NCT07318909尚未招募1 期
A Clinical Exploration Study of Adeno-Associated Virus Vector Expressing Human Phenylalanine Hydroxylase Gene in Adult Classical Phenylketonuria
Gritgen Therapeutics Co., Ltd.1 个研究点 分布在 1 个国家目标入组 9 人开始时间: 2027年12月31日最近更新:
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Incidence of adverse event (AE), including serious adverse event (SAE) and adverse event of special interest (AESI)
研究概览
简要总结
This study is a single-arm, open-label, dose-escalation, exploratory study to evaluate the safety, tolerability, and efficacy of a single administration of GS1168 Injection in Chinese adult phenylketonuria (PKU) with PAH mutation.
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
- 18 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Understand the purpose and risks of the study and voluntarily sign Informed Consent Forms (ICF).
- •Male or female, aged 18-55 years.
- •Diagnosis of PKU due to PAH mutation, and be inadequately controlled with current therapy (e.g., dietary management) in the judgement of the investigator.
- •Intolerant of unresponsive to available treatment such as Kuvan or Palynziq, and have come off the medication for at least 28 days prior to signing the ICF.
- •Ability and willingness to maintain dietary protein intake consistent with baseline
- •Male and female subjects of childbearing potential must agree to use effective contraception methods from signing ICF until at least 52 weeks after GS1168 infusion. Female subjects must not be breastfeeding.
- •Agree to avoid alcohol consumption from 30 days prior to GS1168 infusion until at least 52 weeks after infusion.
- •Ability to communicate well with the Investigator and to understand and comply with the requirements of the study.
排除标准
- •Diagnosis of PKU due to other gene mutations.
- •Body weight ≥100 kg or Body Mass Index (BMI) ≥30 kg/m² at screening.
- •Presence of anti-AAV8 neutralizing antibody.
- •Abnormal liver function tests at screening:
- •ALT or AST >1.25 × Upper Limit of Normal (ULN)
- •GGT >1.25 × ULN
- •Total Bilirubin >1.5 × ULN
- •Abnormal laboratory findings at screening:
- •Hemoglobin <9 g/dL
- •Neutrophil count <1.0 × 10⁹/L
- •Platelet count <100 × 10⁹/L
- •Serum Creatinine ≥1.5 mg/dL (133 μmol/L)
- •Glycated Hemoglobin (HbA1c) >6% or Fasting Blood Glucose >6.1 mmol/L
- •Active or occult hepatitis B virus (HBV) infection, active hepatitis C virus (HCV) infection, positive Human Immunodeficiency Virus (HIV) antibodies and positive syphilis test.
- •Any severe conditions of respiratory, cardiovascular, gastrointestinal, renal, neurological, endocrine, immunological, hematological, psychiatric, etc.
- •History of malignancy.
- •Any other condition that, in the opinion of the Investigator, may affect subject compliance or render the subject unsuitable for participation in this trial.
研究组 & 干预措施
Treatment group Arm of GS1168
Experimental
Dose level 1, level 2 and level 3 will be administered
干预措施: GS1168 injection (Drug)
结局指标
主要结局
Incidence of adverse event (AE), including serious adverse event (SAE) and adverse event of special interest (AESI)
时间窗: Week 52
Incidence of adverse event (AE), including serious adverse event (SAE) and adverse event of special interest (AESI) as assessed by CTCAE V5.0, from baseline to week 52
次要结局
- Phenylketonuria Quality of Life (PKU-QOL) scores(Week 52)
- Plasma Phe levels(Week 52)
- Total protein intake(Week 52)
研究者
研究点 (1)
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