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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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19 February 2015 |
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Main ID: |
NCT00484991 |
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Date of registration:
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08/06/2007 |
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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 Expanded Access Program
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Scientific title:
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Sapropterin Expanded Access Program |
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Date of first enrolment:
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February 2007 |
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Target sample size:
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Recruitment status: |
Approved for marketing |
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URL:
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http://clinicaltrials.gov/show/NCT00484991 |
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Study type:
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Expanded Access |
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Study design:
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N/A
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Phase:
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N/A
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Countries of recruitment
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United States
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Patient has hyperphenylalaninemia due to PKU, a rare and serious disease
2. Patient is not participating in a sapropterin dihydrochloride clinical study
3. Patient is older than 8 years of age
4. Patient is willing and able to provide written informed consent or, in the case of
under the age of 18, provide written assent (if required) and written informed
consent by a parent or legal guardian
5. If female and of child bearing potential, the patient has a negative urine pregnancy
test within 24 hours prior to enrollment (females of child-bearing potential only)
and will be using adequate contraceptive methods to avoid pregnancy while
participating in the program
6. Patient is willing and able to comply with program procedures
7. Patient lives in the United States
Exclusion Criteria:
1. Patient is perceived to be unreliable or unwilling to comply with program
participation or, if under the age of 18, have parents or legal guardians who are
perceived to be unreliable or unwilling to comply with program participation
2. Patient has a concurrent disease or condition that would interfere with program
participation or safety
3. Patient is 8 years old or younger
4. Patients is eligible for enrolling in PKU-010
5. Patient is participating in an ongoing study with sapropterin dihydrochloride
6. Patient is pregnant, breast feeding or considering pregnancy
7. Patient is taking levodopa
Age minimum:
9 Years
Age maximum:
N/A
Gender:
Both
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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 dihydrochloride
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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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