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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.

Study design
This is a Phase 1/2 study, meaning it looks at both safety and early effectiveness. It plans to enroll 12 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for safety for up to 104 weeks (about 2 years) and for efficacy for 52 and 104 weeks.

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NCT07293546

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

Enrolling by Invitation
PHASE1Ages 2–20InterventionalTreatment
FOXG1 Research Foundation
~12 participants
Updated 2026-07-24 on ClinicalTrials.gov
What's tested:FRF-001

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To evaluate the safety and tolerability of single-dose FRF-001 in participants with FOXG1 syndrome
Measured over Through Week 104
+1 more outcome measured

Conditions

FOXG1 Syndrome

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Rett Syndrome, Congenital VariantForkhead Box Protein G1

Where it's being run

1 sites across 1 states
Texas1

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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.

What this trial measures

  • To evaluate the safety and tolerability of single-dose FRF-001 in participants with FOXG1 syndromeThrough Week 104

    Incidence, severity, and causality of treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), and adverse events of special interest (AESIs)

  • To evaluate the efficacy of single-dose FRF-001 in participants with FOXG1 syndromeWeek 52 and Week 104

    Attainment of motor milestones, as assessed by the Peabody Developmental Motor Scales - Third Edition (PDMS-3)