Status:
TERMINATED
Response to Phenylketonuria to Tetrahydrobiopterin (BH4)
Lead Sponsor:
The University of Texas Medical Branch, Galveston
Conditions:
Phenylketonuria
Eligibility:
All Genders
10+ years
Phase:
PHASE1
Brief Summary
The purpose of this study is to determine whether tetrahydrobiopterin (BH4)is effective in treating patients with PKU.
Detailed Description
Phenylketonuria(PKU) is an autosomal recessive disorder caused by a defect in the enzyme phenylalanine hydroxylase(PAH). this incidence of PKU in the US is about 1:15,000 births. The disease is pan et...
Eligibility Criteria
Inclusion
- Subject and/or parent or guardian must be capable of understanding and providing written informed consent
- Subjects must have Phenylketonuria (PKU)or hyperphenylalaninemia (HPA), defined as baseline blood Phe levels of \>600 umol/L
- Subjects must be at least 10 years of age, and may be of either gender and any ethnic group
- Female subjects of childbearing potential must agree to use adequate birth control or refrain from sexual activity throughout study participation
Exclusion
- Female subjects who are pregnant or breastfeeding
- Subjects who have concurrent diseases or conditions that require medication or treatment
- Subjects who require concomitant treatment with any drug known to inhibit folate synthesis
- Subjects who have been treated with any investigational drug within 30 days
Key Trial Info
Start Date :
April 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2015
Estimated Enrollment :
57 Patients enrolled
Trial Details
Trial ID
NCT00244218
Start Date
April 1 2005
End Date
January 1 2015
Last Update
September 21 2021
Active Locations (1)
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1
University of Texas Medical Branch
Galveston, Texas, United States, 77555