Status:
COMPLETED
Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Phenylketonurias
Eligibility:
All Genders
8+ years
Phase:
PHASE2
Brief Summary
The primary objective is to evaluate the degree and frequency of response to Phenoptin™ (sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine (Phe) level among subjects ...
Eligibility Criteria
Inclusion
- Age \>/= 8 years
- Blood Phe level \>/= 450 umol/L at screening
- Clinical diagnosis of PKU with hyperphenylalaninemia documented by past medical history of at least one blood Phe measurement \>/= 360 umol/L (6 mg/dL)
- 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 (non-sterile females of child-bearing potential only)
- Male and Female subjects of childbearing potential childbearing potential (if sexually active and non-sterile) 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
Exclusion
- 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 within 30 days prior to screening, or requirement for any investigational agent or 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)
- Serious neuropsychiatric illness (e.g., major depression) not currently under medical control
- Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)
- Concurrent use of levodopa
- Clinical diagnosis of primary BH4 deficiency
Key Trial Info
Start Date :
December 1 2004
Trial Type :
INTERVENTIONAL
End Date :
November 1 2005
Estimated Enrollment :
700 Patients enrolled
Trial Details
Trial ID
NCT00104260
Start Date
December 1 2004
End Date
November 1 2005
Last Update
April 9 2007
Active Locations (13)
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1
Los Angeles, California, United States
2
Oakland, California, United States
3
New Haven, Connecticut, United States
4
Chicago, Illinois, United States