Conditions: Phenylketonuria (PKU)
Keywords: PKU PEG-PAL Prism BioMarin rAvPAL-PEG BMN165
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 215
Healthy volunteers: no

Brief 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 adults with PKU.

Detailed description:
165-302 is a Phase 3, 4 Part, Randomized, Double-Blind, Placebo-Controlled, Discontinuation Study to Evaluate the Efficacy and Safety of Self Administered Subcutaneous Injections of BMN 165 by Adults With PKU.

This study is open only to adults who have been exposed to BMN165 in a previous study.

Study BMN 165-302 is a four-Part, Phase 3 study.

* Part 1: Open-label period
* Part 2: A Randomized, double-blind, placebo-controlled period of 8 weeks
* Part 3: Two sub-Parts to Part 3:- Part 3A - BMN 165 administered using vial and syringe during this open label period. Part 3B - BMN 165 administered using prefilled syringe during this open-label period.
* Part 4: A long-term, open-label extension period.

Arms:
arm 1: BMN165 20mg/day self-administered daily arm 2: 20 mg/day Placebo self-administered daily arm 3: BMN165 40mg/day self-administered daily arm 4: 40 mg/day Placebo self-administered daily

Interventions: intervention 1: BMN165 20mg/day intervention 2: BMN165 40mg/day intervention 3: Placebo
Intervention descriptions: intervention 1: BMN165 20mg/day self-administered daily intervention 2: BMN165 40mg/day self-administered daily intervention 3: Non-drug placebo, self-administered daily

Protocol document: