FOXG1 SyndromePhase 1/2NCT07293546
Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)
A Phase 1/2 study testing FRF-001 in people with FOXG1 Syndrome, run by FOXG1 Research Foundation at 2 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 20 years
- Conditions the study is for
- FOXG1 Syndrome
- What is being tested?
- FRF-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- 20 Jul 2026to Dec 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites
Texas Children's Hospital (Baylor College of Medicine)
Houston, Texas, 77030
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
About this study
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
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.
- Conditions
- FOXG1 Syndrome
- Being tested
- FRF-001
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FRF-001Genetic
AAV-9 gene therapy delivered by intracerebroventricular injection
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Not described Receives: Genetic: FRF-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: Through Week 104
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)
Measured: Week 52 and Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2025
- Study start20 Jul 2026
- Primary completionJul 2029 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for FOXG1 SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- FOXG1 Research Foundation
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07293546
- Sponsor's protocol ID
- FRF-001-CL101
- Status last verified
- Oct 2026
- First submitted
- 16 Dec 2025
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07293546
- Acronym / sponsor ID
- FRF-001-CL101
- Conditions
- FOXG1 Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- FOXG1 Research FoundationUniversity, hospital or other
- Study start
- 20 Jul 2026 (actual)
- Primary completion
- Jul 2029 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 19 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Genetic: FRF-001 |
Who may qualify
- Ages
- 2 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FRF-001AAV-9 gene therapy delivered by intracerebroventricular injection | Genetic | Treatment |
Primary outcome
To evaluate the safety and tolerability of single-dose FRF-001 in participants with FOXG1 syndrome
Time frame: Through 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 syndrome
Time frame: Week 52 and Week 104
Attainment of motor milestones, as assessed by the Peabody Developmental Motor Scales - Third Edition (PDMS-3)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2026.
- Individual participant dataSharing planned: Undecided
Related trials for FOXG1 Syndrome
Enrolling by invitation
Palliative Care Needs of Children With Rare Diseases and Their Families
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing FRF-001 in people with FOXG1 Syndrome, run by FOXG1 Research Foundation at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of single-dose FRF-001 in participants with FOXG1 syndrome (time frame: Through Week 104). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Oct 2026; AzTrials last refreshed its copy on 6 Nov 2026.