NCT07293546Enrolling By Invitation1 期
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 Foundation1 个研究点 分布在 1 个国家目标入组 12 人开始时间: 2026年7月20日最近更新:
干预措施
试验速览
- 阶段
- 1 期
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- To evaluate the safety and tolerability of single-dose FRF-001 in participants with FOXG1 syndrome
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Treatment
Experimental
干预措施: FRF-001 (Genetic)
结局指标
主要结局
To evaluate the safety and tolerability of single-dose FRF-001 in participants with FOXG1 syndrome
时间窗: 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
时间窗: Week 52 and Week 104
Attainment of motor milestones, as assessed by the Peabody Developmental Motor Scales - Third Edition (PDMS-3)
次要结局
未报告次要终点
研究者
研究点 (1)
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