[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05868499":3,"trial-entities:NCT05868499":87,"trial-summary:NCT05868499":90},{"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":51,"sex":58,"minimum_age":59,"maximum_age":60,"healthy_volunteers":61,"eligibility_criteria":62,"std_ages":66,"locations":69,"central_contacts":79,"overall_officials":80,"references":85,"see_also_links":86},"NCT05868499","EXG-US-02","Rollover Study From EXG-US-01","Long Term Follow-up Study in Patients With Telomere Biology Disorders With Bone Marrow Failure Who Completed Study EXG-US-01","ENROLLING_BY_INVITATION","2027-01-30","2024-07","2024-07-12","2024-01-01","Elixirgen Therapeutics, Inc.","INDUSTRY",true,"This is a long-term rollover follow-up study for Phase I\u002FII study (Protocol EXG-US-01).","Patients who received EXG34217 treatment and had at least one follow-up visit (Month 1, 3, 6, or 12) in Study EXG-US-01 will be eligible for this rollover study. Patient will sign a consent form prior to any study related procedure. This study is to add additional follow-up assessments up to 6 years after EXG34217 treatment. This study does not have additional intervention. Additional visits will be every 6 months in the first 3 years and once a year for two years.",[19],"Telomere Biology Disorders With Bone Marrow Failure",[],"INTERVENTIONAL","SUPPORTIVE_CARE",[24],"PHASE1",{"count":26,"type":27},12,"ESTIMATED",[29],{"type":30,"name":31,"description":32,"armGroupLabels":33},"BIOLOGICAL","EXG34217","Single infusion",[31],[35,39,42,45,48],{"measure":36,"description":37,"timeFrame":38},"Number of participants with adverse events -Safety by Incidence of Treatment-Emergent","Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.","Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72",{"measure":40,"description":41,"timeFrame":38},"umber of participants with a change in in physical examination","Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)",{"measure":43,"description":44,"timeFrame":38},"Number of participants with a change in Electrocardiography (ECG)","ECG (standard digital 12-lead in singlicate)",{"measure":46,"description":47,"timeFrame":38},"Number of participants with a change in clinical laboratory evaluations","Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)",{"measure":49,"description":50,"timeFrame":38},"Number of participants with a change of Immunogenicity","Change in Antibody against virus vector and transgene",[52,55],{"measure":53,"description":54,"timeFrame":38},"Number of participants with a change in telomere length","Change in telomere length in any peripheral blood cells",{"measure":56,"description":57,"timeFrame":38},"Number of participants with improvement of blood counts.","Blood counts: neutrophils,platelets, or hemoglobin","ALL","18 Years",null,false,{"inclusion":63,"exclusion":64,"raw_text":65},[],[],"Inclusion Criteria:\n\n1. Signed an Institutional Review Board (IRB) approved informed consent document indicating that they understand the purpose of, and procedures required by the study and are willing to participate in the study and comply with all study procedures and restrictions. Informed consent must be obtained from the subject prior to initiating procedures.\n2. Have completed the 12-month visit of Study EXG-US-01.\n\nExclusion Criteria:\n\n\\-",[67,68],"ADULT","OLDER_ADULT",[70],{"facility":71,"city":72,"state":73,"zip":74,"country":75,"geoPoint":76},"Cincinnati Children's Hospital","Cincinnati","Ohio","45229","United States",{"lat":77,"lon":78},39.12711,-84.51439,[],[81],{"name":82,"affiliation":83,"role":84},"Kasiani Myers, MD","Cincinnati Children Hospital Medical Center","PRINCIPAL_INVESTIGATOR",[],[],{"nct_id":4,"conditions":88,"biomarkers":89},[19],[],{"nct_id":4,"found":61,"summary":60,"prompt_version":60}]