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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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12 December 2020 |
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Main ID: |
NCT00730080 |
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Date of registration:
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01/08/2008 |
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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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Sapropterin in Individuals With Phenylketonuria
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Scientific title:
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Effects of Sapropterin on Brain and Cognition in Individuals With Phenylketonuria |
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Date of first enrolment:
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July 2008 |
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Target sample size:
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45 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00730080 |
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Study type:
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Observational |
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Study design:
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Phase:
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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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Desiree White, Ph.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Washington University School of Medicine |
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Name:
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Dorothy K. Grange, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Washington University School of Medicine |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Willing and able to provide informed consent or assent.
- Willing and able to comply with study procedures.
- Greater than or equal to 6 years of age.
- For phenylketonuria,intention of physician to prescribe sapropoterin.
- For phenylketonuria,phenylalanine level greater than or equal to 450µmol/L.
- For phenylketonuria, negative pregnancy test if of childbearing potential.
- For phenylketonuria, willing to use contraception if sexually active.
Exclusion Criteria:
- Pregnant, breastfeeding, or planning to become pregnant during study.
- Use of investigational product less than 30 days prior to or during study.
- Concurrent condition that could interfere with participation or safety.
- Any condition creating high risk of poor compliance with study.
- Perceived to be unreliable or unavailable for study.
- Use of L-Dopa, methotrexate, or other drugs that inhibit folate metabolism.
- For phenylketonuria, known hypersensitivity to sapropterin or excipients.
Age minimum:
6 Years
Age maximum:
50 Years
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: Sapropterin (Kuvan)
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Primary Outcome(s)
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diffusion tensor imaging of the brain
[Time Frame: baseline & follow-ups]
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Secondary Outcome(s)
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list learning task
[Time Frame: baseline & follow-ups]
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n-back task
[Time Frame: baseline & follow-ups]
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structural magnetic resonance imaging of the brain
[Time Frame: baseline & follow-ups]
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Wechsler Abbreviated Scale of Intelligence
[Time Frame: baseline & follow-ups]
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stimulus-response compatibility task
[Time Frame: baseline & follow-ups]
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go/no-go task
[Time Frame: baseline & follow-ups]
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verbal fluency task
[Time Frame: baseline & follow-ups]
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recognition span task
[Time Frame: baseline & follow-ups]
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Secondary ID(s)
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PKU/Kuvan/White- 201104287
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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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