A study to compare RAVICTI® against Sodium Phenylbutyrate (NaPBA) in the treatment of Urea Cycle Disorders (UCDs) in patients who have not received Phenylbutyrate treatment before.
- Conditions
- rea Cycle Disorders (UCDs)MedDRA version: 20.1 Level: LLT Classification code 10013373 Term: Disorders of urea cycle metabolism System Organ Class: 200000003094Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
- Registration Number
- EUCTR2015-000075-27-BE
- Lead Sponsor
- Horizon Therapeutics, LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Not specified
- Target Recruitment
- 30
• Signed informed consent given by the subject or the subject’s parent/legal guardian for those under 18 years of age or the age of consent by local regulation
• Male and female subjects with a suspected or confirmed UCD diagnosis of any subtype, except NAGS deficiency
- Suspected diagnosis is defined as having experienced an HAC or a documented high ammonia of =100 µmol/L
- Confirmed diagnosis is determined via enzymatic, biochemical, or genetic testing
• Requires nitrogen binding agents according to the judgment of the Investigator
• = 2 months of age and older
• 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 an oral phenylbutyrate (RAVICTI, NaPBA, Pheburane, or other) longer than 14 consecutive days within one year prior to enrollment
- Temporary use of oral PBA 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 transition to a PAA prodrug during the Initial Treatment Period
• 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method