A Randomised, Controlled, Open-Label Crossover Study of the Safety, Pharmacokinetics and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate [GPB]) Oral Liquid and Sodium Phenylbutyrate (NaPBA) in Phenylbutyrate Treatment Naïve Patients with Urea Cycle Disorders (UCDs)

Horizon Therapeutics, Inc0 个研究点目标入组 16 人开始时间: 2015年12月1日最近更新: 2024年3月12日

适应症

试验速览

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•Signed informed consent given by the subject or the subject’s parent/legal guardian for those under 18 years of age
•Male and female subjects with a suspected or confirmed UCD diagnosis of any subtype, except NAGS deficiency
•- Suspected diagnosis is defined as having experienced a HAC or a documented high ammonia of =100 µmol/L (see protocol Section 3.3 for HAC definition)
•- Confirmed diagnosis is determined via enzymatic, biochemical, or genetic testing
•Requires nitrogen-binding agents according to the judgment of the Investigator
•2 years of age and older
•>15 kg in body weight
•All females of childbearing potential and all sexually active males must agree to use an acceptable method of contraception from signing the informed consent throughout the duration of the study and for 30 days after the last dose of study drug.. Appropriate contraceptive methods include hormonal contraceptives (oral, injected, implanted, or transdermal), tubal ligation, intrauterine device, hysterectomy, vasectomy, or double barrier methods. Abstinence is an acceptable form of birth control, though appropriate contraception must be used if the subject becomes sexually active.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 23
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 7
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

•Subject has received chronic treatment with RAVICTIor NaPBA longer than 14 days within one year prior to enrollment
•- Temporary use of NaPBA for acute management of a hyperammonemic crisis in the past is acceptable.
•Any concomitant illness (e.g., malabsorption or clinically significant liver or bowel disease) which would preclude the subject’s safe participation, as judged by the Investigator
•Have undergone liver transplantation, including hepatocellular transplant
•- Subjects on NaBz at Baseline will be excluded if they are viewed by the Investigator as being unable to undergo NaBz withdrawal with concomitant replacement by a PAA prodrug during Period 1
•Known hypersensitivity to PBA or any excipients of the NaPBA/PBA formulations.
•Pregnant or breast-feeding patients. Women of childbearing potential must have a pregnancy test performed at the Baseline Visit prior to the start of study drug.

发起方

Horizon Therapeutics, Inc