NCT03335488 - Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

February 20, 2018

Primary Completion Date

July 5, 2022

Study Completion Date

December 20, 2022

Conditions

Urea Cycle Disorder

Interventions

DRUG

RAVICTI

RAVICTI, Oral Liquid Product 17.5 mL maximum total daily dose

DRUG

NaPBA

"* NaPBA in patients weighing \< 20 Kg - 600 mg/Kg, maximum total daily dose~* NaPBA in patients weighing \> 20 Kg - 13 g/m2, maximum total daily dose"

Trial Locations (11)

3010

Universitätsspital, Inselspital Bern, Bern

10029

Mount Sinai School of Medicine, New York

15224

Children's Hospital of Pittsburgh of UPMC, Pittsburgh

29006

Hospital Materno-Infantil (HRU Carlos Haya), Málaga

35128

Azienda Ospedaliera Universitaria Di Padova, U.O.C. Malattie Metaboliche Ereditarie, Dipartimento della Salute della Donna e del Bambino, Padua

48903

Hospital Universitario de Cruces, Barakaldo

84132

University of Utah, Salt Lake City

753908565

University of Texas, Southwestern Medical Centre, Dallas

32610-0214

University of Florida (UF) - Shands Hospital, Gainesville

44106-6005

University Hospitals Case Medical Center, Cleveland

00165

Bambino Gesù Children's Research Hospital, Rome