NCT03335488

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

A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Amgen · Industry
• Sex: All
• Age: 99 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

Study acquired from Horizon in 2024.

Conditions

• Urea Cycle Disorder

Interventions

Type	Name	Description
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

Timeline

• Start date: 2018-02-20
• Primary completion: 2022-07-05
• Completion: 2022-12-20
• First posted: 2017-11-07
• Last updated: 2024-07-01
• Results posted: 2023-07-10

Locations

11 sites across 4 countries: United States, Italy, Spain, Switzerland

Regulatory

• FDA-regulated drug study