|

AUCD Community Inclusion

AUCD’s Community Inclusion Team supports the University Centers for Excellence in Developmental Disabilities (UCEDD) Network in partnership with the Administration for Community Living.

Learn More
|

AUCD Maternal and Child Health Engagement

AUCD’s Maternal and Child Health Engagement supports the Autism CARES Programs, including LEND and DBP Programs.

Learn More
|

AUCD Public Health

AUCD’s Public Health Team works to create health promotion and public health efforts that include people with disabilities.

Learn More
|

AUCD Emerging Leaders Community

AUCD Emerging Leaders are a part of a community that includes trainees, early career professionals, self-advocates, and family members from AUCD member Centers and Programs.

Learn More

Donate

Project

A Phase 3, Open-Label, Randomized, Multi-Center Study to Assess the Safety and Tolerability of an Induction, Titration...

Center:

TX-LoneStar, LEND

Fiscal Year:

2015

Contact Information:

Deborah Pearson
[email protected]
713-486-2588

Project Description:

The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in patients 18 to 70 years old with hyperphenylalaninemia due to PKU. Study BMN 165-301 is a Phase 3, open-label, randomized study designed to further characterize the safety of BMN 165 during two induction, titration, and maintenance dose regimens in adults with PKU who have not had previous exposure to BMN 165 (naive). Subjects will be randomized (1:1) to titrate up to one of two dose regimens. Other key features of this study are the dose regimens chosen for induction and titration; the study duration; self administration of study drug; and the chosen tertiary objectives.

Keyword(s):

Core Function(s):

Performing Research or Evaluation

Area of Emphasis

Quality Assurance, Education & Early Intervention, Child Care-Related Activities, Health-Related Activities

Target Audience:

Professionals and Para-Professionals, Family Members/Caregivers, Children/Adolescents with Disabilities/SHCN

Unserved or Under-served Populations:

None

Primary Target Audience Geographic Descriptor:

Single-County

Funding Source: