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NCT03505125 · ClinicalTrials.gov registry record
A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
A clinical trial of Phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- 63
- Enrollment target
- 14
- Study locations
NCT03505125: Completed study of Phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical.
NCT03505125 is a study of Phenylketonuria (PKU) that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 63 participants, below the 172-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (63% lower). The trial reports 14 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03505125, a study of Phenylketonuria (PKU), has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- 63 participants
- Enrollment target
- 14
- Study locations
Study Summary
The 165-901 study is designed to identify the appropriate tools for use in future interventional studies on the neurocognitive effects of pegvaliase on adults with PKU.
Primary Outcome
60 item survey asking patients, observers and clinical experts about the symptoms and behaviors associated with PKU and changes in Phe level.
Conditions Studied
Study Locations (14)
Other
- Rheumatology Unit, Clinic for Inborn Errors of Metabolism - Leipzig
- Hospital of Padova - Padua
- Umberto I Polyclinic of Rome - Roma
- Hacettepe University - Ankara
- Ege University School of Medicine - Izmir
Florida
- University of Florida - Gainesville
Georgia
- Emory University Department of Human Genetics - Decatur
Illinois
- Ann and Robert H Lurie Children's Hospital - Chicago
Massachusetts
- Boston Children's Hospital - Boston
British Columbia
- Adult Metabolic Diseases Clinic, Vancouver General Hospital - Vancouver
Cedex 15
- Necker Children's Hospital - Paris
Cedex 9
- Bretonneau Hospital, Internal Medicine Department - Tours
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 63 participants |
| Start Date | 2018-03-31 |
| Est. Completion | 2019-07-31 |
What the finished NCT03505125 record still lists
NCT03505125 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 63 participants, a relatively small participant target, below the 172-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (63% lower).
The record links to 1 condition, with Phenylketonuria (PKU) appearing as the primary indexed condition, and to 0 interventions.
NCT03505125 lists 14 locations in 10 states (Other, Florida, Georgia).
Frequently Asked Questions
What is clinical trial NCT03505125 about?
NCT03505125 is a clinical study titled "A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults". The 165-901 study is designed to identify the appropriate tools for use in future interventional studies on the neurocognitive effects of pegvaliase on adults with PKU.
What is the current status of trial NCT03505125?
This trial is currently completed. The enrollment target is 63 participants. The study started on 2018-03-31. Estimated completion is 2019-07-31.
What conditions does trial NCT03505125 study?
This clinical trial studies the following conditions: Phenylketonuria (PKU).
Who is sponsoring clinical trial NCT03505125?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03505125 being conducted?
This trial has 14 study locations across Florida, Georgia, Illinois, Massachusetts, British Columbia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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