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A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
NCT02246218 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02246218
The ClinicalTrials.gov registry entry for NCT02246218 describes a study currently listed as completed. It is categorized as Phase 4, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 27 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Amgen, which has 266 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT02246218 reports 17 study locations spanning 15 distinct geographic areas — top geographies include California, Pennsylvania, District of Columbia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT02246218?
The interventions under investigation include: RAVICTI (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT02246218?
This trial is sponsored by Amgen, which has 266 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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