[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07293546":3,"trial-entities:NCT07293546":80,"trial-summary:NCT07293546":85},{"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":45,"sex":46,"minimum_age":47,"maximum_age":48,"healthy_volunteers":49,"eligibility_criteria":50,"std_ages":63,"locations":66,"central_contacts":76,"overall_officials":77,"references":78,"see_also_links":79},"NCT07293546","FRF-001-CL101","Phase 1\u002F2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)","An Open-Label First-in-Human Phase 1\u002F2 Study to Evaluate the Safety, Tolerability, and Efficacy of FRF-001, an AAV-9 Gene Therapy Administered by Intracerebroventricular Injection in Participants With FOXG1 Syndrome","ENROLLING_BY_INVITATION","2029-12","2026-07","2026-07-24","2026-07-20","FOXG1 Research Foundation","OTHER",true,"The goal of this clinical trial is to learn if FRF-001 is a safe, tolerable, and efficacious treatment for children and adults with FOXG1 syndrome.",null,[19],"FOXG1 Syndrome",[],"INTERVENTIONAL","TREATMENT",[24,25],"PHASE1","PHASE2",{"count":27,"type":28},12,"ESTIMATED",[30],{"type":31,"name":32,"description":33,"armGroupLabels":34},"GENETIC","FRF-001","AAV-9 gene therapy delivered by intracerebroventricular injection",[35],"Treatment",[37,41],{"measure":38,"description":39,"timeFrame":40},"To evaluate the safety and tolerability of single-dose FRF-001 in participants with FOXG1 syndrome","Incidence, severity, and causality of treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), and adverse events of special interest (AESIs)","Through Week 104",{"measure":42,"description":43,"timeFrame":44},"To evaluate the efficacy of single-dose FRF-001 in participants with FOXG1 syndrome","Attainment of motor milestones, as assessed by the Peabody Developmental Motor Scales - Third Edition (PDMS-3)","Week 52 and Week 104",[],"ALL","2 Years","20 Years",false,{"inclusion":51,"exclusion":55,"raw_text":62},[52,53,54],"Participant must have a FOXG1 mutation confirmed as likely pathogenic or pathogenic by whole exome sequencing, whole genome sequencing, gene panel, single gene testing, or microarray performed at an accredited lab, with clinical phenotype consistent with FS in the opinion of the investigator.","The participant, or the participant's parent or legal guardian, is registered at the time of signing the informed consent in the FRF Citizen Natural History Study.","The participant, or the participant's parent, legal guardian, or caregiver are willing and able to complete all aspects of the study, adhere to the study visit schedule, and comply with all assessments.",[56,57,58,59,60,61],"Another genetic mutation or clinical comorbidity which could potentially confound the typical FOXG1 syndrome phenotype; FOXG1 gene duplication; or FOXG1 gene deletions that include regions outside of the FOXG1 coding region.","Prior treatment with a gene, cell therapy, or investigational treatment for FS.","Concurrent enrollment in another clinical study unless it is observational (noninterventional) and the study that does not interfere with the requirements of the current protocol and does not have the potential to impact the evaluation of safety or efficacy of FRF-001.","Any current or prior condition or contraindication that would render the participant unable to safely receive prophylactic corticosteroids, as assessed and determined by the Investigator.","Contraindications to or unwilling to undergo MRI or lumbar puncture (LP) procedures.","Any medical condition, comorbidity, or anatomical abnormality that, in the opinion of the Investigator and\u002For the attending anesthesiologist, would contraindicate the safe administration of sedation or general anesthesia required for study procedures.","Inclusion Criteria:\n\n* Participant must have a FOXG1 mutation confirmed as likely pathogenic or pathogenic by whole exome sequencing, whole genome sequencing, gene panel, single gene testing, or microarray performed at an accredited lab, with clinical phenotype consistent with FS in the opinion of the investigator.\n* The participant, or the participant's parent or legal guardian, is registered at the time of signing the informed consent in the FRF Citizen Natural History Study.\n* The participant, or the participant's parent, legal guardian, or caregiver are willing and able to complete all aspects of the study, adhere to the study visit schedule, and comply with all assessments.\n\nExclusion Criteria:\n\n* Another genetic mutation or clinical comorbidity which could potentially confound the typical FOXG1 syndrome phenotype; FOXG1 gene duplication; or FOXG1 gene deletions that include regions outside of the FOXG1 coding region.\n* Prior treatment with a gene, cell therapy, or investigational treatment for FS.\n* Concurrent enrollment in another clinical study unless it is observational (noninterventional) and the study that does not interfere with the requirements of the current protocol and does not have the potential to impact the evaluation of safety or efficacy of FRF-001.\n* Any current or prior condition or contraindication that would render the participant unable to safely receive prophylactic corticosteroids, as assessed and determined by the Investigator.\n* Contraindications to or unwilling to undergo MRI or lumbar puncture (LP) procedures.\n* Any medical condition, comorbidity, or anatomical abnormality that, in the opinion of the Investigator and\u002For the attending anesthesiologist, would contraindicate the safe administration of sedation or general anesthesia required for study procedures.",[64,65],"CHILD","ADULT",[67],{"facility":68,"city":69,"state":70,"zip":71,"country":72,"geoPoint":73},"Texas Children's Hospital (Baylor College of Medicine)","Houston","Texas","77030","United States",{"lat":74,"lon":75},29.76328,-95.36327,[],[],[],[],{"nct_id":4,"conditions":81,"biomarkers":83},[82],"Rett Syndrome, Congenital Variant",[84],"Forkhead Box Protein G1",{"nct_id":4,"found":15,"summary":86,"prompt_version":96},{"design":87,"status":88,"heading":89,"summary":90,"follow_up":91,"word_count":92,"commitments":93,"compensation":94,"drugs_mentioned":95},"This is a Phase 1\u002F2 study, meaning it looks at both safety and early effectiveness. It plans to enroll 12 participants.","completed","FRF-001 Gene Therapy for FOXG1 Syndrome","This study is testing a new gene therapy called FRF-001 for people with FOXG1 syndrome. FRF-001 is given through an injection into the fluid around the brain (intracerebroventricular injection). The main goals are to see if FRF-001 is safe and well-tolerated, and if it can help improve symptoms of FOXG1 syndrome. To join, you must be between 2 and 20 years old and have a confirmed FOXG1 genetic change. The study plans to enroll 12 participants. We don't know the current recruitment status from this information.","Participants will be followed for safety for up to 104 weeks (about 2 years) and for efficacy for 52 and 104 weeks.",86,"Not specified in the trial record.","Not stated in the trial record.",[32],"v2"]