Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)

Completed · Phase 4

Conditions studied: Urea Cycle Disorder

In brief

This is a randomized, controlled, open-label parallel arm study to assess the safety, tolerability, pharmacokinetics and ammonia control, of RAVICTI® as compared to Sodium phenylbutyrate (NaPBA) in urea cycle disorder subjects not currently or previously chronically treated with phenylacetic acid (phenylacetate; PAA) prodrugs. The study design will include: 1) Baseline Period; 2) Initial Treatment Period; 3) a RAVICTI only Transition Period 4) a RAVICTI only Maintenance Period; and 5) a RAVICTI only Safety Extension Period. The study will run for approximately 25 weeks.

Key facts

Study ID: NCT03335488
Run by: Amgen
People needed: 16
Starts: 2018-02-20
Expected to finish: 2022-12-20
Last updated by the study team: 2024-07-01

Who can join

Age: any, up to 99. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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 n-acetylglutamate synthetase (NAGS) deficiency.
Suspected diagnosis is defined as having experienced a hyperammonemic crisis (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
Birth 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 study and for 30 days after the last dose of study drug. Acceptable forms of contraception are (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.

You may not qualify if…

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 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.
Has undergone liver transplantation, including hepatocellular transplant.
Subjects on sodium benzoate (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 phenylbutyric acid (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.

Where it is running

University of Florida (UF) - Shands Hospital — Gainesville, Florida, United States
Mount Sinai School of Medicine — New York, New York, United States
University Hospitals Case Medical Center — Cleveland, Ohio, United States
Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
University of Texas, Southwestern Medical Centre — Dallas, Texas, United States
University of Utah — Salt Lake City, Utah, United States
Azienda Ospedaliera Universitaria Di Padova, U.O.C. Malattie Metaboliche Ereditarie, Dipartimento della Salute della Donna e del Bambino — Padua, Veneto, Italy
Bambino Gesù Children's Research Hospital — Rome, Italy
Hospital Materno-Infantil (HRU Carlos Haya) — Málaga, Andalusia, Spain
Hospital Universitario de Cruces — Barakaldo, Vizcaya, Spain
Universitätsspital, Inselspital Bern — Bern, Switzerland

Full record on ClinicalTrials.gov

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