NCT01889862

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU

A Four-Part, Phase 3, Randomized, Double-Blind, Placebo- Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self-Administered by Adults With Phenylketonuria (PKU)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 215 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Phenylketonuria (PKU)

Interventions

Type	Name	Description
DRUG	BMN165 20mg/day	BMN165 20mg/day self-administered daily
DRUG	BMN165 40mg/day	BMN165 40mg/day self-administered daily
DRUG	Placebo	Non-drug placebo, self-administered daily

Timeline

• Start date: 2013-07-29
• Primary completion: 2016-01-13
• Completion: 2019-02-05
• First posted: 2013-07-01
• Last updated: 2021-05-21
• Results posted: 2019-07-12

Locations

29 sites across 1 country: United States