FRF's ultimate goal is to develop a safe and effective FDA-approved treatment for FOXG1 syndrome, which can be available to the broader FOXG1 patient community as quickly as possible. A clinical trial is an essential step on the way to achieving our goal, rather than being the goal itself.

We recognize that the trial size, which will be optimized for testing the safety and potential benefit of the treatment and enabling the fastest possible approval path if proven successful, is unlikely to accommodate the number of families interested in participating. We are determined to do everything we can to bring a treatment across the approval finish line so that it is available to the FOXG1 community as early as possible.

The gene therapy will be delivered via an Intracerebroventricular (ICV) injection. This is an injection into a space within the brain, through a surgical procedure.

Many parameters influence the design of clinical trials, including their size (number of patients). There are multiple criteria for participation in clinical trials, including clinical considerations and others, which are ultimately determined through a process of discussion with and approval by the FDA and other regulatory agencies. Enrollment is done via a randomized process, ensuring that we operate in full compliance with all rules and regulations. Enrollment in the clinical trial cannot be guaranteed.

Parents of patients enrolled in the FOXG1 Citizen Health platform will receive a survey as a first step to be pre-screened for trial eligibility.

Please make sure you are receiving emails from the FOXG1 Research Foundation.

And keep a look out for emails from Citizen Health with the survey.

The FRF-001 clinical trial is designed for 12-18 patients.

Age range is one of many elements of clinical trial design that has been discussed with the regulatory agencies. We seek to test the potential benefit of our gene therapy across a range of ages.

We will provide specific age requirements for FRF-001 shortly.

The number and location of clinical trial sites (including countries) will be determined based on various considerations, and we cannot guarantee that a site will be available in close proximity to every family interested in participating. At the same time, FRF will do its best to minimize the impact of geographical considerations on trial enrollment (while recognizing that regulatory or legal considerations could affect international participation).

Our gene therapy is designed to be potentially beneficial to the broad FOXG1 patient population that does not have two fully functional copies of the FOXG1 gene. We aim to open trial enrollment to as broad a spectrum of FOXG1 mutations as possible, pending safety considerations and approval by the regulatory agencies. Additional information will be provided when it becomes available.

The FOXG1 syndrome registry, Natural History Study, and other clinical research activities FRF will be conducting in 2025 (which do not involve treatment; these were presented at the 2024 parent conference) are essential for the design and execution of an optimal clinical trial. Participation in these activities does not guarantee enrollment in a future clinical trial; however, its contribution to the clinical trial cannot be overstated, and we deeply appreciate the community's continued active engagement and involvement.

No. There is no fee or payment associated with participation in the clinical trial, and it is not possible to “purchase a spot” in the trial. The cost of the trial will be covered by FRF through the generous donations we receive from supporters.

Participation in the clinical trial cannot be secured or guaranteed.

Clinical trial design and logistical considerations are informed, among other things, by the data we have from the FOXG1 syndrome patient registry and the FOXG1 Natural History Study (NHS).

It is a requirement to be enrolled in the Citizen Health platform to be screened for trial eligibility.

• Make sure your child's FOXG1 syndrome registry profile is up to date, with their genetics report uploaded: https://foxg1.beneufit.com/.

• Enroll / Update your Citizen Health FOXG1 NHS account with the latest medical records. If you are in the U.S., Citizen Health will access the records for you. Outside the U.S., you can upload any recent hospitalization visit records: https://www.citizen.health/partners/foxg1.

• Stay informed! If you are not receiving emails from the FRF, sign up here.

If you haven’t already, sign up for our newsletter and follow our social media communication channels. Any updates will be posted there. Sign Up Here. Please check your spam/junk folder for missed emails and add contact@foxg1research.org to your contacts, along with other FRF email addresses you receive.

As always, FRF will continue to share important updates with the FOXG1 community. In fact, the FRF will over-communicate to ensure you do not miss anything.

If you have additional questions related to the FOXG1 gene therapy program, please submit them through the form below for consideration to be answered on this page. We will do our best to address every inquiry.