Skip to main content
MedPathMedPath Home
Clinical Trials/NCT07293546
NCT07293546
Not yet recruiting
Phase 1

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

FOXG1 Research Foundation0 sites12 target enrollmentStarted: April 1, 2026Last updated:
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit

Overview

Phase
Phase 1
Status
Not yet recruiting
Sponsor
FOXG1 Research Foundation
Enrollment
12
Primary Endpoint
To evaluate the safety and tolerability of single-dose FRF-001 in participants with FOXG1 syndrome
OverviewStudy DesignEligibility CriteriaOutcomesInvestigatorsRecords & IdentifiersSimilar Trials

Overview

Brief 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.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
2 Years to 20 Years (Child, Adult)
Sex
All
Accepts Healthy Volunteers
No

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-
  • •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.

Outcomes

Primary Outcomes

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)

Secondary Outcomes

No secondary outcomes reported

Investigators

Sponsor
FOXG1 Research Foundation
Sponsor Class
Other
Responsible Party
Sponsor

Similar Trials

Active, not recruiting
Not Applicable
A Study to Learn if a Medicine That Reduces Stomach Acid Affects the Blood Level of Study Medicine PF-08049820 in Healthy Chinese Adults.
NCT07284173Pfizer18
Not yet recruiting
Not Applicable
A Study of DB-1324 in Advanced/Metastatic Gastrointestinal Tumors
NCT07263594DualityBio Inc.127
Recruiting
Early Phase 1
A Phase I Study to Evaluate the Safety, Tolerability, and PK of HLX37 in Advanced/Metastatic Solid Tumors
NCT07274813Shanghai Henlius Biotech254
Recruiting
Early Phase 1
Study of JANX011 in Healthy Adult Volunteers to Assess Safety and Tolerability.
NCT07291323Janux Therapeutics60
Recruiting
Phase 1
A Clinical Study of SHR-A1811(sc) Subcutaneous Injection in Patients With Solid Tumor
NCT07275242Suzhou Suncadia Biopharmaceuticals Co., Ltd.107