A Three-Part, Phase 3, Randomized, Double-Blind, Placebo-Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneo
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 adults with PKU.Primary Outcome Measures:
Incidence of adverse events experienced by subjects. [ Time Frame: Weekly throughout study. ] [ Designated as safety issue: Yes ]
Subjects will be assessed weekly for adverse events, including laboratory abnormalities.
Secondary Outcome Measures:
Cognitive and mood symptoms BMN 165 [ Time Frame: Throughout the study ] [ Designated as safety issue: No ]
ADHD-RS IV and the POMS (Profile of Mood States) will be used to evaluate cognitive and mood symptoms.
Plasma Phenylalanine (phe) levels [ Time Frame: Throughout the study ] [ Designated as safety issue: No ]
Other Outcome Measures:
Protein intake as reported by subjects in diet diary [ Time Frame: Every 4 weeks, an expected average of 3 years ] [ Designated as safety issue: No ]
Diet diary collected monthly to assess protein intake from medical food and from natural protein.
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
COVID-19 Related Data:
N/A
