NCT07135050招募中1 期
Phase 1/2 First-in-Human Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Mahzi Therapeutics5 个研究点 分布在 2 个国家目标入组 12 人开始时间: 2025年12月19日最近更新:
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 12
- 试验地点
- 5
- 主要终点
- To evaluate the safety and tolerability of MZ-1866 in participants with Pitt Hopkins Syndrome
研究概览
简要总结
The goal of this clinical trial is to learn if MZ-1866 is a safe and tolerable treatment for children and adults with Pitt Hopkins Syndrome.
To evaluate the safety of MZ-1866, the following will be evaluated:
- frequency and severity of adverse events
- physical exam, laboratory results and electrocardiogram findings
Participants will:
- receive a single dose of MZ-1866 by intracerebroventricular injection
- be seen by the study physician and site staff periodically to assess changes to their health status
- be periodically evaluated using neurodevelopmental tools
Caregivers will:
- be interviewed periodically about the health status and development of the participant
- keep diaries and complete periodic questionnaires regarding participant symptoms
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant has a TCF4 gene mutation confirmed as "pathogenic" or "likely pathogenic" by whole exome sequencing, whole genome sequencing, gene panel, single gene testing, or microarray, performed at an accredited laboratory
- •Clinical phenotype consistent with Pitt Hopkins Syndrome, in the opinion of the investigator
- •The participant, or the participant's parent or legal guardian, is willing to provide access to prior medical records for the collection of demographics and diagnostic and treatment history
排除标准
- •A deletion that includes the TCF4 gene that is over 12 Mbp in size
- •Another genetic mutation or clinical comorbidity not associated with Pitt Hopkins Syndrome that could potentially confound interpretation of the study data
- •A central nervous system structural or vascular abnormality that is a contraindication to the ICV administration procedure, including but not limited to: signs or symptoms of increased intracranial pressure, history of a space-occupying lesion, or presence of a ventricular shunt that would preclude ICV procedures or safety assessments, or increase risk to the participant
- •Not able to receive prophylactic corticosteroids due to a medical contraindication or participant has a history of a condition that could worsen with corticosteroid therapy as assessed and determined by the Investigator
- •Not able to undergo MRI procedures
- •Cannot be anesthetized for the ICV injection
研究组 & 干预措施
Cohort 1 (MZ-1866)
Experimental
Participants ages 12-25 years
干预措施: MZ-1866 (Genetic)
Cohort 2 (MZ-1866)
Experimental
Participants aged 2-11 years
干预措施: MZ-1866 (Genetic)
结局指标
主要结局
To evaluate the safety and tolerability of MZ-1866 in participants with Pitt Hopkins Syndrome
时间窗: Week 104
Frequency and severity of adverse events of special interest
次要结局
- To evaluate the safety and tolerability of MZ-1866 in participants with Pitt Hopkins Syndrome(Week 104)
- To further assess the safety and tolerability of MZ-1866 in participants with Pitt Hopkins Syndrome(Week 104)
研究者
研究点 (5)
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