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Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU | Clinical Trials | Clareo Health

COMPLETEDPHASE3

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)

NCT01889862•BioMarin Pharmaceutical

View on ClinicalTrials.gov

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.

Eligibility

Age

18 - 70 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Individuals eligible to participate in this study must meet all of the following criteria:
•Have completed a prior BMN 165 study (PAL-003, 165-205, or 165-301) prior to screening
•Have had a stable BMN 165 dose regimen for at least 14 days prior to screening
•Are at least 18 y/o and no older than 70 y/o at screening
•* Subjects who are \< 18 y/o and are already enrolled into Study 165-301 under Amendment #1 (10JAN2014) may enroll into this study
•Has identified a person who is \> 18 y/o who has the neurocognitive and linguistic capacities to comprehend and complete the Profile of Mood States (POMS)-Observer rated scale
•Has identified a competent person(s) \> 18 y/o who can observe the subject during study drug administration at certain points in the study
•* A home healthcare nurse may perform the study drug observations
•Are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any research-related procedures; for minors, parent or guardian provides written consent and assent may be requested
•Are willing and able to comply with all study procedures
+8 more criteria

Exclusion Criteria

•Use of any investigational product (except BMN 165) or investigational medical device within 30 days prior to screening or requirement for any investigational agent prior to completion of all scheduled study assessments
•Use of any medication (except BMN 165) intended to treat PKU, including the use of large neutral amino acids, within 2 days prior to the administration of study drug
•Have known hypersensitivity to Dextran® or components of Dextran
•Use or planned use of any injectable drugs containing PEG (except for BMN 165), including medroxyprogesterone injection, within 3 months prior to screening and during study participation
•Current use of levodopa
•A positive test for HIV antibody, hepatitis B surface antigen, or hepatitis C antibody
•A history of organ transplantation or taking chronic immunosuppressive therapy
•A history of substance abuse in the past 12 months or current alcohol or drug abuse
•Current participation in the Kuvan registry study (PKUDOS). Patients may discontinue the PKUDOS registry trial to allow enrollment in this study
•Pregnant or breastfeeding at screening or planning to become pregnant (self or partner) or breastfeed at any time during the study
+5 more criteria

Locations (29)

University of California, San Diego Clinical and Translational Research Institute

La Jolla, California, United States

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

University of Florida Clinical Research Center

Gainesville, Florida, United States

University of South Florida

Tampa, Florida, United States

Emory Universty, Department of Human Genetics, Division of Medical Genetics

Atlanta, Georgia, United States

Ann and Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Indiana CTSI Clinical Research Center

Indianapolis, Indiana, United States

University of Kentucky Medical Center

Lexington, Kentucky, United States

University of Louisville, Kosair Charities Pediatric Clinical Research Unit

Louisville, Kentucky, United States

Key Dates

Start Date

July 29, 2013

Primary Completion Date

January 13, 2016

Completion Date

February 5, 2019

Last Updated

May 21, 2021

Enrollment

215

ACTUAL participants

Conditions

Phenylketonuria (PKU)

Interventions

BMN165 20mg/day

DRUG

BMN165 40mg/day

DRUG

Placebo

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: May 21, 2021Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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

AAV Gene Therapy Study for Subjects with PKU

A Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With Phenylketonuria

Iron Supplements Effect on B-Phe Levels in PKU Patients During 3 Years Study

PANDA: PKU Amino Acid Evaluation

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency