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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 October 2017 |
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Main ID: |
NCT01806051 |
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Date of registration:
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04/03/2013 |
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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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A Pilot Study on Diurnal Variation
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Scientific title:
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A Pilot Study on the Diurnal Variation in PKU Patients With Kuvan |
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Date of first enrolment:
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March 2013 |
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Target sample size:
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6 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT01806051 |
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Study type:
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Interventional |
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Study design:
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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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Contacts
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Name:
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Linda M. Randolph, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Children's Hospital Los Angeles |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
PKU PARTICIPANTS (ARM 1):
- Subject has a confirmed diagnosis of PKU with hyperphenylalaninemia documented by a
fasting Phenylalanine level of at least 360 umol/L (6 mg/dL)
- Patient is at least 4 years old (there is no upper age limit for this study)
- Willing and able to provide written authorization or, if under the age of 18 years,
provide written assent (if required) and written patient authorization by a parent or
legal guardian
- Willing to undergo study related procedures including commencing Kuvan treatment for
patient not currently on treatment; temporary discontinuation of Kuvan for patient on
treatment; and completing the 24-Hour Blood Assessment
- Authorized to provide personal health information
- Subjects should not be pregnant and willing to use appropriate birth control during
the study
CONTROL GROUP (ARM 2):
- Healthy, non-PKU individuals. They will be age-sex matched to the PKU group. They may
be a relative (ex: sibling) of a PKU participant, but they don't have to be a blood
relation.
- Individual is at least 4 years old (there is no upper age limit)
- Willing and able to provide written consent or, if under the age of 18 years, provide
written assent and written participant authorization by a parent or legal guardian
- Authorized to provide personal health information
Exclusion Criteria (BOTH ARMS):
- Subjects who do not meet all the inclusion criteria
- Age < 4 years
- Concomitant medical problems or medications which at the discretion of the principal
investigator would put participant at health risk or prevent them from completing
study.
- If female, unwillingness to use birth control during the period of the study drug
administration (this doesn't apply to Arm 2)
Age minimum:
4 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Phenylketonuria (PKU)
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Intervention(s)
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Drug: Kuvan
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Primary Outcome(s)
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Changes in Plasma Phe and Tyrosine Levels
[Time Frame: Baseline and 4 weeks]
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Secondary ID(s)
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CCI-12-00178
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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