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NCT02246218 · ClinicalTrials.gov registry record · Phase 4
A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
A Phase 4 study of Urea Cycle Disorder, sponsored by Amgen.
- Completed
- Registry status
- Phase 4
- Development phase
- 27
- Enrollment target
- 17
- Study locations
NCT02246218: Completed Phase 4 study of Urea Cycle Disorder, sponsored by Amgen.
NCT02246218 is a Phase 4 study of Urea Cycle Disorder that has completed, run by Amgen. The registered enrollment target is 27 participants, below the 43-participant average among 4 other Urea Cycle Disorder trials with a reported enrollment target (37% lower). The trial reports 17 study locations across 15 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02246218, a Phase 4 study of Urea Cycle Disorder, has completed, sponsored by Amgen.
- COMPLETED
- Registry status
- Phase 4
- Development phase
- 27 participants
- Enrollment target
- 17
- Study locations
Study Summary
This is an open-label study consisting of a transition period to RAVICTI, followed by a safety extension period for at least 6 months and up to 24 months of treatment with RAVICTI, depending on age at enrollment. It is designed to capture information important for evaluating safety, pharmacokinetics and efficacy in young children. Subjects who are followed by or referred to the Investigator for management of their UCD. Subjects eligible for this study will include patients ranging from newborn to \< 2 years of age with either a diagnosed or clinically suspected UCD.
Primary Outcome
The percentage of participants with successful transition is based on Investigator response to the question, "Has transition to 100% RAVICTI been successful with controlled ammonia?" For participants 2 months of age and older, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normaliza
Conditions Studied
Interventions
- DRUG RAVICTI
Study Locations (17)
California
- Kaiser Permanente Regional Metabolic Center - Los Angeles
- Stanford Center for Clinical & Translational Research & Education - Palo Alto
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
- Children's Hospital of Pittsburgh - Pittsburgh
District of Columbia
- Children's National Medical Center - Washington D.C.
Florida
- Shands at University of Florida - Gainesville
Georgia
- Emory University - Decatur
Indiana
- Indiana University - Indianapolis
Maine
- Maine Medical Center - Portland
Michigan
- Children's Hospital of Michigan - Detroit
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 27 participants |
| Start Date | 2014-12-31 |
| Est. Completion | 2017-07-17 |
| Phase | Phase 4 |
What the finished NCT02246218 record still lists
NCT02246218 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 27 participants, a relatively small participant target, below the 43-participant average among 4 other Urea Cycle Disorder trials with a reported enrollment target (37% lower).
The record links to 1 condition, with Urea Cycle Disorder appearing as the primary indexed condition, and to 1 intervention - of which RAVICTI is the first listed.
NCT02246218 lists 17 locations in 15 states (California, Pennsylvania, District of Columbia).
Frequently Asked Questions
What is clinical trial NCT02246218 about?
NCT02246218 is a clinical study titled "A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders". This is an open-label study consisting of a transition period to RAVICTI, followed by a safety extension period for at least 6 months and up to 24 months of treatment with RAVICTI, depending on age at enrollment. It is designed to capture information important for evaluating safety, pharmacokinetics...
What is the current status of trial NCT02246218?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 27 participants. The study started on 2014-12-31. Estimated completion is 2017-07-17.
What conditions does trial NCT02246218 study?
This clinical trial studies the following conditions: Urea Cycle Disorder.
What interventions are being tested in trial NCT02246218?
The interventions under investigation include: RAVICTI (DRUG).
Who is sponsoring clinical trial NCT02246218?
This trial is sponsored by Amgen, which has 558 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02246218 being conducted?
This trial has 17 study locations across California, District of Columbia, Florida, Georgia, Indiana. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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