Status:
COMPLETED
Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Phenylketonurias
Eligibility:
All Genders
4-12 years
Phase:
PHASE3
Brief Summary
The primary objective of this trial is to evaluate the ability of Phenoptin to increase phenylalanine (phe) tolerance in children with phenylketonuria who are following a phe-restricted diet.
Eligibility Criteria
Inclusion
- Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by at least one blood Phe measurement \>/=360 umol/L (6 mg/dL)
- Under dietary control with a Phe-restricted diet as evidenced by:· Estimated daily Phe tolerance \</=1000 mg/day
- At least 6 months of blood Phe control (mean level of \</=480 μmol/L) prior to enrolling in the study
- Aged 4 to 12 years inclusive at screening
- A blood Phe level \</=480 μmol/L at screening
- Female subjects of childbearing potential (as determined by the principal investigator) must have a negative blood or urine pregnancy test at entry (prior to the first dose). Note: All female subjects of childbearing potential and sexually mature male subjects must be advised to use a medically accepted method of contraception throughout the study. Female subjects of childbearing potential must be willing to undergo periodic pregnancy tests during the course of the study
- Willing and able to comply with all study procedures
- Willing to provide written assent (if applicable) and written informed consent by a parent or legal guardian after the nature of the study has been explained and prior to any research-related procedures
Exclusion
- Any condition that, in the view of the PI, renders the subject at high risk from treatment compliance and/or completing the study
- Prior history of organ transplantation
- Perceived to be unreliable or unavailable for study participation or have parents or legal guardians who are perceived to be unreliable or unavailable
- Use of any investigational agent within 30 days prior to screening, or requirement for any investigational agent or vaccine prior to completion of all scheduled study assessments
- ALT \> 2 times the upper limit of normal (i.e., Grade 1 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)
Key Trial Info
Start Date :
February 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2006
Estimated Enrollment :
45 Patients enrolled
Trial Details
Trial ID
NCT00272792
Start Date
February 1 2006
End Date
November 1 2006
Last Update
August 13 2015
Active Locations (9)
Enter a location and click search to find clinical trials sorted by distance.
1
Los Angeles, California, United States
2
Oakland, California, United States
3
Sacramento, California, United States
4
San Jose, California, United States