NCT04480567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
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 100
- 试验地点
- 3
- 主要终点
- Change from baseline in mean Plasma Phe levels
研究概览
简要总结
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
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
- 15 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency
- •Ability and willingness to maintain dietary protein intake consistent with baseline intake
- •Willingness to abstain from hepatotoxic substances post-BMN 307 administration
- •Willingness and capable per investigator opinion to comply with study procedures and requirements
- •Willingness to use effective methods of contraception
- •Plasma Phe levels > 600 µmol/L
排除标准
- •Subjects with primary BH4 deficiency or other forms of BH4 metabolism deficiency
- •Clinically significant liver dysfunction or disease
- •Prior treatment with gene therapy
- •Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study
- •History of malignancy
研究组 & 干预措施
Dose 1 of BMN 307
Experimental
干预措施: BMN 307 (Drug)
Dose 2 of BMN 307
Experimental
干预措施: BMN 307 (Drug)
Dose 3 of BMN 307
Experimental
干预措施: BMN 307 (Drug)
结局指标
主要结局
Change from baseline in mean Plasma Phe levels
时间窗: baseline, week 12
次要结局
- Change from baseline in mean Plasma Phe levels(baseline, week 96)
- Change from baseline in dietary protein intake from intact food(baseline, week 96)
- Number of participants with treatment-emergent adverse event(At 5 years)
研究者
研究点 (3)
Loading locations...
相似试验
已完成
2 期
Open Label, Dose Escalation Study for the Safety and Efficacy of STP705 in Adult Patients With Basal Cell CarcinomaBasal Cell CarcinomaNCT04669808Sirnaomics32
招募中
1 期
A Study of Oral Nuvisertib (TP-3654) in Patients With MyelofibrosisMyelofibrosisNCT04176198Sumitomo Pharma America, Inc.240
招募中
1 期
ADG126 in Combination With Pembrolizumab in Patients With Advanced/Metastatic Solid TumorsAdvanced/Metastatic Solid TumorsNCT05405595Adagene Inc186
已完成
1 期
RBM-007 in Subjects witH ExudatIve Age-related Macular DegenerationAge-related Macular DegenerationNCT03633084Ribomic USA Inc9
招募中
1 期
Phase I/II Study of IPG1094 in Advanced Solid Tumors PatientsSolid TumorNCT06212076Nanjing Immunophage Biotech Co., Ltd60