Long Term Use of HPN-100 in Urea Cycle Disorders
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 88
- 试验地点
- 16
- 主要终点
- Number of Participants With at Least One Adverse Event
研究概览
简要总结
This was an open-label, long-term safety study of HPN-100 (RAVICTI; glycerol phenylbutyrate) in participants with a urea cycle disorder (UCD) who completed the safety extensions of HPN-100-005 (NCT00947544; HPN-100-005SE), HPN-100-006 (NCT00947297; HPN-100-007), or HPN-100-012 (NCT01347073; HPN-100-012SE). The initial studies were 1- to 2-week crossover studies, and their associated safety extensions were 12-month, open-label studies. All participants who completed the initial studies were eligible to enroll in the associated safety extension studies, and new participants were also permitted to enroll directly into the safety extension studies.
详细描述
The duration of treatment in this study was open-ended. Participants were to return for clinic visits as prescribed by the investigator, and were to be seen at a minimum of every 6 months. At each clinic visit, participants were queried about any adverse events (AEs) or hyperammonemic crises (HACs) that occurred since the last visit. Physical and neurological examinations were performed, and blood samples were collected for the analysis of ammonia, amino acid panels, and routine clinical laboratory safety tests. Participants underwent neuropsychological testing at baseline, every 12 months thereafter, and at the final study visit.
Study acquired from Horizon in 2024.
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
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects who completed Studies HPN-100-005SE, HPN-100-007, or HPN-100-012SE
- •Signed informed consent by participant and/or participant's legally authorized representative
- •Negative pregnancy test for all females of childbearing potential
排除标准
- •Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may have put the participant at increased risk when participating
- •Known hypersensitivity to PAA (phenylacetate) or PBA (phenylbutyrate).
- •Liver transplant, including hepatocellular transplant
- •Pregnant, breastfeeding or lactating females
研究组 & 干预措施
HPN-100
Participants continued HPN-100 treatment after completion of HPN-100-005SE, HPN-100-007, or HPN-100-012SE.
干预措施: HPN-100 (Drug)
结局指标
主要结局
Number of Participants With at Least One Adverse Event
时间窗: From the time of informed consent until 7 days after the last dose of study drug, up to 66 months
Safety was assessed by the incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (SAEs). An AE/adverse experience was any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which did not necessarily have a causal relationship with this treatment. For additional information regarding adverse events, please see the safety section of the record.
次要结局
- Mean Wechsler Abbreviated Scale of Intelligence (WASI) Scores(Baseline, Month 12, Month 24, Month 36, Month 48, and study exit visit (up to 66 months))
- Mean Behavior Rating Inventory of Executive Function (BRIEF) Scores(Baseline, Month 12, Month 24, and study exit visit (up to 66 months))
- Mean Normalized Blood Ammonia Levels(From baseline through the end of the study, up to 66 months)
- Number of Hyperammonemic Crises(From the time of informed consent until 7 days after the last dose of study drug, up to 66 months)
- Causes of Hyperammonemic Crises(From the time of informed consent until 7 days after the last dose of study drug, up to 66 months)
- Mean Child Behavior Checklist (CBCL) Problems Scores(Baseline, Month 12, Month 24, and study exit visit (up to 66 months))
- Mean California Verbal Learning Test Scores: Short and Long Delay Free Recall, Short and Long Delay Cued Recall, CVLT-II-Learning Slope, and Total Word Recognition Discrimination(Baseline, Month 12, Month 24, Month 36, Month 48, and study exit visit (up to 66 months))
- Mean California Verbal Learning Test Scores: List A Total 1-5 T-Scores(Baseline, Month 12, Month 24, Month 36, Month 48, and study exit visit (up to 66 months))