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NCT01560286 · ClinicalTrials.gov registry record · Phase 2

A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks

A Phase 2 study of Phenylketonuria, sponsored by BioMarin Pharmaceutical.

Completed
Registry status
Phase 2
Development phase
24
Enrollment target
6
Study locations

NCT01560286: Completed Phase 2 study of Phenylketonuria, sponsored by BioMarin Pharmaceutical.

NCT01560286 is a Phase 2 study of Phenylketonuria that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 24 participants, below the 97-participant average among 12 other Phenylketonuria trials with a reported enrollment target (75% lower). The trial reports 6 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT01560286, a Phase 2 study of Phenylketonuria, has completed, sponsored by BioMarin Pharmaceutical.

COMPLETED
Registry status
Phase 2
Development phase
24 participants
Enrollment target
6
Study locations

Study Summary

The primary objective of the study is to evaluate the effect of dosing regimens of multiple subcutaneous (SC) doses of rAvPAL-PEG to induce an early and sustained Phe reduction while decreasing the frequency and severity of hypersensitivity reactions in patients with PKU.

Primary Outcome

All patients will have their plasma Phenylalanine (Phe) assessed. Please note that "Pharmacokinetics Sub-study" was not performed, and thus no results are available.

Conditions Studied

Interventions

  • BIOLOGICAL BMN 165 (rAvPAL-PEG)

Study Locations (6)

Colorado

  • The Children's Hospital - Aurora

Florida

  • University of Florida, Gainesville - Gainesville

Massachusetts

  • Children's Hospital Boston - Boston

Nebraska

  • Nebraska Medical Center - Omaha

New York

  • Albany Medical Center - Albany

Utah

  • University of Utah Hospital - Salt Lake City

Trial Details

FieldValue
Enrollment Target 24 participants
Start Date 2012-05
Est. Completion 2015-07
Phase Phase 2
BioMarin Pharmaceutical

80 total trials

What the finished NCT01560286 record still lists

NCT01560286 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 24 participants, a relatively small participant target, below the 97-participant average among 12 other Phenylketonuria trials with a reported enrollment target (75% lower).

The record links to 1 condition, with Phenylketonuria appearing as the primary indexed condition, and to 1 intervention - of which BMN 165 (rAvPAL-PEG) is the first listed.

NCT01560286 lists 6 locations in 6 states (Colorado, Florida, Massachusetts).

Frequently Asked Questions

What is clinical trial NCT01560286 about?

NCT01560286 is a clinical study titled "A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks". The primary objective of the study is to evaluate the effect of dosing regimens of multiple subcutaneous (SC) doses of rAvPAL-PEG to induce an early and sustained Phe reduction while decreasing the frequency and severity of hypersensitivity reactions in patients with PKU.

What is the current status of trial NCT01560286?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 24 participants. The study started on 2012-05. Estimated completion is 2015-07.

What conditions does trial NCT01560286 study?

This clinical trial studies the following conditions: Phenylketonuria.

What interventions are being tested in trial NCT01560286?

The interventions under investigation include: BMN 165 (rAvPAL-PEG) (BIOLOGICAL).

Who is sponsoring clinical trial NCT01560286?

This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT01560286 being conducted?

This trial has 6 study locations across Colorado, Florida, Massachusetts, Nebraska, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Similar trials for Phenylketonuria

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT01560286's enrollment target sits among peer trials

24 11th of 12 higher than 2 of 12 other Phenylketonuria trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phenylketonuria 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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Source: ClinicalTrials.gov NCT01560286, the US trial registry maintained by the National Library of Medicine. NCT01560286 (small enrollment · multi site footprint · completed) retrieved and formatted by PlainTrial, see methodology.