EUCTR2019-001878-28-GB进行中（未招募）1 期
A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 µmol/L - A Phase 1/2 study to evaluate the safety and efficacy of BMN 307
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 100
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. age 15 or 18 years and older at Screening
- •2. Diagnosis of PKU which is a condition characterized by PAH deficiency
- •3. Average of two plasma Phe levels > 600 µmol/L during the Screening period
- •4. Ability and willingness to maintain dietary protein intake consistent with baseline intake for the duration of the study unless otherwise directed.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 8
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 100
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •1. Subjects with primary BH4 deficiency or other forms of BH4 metabolism deficiency.
- •2. Clinically significant liver disease as assessed by ultrasound at Screening.
- •3. Prior treatment with gene therapy.
- •4. Detectable antibodies to the AAV5 capsid at Screening.
- •5. Contraindication to use of corticosteroids (CS) or history of condition that could worsen with CS therapy.
- •6. Hemoglobin A1C = 8.0% or glucose = 250 mg/dL at Screening.
研究者
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