Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU — Prism-2

Phenylketonuria (PKU) Clinical Trial

Official title:
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

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT01889862
Study type	Interventional
Source	BioMarin Pharmaceutical
Contact
Status	Completed
Phase	Phase 3
Start date	July 29, 2013
Completion date	February 5, 2019

View Details

See also
Status	Clinical Trial	Phase
Active, not recruiting	`NCT04480567` - AAV Gene Therapy Study for Subjects With PKU	Phase 1/Phase 2
Completed	`NCT03694353` - Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU	Phase 3
Completed	`NCT04086511` - PANDA: PKU Amino Acid Evaluation
Completed	`NCT02440932` - Impact of Phenylketonuria-type Diet on Appetite, Appetite Hormones and Diet Induced Thermogenesis	N/A
Completed	`NCT01617070` - Effects of Kuvan on Melatonin Secretion	Phase 4
Completed	`NCT03505125` - A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
Terminated	`NCT01806051` - A Pilot Study on Diurnal Variation	N/A
Recruiting	`NCT05813678` - A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
Active, not recruiting	`NCT05270837` - Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria	Phase 3
Completed	`NCT02009904` - Simple Breath Test to Examine Phenylalanine Metabolism
Recruiting	`NCT06305234` - A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
Recruiting	`NCT03309345` - Body Composition, Energy Intake and Expenditure in People With Phenylketonuria	N/A