Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

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NCT05270837 · results posted

Sponsored by BioMarin Pharmaceutical (industry) · BMRN — their whole pipeline →.

Phase

Phase 3

Status

Active, not recruiting

Study type

Interventional

Design

Randomized · None · Treatment

Enrollment

55actual

Sites

Countries

Germany, United States

Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →

Source: ClinicalTrials.gov, retrieved 2026-08-19

Dates

each axis at the precision it was filed, with the registry's own basis

Date

Filed

Basis

What it is

Start

2022-06-17

actual

When the study began enrolling

Primary completion

2025-01-14

actual

The date the last participant is measured for the primary outcome — the readout window

Study completion

Oct 2027

estimated

The whole study's end, after follow-up

First posted

2022-03-08

actual

When this record first appeared on the registry

Results posted

2026-07-21

actual

When the sponsor posted results to the registry

Record updated

2026-07-21

actual

The sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

Phenylketonuria (PKU)

Interventions

Drug: Pegvaliase — also filed as PEG PAL, BMN 165
Other: Diet Only

Primary outcomes

what the primary-completion date above is the date OF

Change From Baseline in Blood Phe Concentration Following 72 Weeks (Average of the Week 69 and Week 73) on Study.

measured Baseline to Week 72 (average of the Week 69 and Week 73)

Incidence of Treatment-emergent Adverse Events (Part 1)

measured Up to Week 73

Percentage of Participants With Positive Anti-pegvaliase Total Antibody (TAb)