MedTrace Logo

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

NCT03335488 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2024-07-01

Study results available
· View outcomes & findings →

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.

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

Sponsors & Collaborators

Principal Investigators

  • MD · Amgen

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-02-20
Primary Completion
2022-07-05
Completion
2022-12-20
FDA Drug
Yes

Countries

  • United States
  • Italy
  • Spain
  • Switzerland

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03335488 on ClinicalTrials.gov