A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
Public ClinicalTrials.gov record NCT02246218. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI®) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT02246218
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 4
- Enrollment
- 27 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 1 Day to 2 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 30, 2014
- Primary completion
- Oct 16, 2016
- Completion
- Jul 16, 2017
- Last update posted
- Jun 30, 2024
2014 – 2017
United States locations
- U.S. sites
- 16
- U.S. states
- 14
- U.S. cities
- 16
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Kaiser Permanente Regional Metabolic Center | Los Angeles | California | 90027 | — |
| Stanford Center for Clinical & Translational Research & Education | Palo Alto | California | 94034 | — |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | — |
| Shands at University of Florida | Gainesville | Florida | 32610 | — |
| Emory University | Decatur | Georgia | 30033 | — |
| Indiana University | Indianapolis | Indiana | 46202 | — |
| Maine Medical Center | Portland | Maine | 04102 | — |
| Children's Hospital of Michigan | Detroit | Michigan | 48201 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Mount Sinai School of Medicine | New York | New York | 10029 | — |
| University Hospitals Case Medical Center | Cleveland | Ohio | 44106 | — |
| Oregon Health & Science University | Portland | Oregon | 97239 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| University of Utah | Salt Lake City | Utah | 84132 | — |
| Seattle Children's Hospital | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02246218, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 30, 2024 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02246218 live on ClinicalTrials.gov.