[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07721025":3,"trial-entities:NCT07721025":152,"trial-summary:NCT07721025":157},{"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":21,"study_type":42,"primary_purpose":43,"phases":44,"enrollment_info":46,"interventions":49,"primary_outcomes":59,"secondary_outcomes":63,"sex":101,"minimum_age":102,"maximum_age":17,"healthy_volunteers":103,"eligibility_criteria":104,"std_ages":121,"locations":125,"central_contacts":141,"overall_officials":146,"references":150,"see_also_links":151},"NCT07721025","LX2006-03","Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy","A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)","RECRUITING","2032-06","2026-07","2026-07-22","2026-06-25","Lexeo Therapeutics","INDUSTRY",true,"The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).",null,[19,20],"Friedreich Ataxia","Cardiomyopathy, Secondary",[22,23,19,24,25,26,27,28,29,30,31,32,33,34,35,36,37,38,39,40,41],"Friedreich Ataxia Cardiomyopathy","FA-CM","Friedreich's Ataxia","FA","FRDA","Cardiomyopathy","Genetic Cardiomyopathy","Mitochondrial Cardiomyopathy","Cardiac Disease","Left Ventricular Hypertrophy","Hypertrophic Cardiomyopathy (Non-Obstructive)","HCM","Gene Therapy","FXN Gene","FXN","Frataxin Gene","LX2006","SUNRISE-FA 2","SUNRISE-FA","CLARITY","INTERVENTIONAL","TREATMENT",[45],"PHASE2",{"count":47,"type":48},26,"ESTIMATED",[50,54],{"type":51,"name":38,"description":52,"armGroupLabels":53},"GENETIC","Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)",[38],{"type":55,"name":56,"description":57,"armGroupLabels":58},"OTHER","Usual Care","Cohort 1: Participants ≥16 years of age with FA-CM Participants will receive usual care for 26 weeks before receiving treatment with LX2006 (single crossover).",[56],[60],{"measure":61,"timeFrame":62},"Percent change from baseline in left ventricular mass index by cardiac MRI","At Week 26",[64,67,69,72,76,79,82,84,86,88,90,92,94,96,98],{"measure":65,"timeFrame":66},"Change from baseline in high-sensitivity troponin I","At Week 26 and through Month 60",{"measure":68,"timeFrame":66},"Change from baseline in left ventricular wall thickness and additional imaging outcomes",{"measure":70,"description":71,"timeFrame":66},"Change from baseline in modified Friedreich's Ataxia Rating Scale","A neurological assessment tool for Friedreich ataxia that is measured on a scale of 0 to 93 with high scores indicating greater physical impairment",{"measure":73,"description":74,"timeFrame":75},"Change from baseline in Kansas City Cardiomyopathy Questionnaire","A cardiac assessment tool scored from 0 to 100, with higher scores indicating better health status and lower scores reflecting more severe heart failure symptoms and functional limitations","Through Month 60",{"measure":77,"description":78,"timeFrame":75},"Healthcare resource use","Time from baseline to first occurrence of any of the following as captured via electronic data capture system:\n\n* Death due to any cause, and\n* Any of the following due to cardiovascular events:\n\n  * Death\n  * Hospitalization for heart failure\n  * Non-fatal stroke\n  * Non-fatal heart attack\n  * Non-fatal life-threatening rapid or irregular heartbeat\n  * Heart transplant\n  * Insertion of a left ventricular assist device (pump) in the heart",{"measure":80,"description":81,"timeFrame":75},"Cardiovascular events","Number of the following cardiovascular events as collected from the patient by the study doctor during scheduled study visits:\n\n* Hospitalization for heart failure\n* Non-fatal stroke\n* Non-fatal heart attack\n* Non-fatal life-threatening rapid or irregular heartbeat\n* Heart transplant\n* Insertion of a left ventricular assist device (pump) in the heart",{"measure":83,"timeFrame":75},"Patient Global Impressions scales",{"measure":85,"timeFrame":75},"Incidence and severity of treatment-emergent adverse events",{"measure":87,"timeFrame":75},"Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges",{"measure":89,"timeFrame":75},"Change in clinical vital signs (body temperature [°C])",{"measure":91,"timeFrame":75},"Change in clinical vital signs (systolic and diastolic blood pressure [mmHg])",{"measure":93,"timeFrame":75},"Change in clinical vital signs (heart rate [beats per minute])",{"measure":95,"timeFrame":75},"Change in clinical vital signs (respiratory rate [breaths per minute])",{"measure":97,"timeFrame":75},"Change in clinical vital signs (oxygen saturation [%])",{"measure":99,"description":100,"timeFrame":75},"Change in clinical 12-lead ECG findings","ECG machine automatically calculates the heart rate \\[beats per minute\\] and measures PR, QT, QT interval corrected using Fridericia's method intervals, and QRS complex \\[milliseconds\\]","ALL","6 Years",false,{"inclusion":105,"exclusion":112,"raw_text":120},[106,107,108,109,110,111],"Male or female, age at least 6 years at the time of signing the informed consent (and assent, if applicable).","Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on the frataxin gene)","Onset of FA on or before 25 years of age","Confirmed left ventricular hypertrophy and abnormal left ventricular mass index","Left ventricular ejection fraction at least 30%","Anti-AAVrh.10 total antibody titer less than the protocol-specified maximum level",[113,114,115,116,117,118,119],"Presence of other forms of cardiomyopathy that contribute to heart failure","Current use of inotrope infusion or presence of a ventricular assist device","Contraindication to cardiac MRI","Prior organ transplant","Previous gene transfer or cell therapy","Poorly controlled diabetes (hemoglobin A1c ≥8%)","Active hematologic or solid organ cancer","Inclusion Criteria:\n\n* Male or female, age at least 6 years at the time of signing the informed consent (and assent, if applicable).\n* Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on the frataxin gene)\n* Onset of FA on or before 25 years of age\n* Confirmed left ventricular hypertrophy and abnormal left ventricular mass index\n* Left ventricular ejection fraction at least 30%\n* Anti-AAVrh.10 total antibody titer less than the protocol-specified maximum level\n\nExclusion Criteria:\n\n* Presence of other forms of cardiomyopathy that contribute to heart failure\n* Current use of inotrope infusion or presence of a ventricular assist device\n* Contraindication to cardiac MRI\n* Prior organ transplant\n* Previous gene transfer or cell therapy\n* Poorly controlled diabetes (hemoglobin A1c ≥8%)\n* Active hematologic or solid organ cancer\n\nOther inclusion\u002Fexclusion criteria to be applied as per protocol.",[122,123,124],"CHILD","ADULT","OLDER_ADULT",[126],{"facility":127,"status":8,"city":128,"state":129,"zip":130,"country":131,"contacts":132,"geoPoint":138},"University of South Florida","Tampa","Florida","33612","United States",[133],{"name":134,"role":135,"phone":136,"email":137},"Lucretia Campbell","CONTACT","813-974-5633","lcampbel@usf.edu",{"lat":139,"lon":140},27.94752,-82.45843,[142],{"name":143,"role":135,"phone":144,"email":145},"Lexeo Clinical Trials","212-547-9879","clinicaltrials@lexeotx.com",[147],{"name":143,"affiliation":148,"role":149},"Lexeo Therapeutics, Inc.","STUDY_DIRECTOR",[],[],{"nct_id":4,"conditions":153,"biomarkers":155},[154],"Cardiomyopathy in Friedreich's ataxia",[156,35],"Anti-AAVrh.10 antibody",{"nct_id":4,"found":15,"summary":158,"prompt_version":167},{"design":159,"status":160,"heading":6,"summary":161,"follow_up":162,"word_count":163,"commitments":164,"compensation":165,"drugs_mentioned":166},"This is a Phase 2, open-label, randomized, controlled study, meaning some participants will receive usual care before LX2006. It plans to enroll 26 participants.","completed","This study is testing a gene therapy called LX2006 for people with Friedreich ataxia (FA) cardiomyopathy (CM), a heart condition. LX2006 works by delivering a healthy copy of the FXN gene. The study aims to see how well LX2006 works and if it is safe. You might be able to join if you are at least 6 years old, have a confirmed diagnosis of FA with a specific genetic change (GAA expansion), and have heart problems like left ventricular hypertrophy and an abnormal left ventricular mass index. The main way researchers will measure success is by looking at changes in your heart muscle size (left ventricular mass index) after 26 weeks. The current status of this study is unclear.","The primary endpoint is measured at Week 26, suggesting follow-up for at least that duration.",119,"Participants in Cohort 1 will receive usual care for 26 weeks before getting a single treatment with LX2006. The primary endpoint is measured at Week 26.","Not stated in the trial record.",[38],"v2"]