false,"The primary objective of this extension study is to assess the long-term safety and efficacy of AVTX-803 in subjects with LAD II (SLC35C1-CDG).",null,[19],"Leukocyte Adhesion Deficiency",[],"INTERVENTIONAL","TREATMENT",[24],"PHASE3",{"count":26,"type":27},2,"ESTIMATED",[29],{"type":30,"name":31,"description":32,"armGroupLabels":33},"DRUG","AVTX-803","L-fucose crystalline powder",[31],[35],{"measure":36,"timeFrame":37},"Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.","Through study completion, an average of 1 year.",[39,42,45,47,50],{"measure":40,"timeFrame":41},"Sialyl-Lewis X antigen expression on leukocytes","At 6 months",{"measure":43,"description":44,"timeFrame":37},"Change from Baseline in the composite Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)","The NPCRS comprises 3 sections, with each section containing specific items relevant to the assessment of subjects with CDG. Section I (Current Function); Section II (System Specific Involvement); and Section III (Current Clinical Assessment). The sum of the scores is used to scale patients into 3 domains: mild (0-14), moderate (15-25), and severe (\\>26) category.",{"measure":46,"description":44,"timeFrame":37},"Change from Baseline in the individual parameters of the Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)",{"measure":48,"description":49,"timeFrame":37},"Goal Attainment Score (GAS)","The parent (or subject) formulates 3 individual goals for improving quality of life. Typically, these goals affect mobility, independence, and disease-related health aspects. These goals will be re-evaluated and scored by the parent or subject at each study visit within a scale of -2 to +2.",{"measure":51,"description":52,"timeFrame":37},"Clinician Global Impression of Severity (CGI-S)","The CGI-S scale is a 7-point scale that assesses the severity of the subject's severity of disease\u002Fillness. 1=Normal, not ill at all; 2=borderline ill; 3=mildly ill; 4=moderately ill; 5=markedly ill; 6= severely ill; 7=among the most extreme ill.\"","ALL","6 Months","75 Years",{"inclusion":57,"exclusion":60,"raw_text":65},[58,59],"Subject must have completed protocol AVTX-803-LAD-301","Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)",[61,62,63,64],"Subject has severe anemia defined as hemoglobin \\\u003C8.0 g\u002FdL (\\\u003C4.9 mmol\u002FL)","Subject has impaired renal function as defined by an eGFR \\\u003C90 mL\u002Fmin","Subject has known or suspected intolerance or hypersensitivity to fucose or any ingredients of the investigational product","In the investigator's opinion, subject has a history of failure to respond to fucose at adequate dosing","Inclusion Criteria:\n\n* Subject must have completed protocol AVTX-803-LAD-301\n* Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)\n\nExclusion Criteria:\n\n* Subject has severe anemia defined as hemoglobin \\\u003C8.0 g\u002FdL (\\\u003C4.9 mmol\u002FL)\n* Subject has impaired renal function as defined by an eGFR \\\u003C90 mL\u002Fmin\n* Subject has known or suspected intolerance or hypersensitivity to fucose or any ingredients of the investigational product\n* In the investigator's opinion, subject has a history of failure to respond to fucose at adequate dosing",[67,68,69],"CHILD","ADULT","OLDER_ADULT",[71],{"facility":72,"status":8,"city":73,"state":74,"zip":75,"country":76,"contacts":77,"geoPoint":81},"Mayo Clinic","Rochester","Minnesota","55905","United States",[78],{"name":79,"role":80},"Queenie Tan, MD PhD","CONTACT",{"lat":82,"lon":83},44.02163,-92.4699,[85],{"name":86,"role":80,"phone":87,"email":88},"Jennifer Lin","3052833827","huiyil@Augtx.com",[90],{"name":91,"affiliation":72,"role":92},"David Deyle","PRINCIPAL_INVESTIGATOR",[],[],{"nct_id":4,"conditions":96,"biomarkers":97},[19],[98],"SLC35C1 Gene",{"nct_id":4,"found":100,"summary":101,"prompt_version":111},true,{"design":102,"status":103,"heading":104,"summary":105,"follow_up":106,"word_count":107,"commitments":108,"compensation":109,"drugs_mentioned":110},"This is an interventional study, meaning participants will receive a specific treatment. It is an extension study, suggesting it follows a previous trial, and plans to enroll 2 participants.","completed","Extension Study of AVTX-803 for Leukocyte Adhesion Deficiency Type II","This study is looking at the long-term safety and effectiveness of AVTX-803, a crystalline powder form of L-fucose, for people with Leukocyte Adhesion Deficiency Type II (LAD II), also known as SLC35C1-CDG. To join, you must have already completed a previous study (AVTX-803-LAD-301) and have a confirmed diagnosis of LAD II. The study will measure side effects related to AVTX-803 over about one year to see how safe it is. We don't have information on the current recruitment status, but the study plans to enroll 2 participants between 6 months and 75 years old.","Participants will be followed for about one year to track any side effects.",94,"Not specified in the trial record.","Not stated in the trial record.",[31],"v2"]