EUCTR2014-003247-36-Outside-EU/EEA进行中（未招募）不适用
A Phase 2, Fixed-Sequence, Open-Label, Switch-Over Study of the Safety and Tolerability of HPN 100 Compared to Sodium Phenylbutyrate in Children 6–17 Years of Age with Urea Cycle Disorders, with a Long-Term Safety Extension
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 17
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
- •Male and female patients 6–17 years old
- •Signed informed consent by patient’s legally acceptable representative and assent by patient, as applicable
- •Diagnosis of urea cycle disorder (enzyme or transporter deficiency) confirmed via enzymatic, biochemical, or genetic testing
- •On a stable dose of NaPBA for a diagnosis of UCD for at least 1 week prior to the Day 1 visit; if not on a stable dose of NaPBA at the initial screening visit, patients may have been converted to a stable dose of NaPBA during the screening period and enrolled as long as they were on a stable dose of NaPBA for at least 1 week prior to Day 1
- •Able to perform and comply with study activities, including blood draws and urine collections
- •Negative pregnancy test for all females of childbearing potential
- •All females of childbearing age and all sexually active males must have agreed to use an acceptable method of contraception throughout the study. Appropriate contraceptive methods included hormonal contraceptives (oral, injected, implanted, or transdermal), tubal ligation, intrauterine device, hysterectomy, vasectomy, or double barrier methods. Abstinence was an acceptable form of birth control, though appropriate contraception must have been used if the patient became sexually active.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 17
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Screening ammonia level of = 100 µmol/L or signs and symptoms indicative of hyperammonemia; patients may have been rescreened after their ammonia was controlled, at the discretion of the investigator
- •History of 4 or more hyperammonemic events (defined as clinical symptoms associated with ammonia of = 100 µmol/L) in the preceding 12 months
- •Use of any investigational drug within 30 days of Day 1
- •Active infection (viral or bacterial) or any other condition that may have increased ammonia levels
- •Any clinical or laboratory abnormality of Grade 3 or greater severity according to the Common Terminology Criteria for Adverse Events (CTCAE) v3.0, except Grade 3 elevations in liver enzymes, defined as levels 5–20 times upper limit of normal (ULN) in alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma glutamyl transpeptidase (GGT) in a clinically stable patient
- •Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may have put the patient at increased risk by participating in this study
- •Use of any medication known to significantly affect renal clearance (e.g., probenecid) or to increase protein catabolism (e.g., corticosteroids), or other medication known to increase ammonia levels (e.g., valproate), within the 24 hours prior to Day 1 and throughout the study
- •History of QTc interval prolongation or QTc interval > 450 msec at screening or baseline
- •Known hypersensitivity to PAA or PBA
- •Liver transplant, including hepatocellular transplant
- •Currently treated with sodium benzoate or Carbaglu® (carglumic acid) - At the discretion of the investigator, patients on sodium benzoate who were otherwise eligible to participate may have been switched to 100% NaPBA during the 30-day screening period as part of the study, and at least 7 days prior to Day 1 (Visit 2).
- •Breastfeeding or lactating
研究者
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