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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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4 October 2021 |
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Main ID: |
NCT00244218 |
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Date of registration:
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25/10/2005 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Response to Phenylketonuria to Tetrahydrobiopterin (BH4)
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Scientific title:
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Response to Phenylketonuria to Tetrahydrobiopterin (BH4) |
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Date of first enrolment:
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April 2005 |
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Target sample size:
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57 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT00244218 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Reuben Matalon, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Texas |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- 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 Criteria:
- 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
Age minimum:
10 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Phenylketonuria
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Intervention(s)
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Drug: tetrahydrobiopterin (BH4)
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Primary Outcome(s)
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Blood Phe level decrease by 30%
[Time Frame: 9 months]
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Secondary ID(s)
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01-269
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FD-R-002600-01
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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