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NCT01819727 · ClinicalTrials.gov registry record · Phase 3
An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
A Phase 3 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 261
- Enrollment target
NCT01819727: Completed Phase 3 study, sponsored by BioMarin Pharmaceutical.
NCT01819727 is a Phase 3 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 261 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (66% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01819727, a Phase 3 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 261 participants
- Enrollment target
Study Summary
The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in patients 18 to 70 years old with hyperphenylalaninemia due to PKU. Study BMN 165-301 is a Phase 3, open-label, randomized study designed to further characterize the safety of BMN 165 during two induction, titration, and maintenance dose regimens in adults with PKU who have not had previous exposure to BMN 165 (naive). Subjects will be randomized (1:1) to titrate up to one of two dose regimens. Other key features of this study are the dose regimens chosen for induction and titration; the study duration; self administration of study drug; and the chosen tertiary objectives.
Primary Outcome
Hypersensitivity AEs will be identified in two ways: * Broad Algorithmic anaphylactic reaction Standardized MedDRA Queries (SMQ) * Modified Hypersensitivity SMQ to include above additional preferred terms
Interventions
- DRUG BMN 165
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 261 participants |
| Start Date | 2013-05 |
| Est. Completion | 2015-11-25 |
| Phase | Phase 3 |
What the finished NCT01819727 record still lists
NCT01819727 is an interventional study that assigns participants to a tested intervention. The registered 261 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (66% lower).
The record links to 0 conditions, and to 1 intervention - of which BMN 165 is the first listed.
NCT01819727 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01819727 about?
NCT01819727 is a clinical study titled "An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165". The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in patients 18 to 70 years old with hyperphenylalaninemia due to PKU. Study BMN 165-301 is a Phase 3, open-label, randomized study designed to further cha...
What is the current status of trial NCT01819727?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 261 participants. The study started on 2013-05. Estimated completion is 2015-11-25.
What interventions are being tested in trial NCT01819727?
The interventions under investigation include: BMN 165 (DRUG).
Who is sponsoring clinical trial NCT01819727?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01819727's enrollment target sits among peer trials
261 1228th of 2000 higher than 771 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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