Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
BioMarin Pharmaceutical
Age
8–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
8 years of age and older
Received at least 7 out of 8 scheduled doses in Study PKU 001
Responsive to Phenoptin™ in Study PKU-001, defined as a reduction in blood Phenylalanine level of \>/=30% compared with baseline
Blood Phenylalanine level \>/=450 μmol/L at screening
Willing and able to provide written informed consent or, in the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian, after the nature of the study has been explained
Negative urine pregnancy test at screening (females of child-bearing potential)
Male and Female subjects of childbearing potential (if sexually active) must be using acceptable birth control measures, as determined by the investigator, and willing to continue to use acceptable birth control measures while participating in the study
Willing and able to comply with study procedures
Willing to continue current diet unchanged while participating in the study
You may not be if
Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable
Use of any investigational agent other than Phenoptin™ within 30 days prior to screening, or requirement for any investigational agent or investigational vaccine prior to completion of all scheduled study assessments
Pregnant or breastfeeding, or considering pregnancy
ALT \>5 times the upper limit of normal (i.e., Grade 3 or higher based on World Health Organization Toxicity Criteria) at screening
Concurrent disease or condition that would interfere with study participation or safety (e.g., seizure disorder, oral steroid-dependent asthma or other condition requiring oral or parenteral corticosteroid administration, or insulin-dependent diabetes, or organ transplantation recipient)
Serious neuropsychiatric illness (e.g., major depression) not currently under medical management
Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)
