A Phase 2, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 (glyceryl tri-[4-phenylbutyrate]) Compared to Sodium Phenylbutyrate in Adult and Pediatric Subjects with Urea Cycle Disorders

Hyperion Therapeutics Limited0 个研究点目标入组 16 人开始时间: 2008年7月25日最近更新: 2012年8月14日

适应症

试验速览

暂无简介。

•Male and female subjects = 6 years old
•Signed informed consent by subject and/or subject’s legally acceptable representative
•Diagnosis of urea cycle disorder (enzyme or transporter deficiency) confirmed via enzymatic, biochemical or genetic testing
•Currently being treated with sodium PBA for a diagnosis of UCD for at least one week prior to the screening visit
•Able to perform and comply with study activities (including the ability to adhere to 24-hour urine collections both in the clinic and after discharge; i.e., no subjects in diapers)
•Willing to maintain a constant diet throughout the study (unless the investigator recommends a change based on safety considerations)
•Negative pregnancy test for all females of childbearing potential
•All females of childbearing potential and all sexually active males must agree to use an acceptable method of contraception throughout the study
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•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

•Use of any investigational drug (sodium PBA excepted) within 30 days prior to Day 1
•Active infection (viral or bacterial) or any other condition that may increase ammonia levels
•Laboratory values outside the normal range that are determined to be clinically significant by the investigator
•Any clinical or laboratory abnormality of Grade 3 or greater severity according to the CTCAE v3.0 (or for conditions not covered by the CTCAE, a severe or life-threatening toxicity); except that Grade 3 elevations in liver enzymes are allowed in an otherwise clinically stable subject
•Use of any medication known to significantly affect renal clearance (e.g., probenecid) or to increase protein catabolism (e.g., corticosteroids), or other medication (e.g., valproate) known to increase ammonia levels, within the 24 hours prior to Day 1
•History of diagnosis with long QT syndrome or QTc interval > 450 msec at screening
•Other medical conditions which in the judgment of the investigator preclude entry
•Known hypersensitivity to PAA or PBA
•Creatinine levels = 1.5 ? upper limit of normal (ULN)
•Liver transplant, including hepatocellular transplant
•Currently treated with sodium benzoate or Carbaglu® (carglumic acid)

发起方

Hyperion Therapeutics Limited