苯丙酮酸尿(北大) is an inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH).
了解博彩平台网址大全在北京大学的临床试验.
1/2期开放标签, 剂量递增研究确定BMN 307的安全性和有效性, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 µmol/L
苯丙酮酸尿(北大)
这个开放标签, 研究分为两部分，旨在确定剂量和疗效, 安全, bmn307的耐受性
BMN 307
活跃，不招人
第1/ 2期
苯丙酮尿症的前瞻性临床研究
苯丙酮酸尿(北大)
The objective of this study is to collect longitudinal data assessing markers of disease and clinical outcomes in PKU subjects ≥ 14 years old.
活跃，不招人
观察
一个多中心, 观察 Study to Evaluate the Real-World Outcomes of Pegvaliase (BMN 165) in Subjects with Phenylketonuria in Germany
苯丙酮酸尿(北大)
In addition to the efficacy and 安全 profile of pegvaliase that has been established in clinical trials; there remains an important need to provide data to prescribers and the phenylketonuria (PKU) community on the real-world usage of pegvaliase, 以及相关的结果, to support clinical practice optimization and provide greater understanding of important clinical issues in PKU
Pegvaliase (BMN 165)
这项研究目前正在招募参与者
第四阶段
A第3期 Multi Center Study to Evaluate the 安全 and 功效 of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) with Phenylketonuria Featuring an Open Label 随机 Two Arm (Active vs Diet Only Control) Design
苯丙酮酸尿(北大)
Evaluate 安全 and efficacy of Pegvaliase (BMN 165) in adolescents with PKU when administered as an Induction/滴定法 and Maintenance dose regimen.
Pegvaliase (BMN 165)
这项研究目前正在招募参与者
第三阶段
A第3期, 非盲, Multi-Center Study to Assess the 安全 and 功效 of Pegvaliase (BMN 165) in Japanese Subjects 18 Years of Age and Older With Phenylketonuria
苯丙酮酸尿(北大)
The primary objective of the study is to evaluate the efficacy and 安全 of Pegvaliase (BMN 165) in Japanese subjects ≥ 18 years of age with PKU when administered as an Induction/滴定法 and Maintenance dose regimen.
Pegvaliase (BMN 165)
这项研究正在进行中，但没有招募参与者.
第三阶段
An Open-label Extension Study to Evaluate the 安全 and 功效 of Subcutaneous Injections of Pegvaliase (> 40mg/day Dose) in Adults with Phenylketonuria.
苯丙酮酸尿(北大)
Evaluate long-term 安全 and efficacy of Pegvaliase in (> 40mg/day dose) in adults with PKU
聚乙二醇酶，rAvPAL-PEG或bmn165
第三阶段
本研究已经完成
一个四, 第三阶段, 随机, 双盲, 安慰剂对照, 十字, Discontinuation Study to Evaluate the 功效 and 安全 of Subcutaneous Injections of BMN 165 Self Administered by Adults with Phenylketonuria
苯丙酮酸尿(北大)
The BMN 165 clinical development program has been designed to demonstrate the 安全 and efficacy of BMN 165 in reducing blood Phe concentrations in adults with PKU
聚乙二醇酶，rAvPAL-PEG或bmn165
本研究已经完成
第三阶段
第二期工程的长期延期, 评价安全性的开放标签剂量研究, 功效, PKU患者多次皮下剂量的rAvPAL-PEG耐受性
苯丙酮酸尿(北大)
评估长期安全性, tolerability and effectiveness of injections of an investigational drug for people with PKU who have trouble controlling and maintaining their Phe levels
聚乙二醇酶，rAvPAL-PEG或bmn165
本研究已经完成
第二阶段
苯丙酮尿患者皮下注射rAvPAL-PEG治疗24周的研究
苯丙酮酸尿(北大)
Evaluate the effect of dosing regimens of multiple subcutaneous (SC) doses of rAvPAL-PEG to induce an early and sustained Phe reduction while decreasing the frequency and severity of hypersensitivity reactions in patients with PKU
聚乙二醇酶，rAvPAL-PEG或bmn165
本研究已经完成
第二阶段
A第2期, 安全性评价的开放标签研究, 耐受性, 对苯丙酮尿患者每日给予Ravpal-PEG皮下剂量水平的疗效
苯丙酮酸尿(北大)
Evaluate the effect of daily administration of rAvPAL-PEG on the reduction of blood Phe concentrations in subjects with PKU
聚乙二醇酶，rAvPAL-PEG或bmn165
本研究已经完成
第二阶段
A第3期, 非盲, 随机, 评估诱导安全性和耐受性的多中心研究, 滴定法, and Maintenance Dose Regimen of Pegvaliase Self-Administered by Adults with Phenylketonuria Not Previously Treated with Pegvaliase
苯丙酮酸尿(北大)
Evaluate the 安全 and tolerability of injections of an investigational drug (also called PEG PAL) for people with PKU who have not received Pegvaliase in the past
聚乙二醇酶，rAvPAL-PEG或bmn165
本研究已经完成
第三阶段
盐酸沙普霉素(bmn162)对苯丙酮尿患儿认知的影响
苯丙酮酸尿(北大)
IV期开放标签, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5-Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (BMN 162) for 7 Years
盐酸沙普霉素(BMN 162)
本研究已经完成
第四阶段
观察 Study on the Long-Term 安全 of 盐酸沙普霉素(BMN 162) Treatment in Patients With Hyperphenylalaninemia (HPA) Due to 苯丙酮酸尿(北大) or BH4 Deficiency (KAMPER)
苯丙酮酸尿(北大)
评估盐酸沙普霉素(BMN 162)治疗的长期安全性
盐酸沙普霉素(BMN 162)
本研究已经完成
第四阶段，观察性
苯丙酮尿症(PKU)的人口统计学、结局和安全性登记
苯丙酮尿症(PKU)，高苯丙氨酸血症
评价盐酸沙普霉素长期治疗的安全性(bmn162)
盐酸沙普霉素(BMN 162)
本研究已经完成
第四阶段，观察性
盐酸沙普霉素(bmn162)在4岁以下苯丙酮尿症患者中的应用
苯丙酮酸尿(北大)
Evaluate the efficacy after 26 weeks of 盐酸沙普霉素(BMN 162) treatment + Phe-restricted diet therapy in increasing dietary Phe tolerance, as compared to dietary therapy alone in =120 to <360 μmol/L).
盐酸沙普霉素(BMN 162)
本研究已经完成
阶段3 b
阶段3 b 非盲 Study to Evaluate the Effect of 盐酸沙普霉素(BMN 162) on Neurocognitive Function, 血液苯丙氨酸浓度的维持, 安全, 苯丙酮尿症幼儿群体药代动力学研究
苯丙酮酸尿(北大)
主要目标 – To evaluate the long-term efficacy of 盐酸沙普霉素(BMN 162) in preserving neurocognitive function in children with phenylketonuria (PKU) when treatment is initiated at 0 to 6 years of age at date of screening. 二级目标 -评估盐酸沙普蝶素(BMN 162)在研究人群中的长期安全性 – To evaluate the effect of 盐酸沙普霉素(BMN 162) on growth parameters in the study population
盐酸沙普霉素(BMN 162)
本研究已经完成
第四阶段
Study to Evaluate the 安全 and 功效 of Sapropterin Dihydrochloride (BMN 162) in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
苯丙酮酸尿(北大)
Evaluate the efficacy of 盐酸沙普霉素(BMN 162) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria
盐酸沙普霉素(BMN 162)
本研究已经完成
第三阶段
一个双盲, 安慰剂对照, 随机 Study to Evaluate the 安全 and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects with Phenylketonuria
苯丙酮酸尿(北大)
Evaluate the 安全 and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU
盐酸沙普霉素(BMN 162)
本研究已经完成
第三阶段