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Gene Therapy Trial Report

Summary

Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)


NCTID NCT07293546 (View at clinicaltrials.gov)
Description
Development Status πŸ”„ Active
Indication FOXG1 Syndrome
Disease Ontology Term πŸ”„ DOID:0070657
Drug Product Name πŸ”„ FRF-001
Sponsor FOXG1 Research Foundation
Funder Type Other
Recruitment Status πŸ”„
Enrolling by invitation
Enrollment Count 12 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant πŸ”„ FOXG1
Therapeutic Modality πŸ”„ Gene transfer
Therapy Route πŸ”„ in vivo
Mechanism of Action πŸ”„ Functional gene replacement
Route of Administration πŸ”„ Intraventricular
Drug Product Type πŸ”„ Viral vector
Gene Delivery System Type πŸ”„ Viral transduction
Vector Type πŸ”„ Adeno-associated virus
Viral Vector Subtype AAV9
Dose 1 πŸ”„ Undisclosed

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2025-12-16
Completion Date πŸ”„ 2029-12
Last Update πŸ”„ 2026-07-24

Participation Criteria


Eligible Age 2 Years - 20 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * 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. Exclusion Criteria: * 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/or the attending anesthesiologist, would contraindicate the safe administration of sedation or general anesthesia required for study procedures.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites πŸ”„ 1
Locations πŸ”„ United States

Regulatory Information


Has US IND True
FDA Designations πŸ”„ Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates IND was cleared in February 2026, trial is expected to start early 2026

Resources/Links