A Switch-Over, Open-Label Study of the Safety, Pharmacokinetics, and Efficacy of HPN-100, Followed by Long-Term Treatment with HPN-100, in Pediatric Subjects under 6 Years of Age with Urea Cycle Disorders (UCDs)

Hyperion Therapeutics, Inc.0 个研究点目标入组 23 人开始时间: 2015年2月10日最近更新: 2015年2月16日

适应症

试验速览

暂无简介。

•1. Male or female patient aged 29 days to < 6 years old; calculation of the lower age limit began at the corrected gestational age of 40 weeks for patients born prematurely.
•2. Signed informed consent by the patient’s legally acceptable representative
•3. Suspected or confirmed UCD diagnosis of any subtype, except NAGS deficiency
•4. On stable dose of NaPBA powder for at least 5 days before Day 1
•5. Not receiving NaBz for at least 5 days before Day 1
•6. No concomitant illness that would preclude safe participation as judged by the investigator
•7. Able to receive medication orally
•8. Had not undergone liver transplantation, including hepatocellular transplantation
•9. Judged sufficiently stable and compliant with diet and treatment to be suitable for enrollment
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 23
•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

•1. Screening ammonia level > 100 µmol/L and signs and symptoms indicative of hyperammonemia; patients may have been rescreened after their ammonia was controlled
•and they were clinically stable, at the discretion of the investigator.
•2. Use of any investigational drug within 30 days of Day 1
•3. Active infection (viral or bacterial) or any other condition that could increase ammonia levels
•4. Any clinical or laboratory abnormality of Grade 3 or greater severity according to the Common Terminology Criteria for Adverse Events (CTCAE) v4.03, except Grade 3 elevations in ammonia and liver enzymes, defined as levels 5–20 times ULN in alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma glutamyl transpeptidase (GGT) in a clinically stable patient
•5. Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, could have put the patient at increased risk by participating in this study
•6. Known hypersensitivity to PAA or PBA
•7. Liver transplant, including hepatocellular transplant
•8. Currently treated with Carbaglu® (carglumic acid)

发起方