Status:
COMPLETED
Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors
Lead Sponsor:
Shoji Yano
Collaborating Sponsors:
BioMarin Pharmaceutical
Conditions:
Phenylketonuria
Eligibility:
All Genders
18+ years
Brief Summary
To validate the outcome measures and the tolerability of Kuvan treatment in the improvement of behavioral symptoms in 10 selected adults with Phenylketonuria (PKU) with or without mental retardation.
Eligibility Criteria
Inclusion
- Biochemical confirmation of PKU,
- Measurable maladaptive behavior at baseline,
- Signed informed consent,
- Not pregnant or lactating,
- Females on birth control if applicable,
- Willing to comply.
Exclusion
- Subjects or care provider unreliable, use of investigational products within 30 days prior to enrollment,
- Pregnant or lactating,
- Concurrent disease or condition that would interfere with study participation or safety, ie multiple psychotropic meds, any condition the PI renders the subject at high risk, on drugs that inhibit folate synthesis,
- Concurrent use of levodopa.
Key Trial Info
Start Date :
August 1 2008
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 1 2010
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT00728676
Start Date
August 1 2008
End Date
May 1 2010
Last Update
January 18 2013
Active Locations (1)
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1
LAC+USC Medical Center
Los Angeles, California, United States, 90033