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Enrolling by invitation Phase 1Phase 2 Interventional

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

ClinicalTrials.gov ID: NCT07293546

Public ClinicalTrials.gov record NCT07293546. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 26, 2026, 3:52 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

An Open-Label First-in-Human Phase 1/2 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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT07293546
Recruitment status
Enrolling by invitation
Study type
Interventional
Phase
Phase 1, Phase 2
Lead sponsor
FOXG1 Research Foundation
Other
Enrollment
12 participants

Conditions and interventions

Conditions

Interventions

Genetic

Eligibility (public fields only)

Age range
2 Years to 20 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Jul 20, 2026 (Actual)
Primary completion
Jul 2029 (Estimated)
Completion
Dec 2029 (Estimated)
Last update posted
Oct 13, 2026

2026 – 2029

United States locations

U.S. sites
2
U.S. states
2
U.S. cities
2
Facility City State ZIP Site status
UCSF Benioff Children's Hospital Oakland Oakland California 94609 —
Texas Children's Hospital (Baylor College of Medicine) Houston Texas 77030 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT07293546, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Oct 13, 2026 · Synced Oct 26, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT07293546 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →