Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors

Phenylketonuria Clinical Trial

Official title:

Pilot Study to Evaluate the Effects of Kuvan on Adult Individuals With Phenylketonuria With Measurable Maladaptive Behaviors

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00728676
• Other study ID #: BioMarin-300
• Status: Completed
• Phase: N/A
• First received: July 30, 2008
• Last updated: January 17, 2013
• Start date: August 2008
• Est. completion date: May 2010

Study information

• Verified date: January 2013
• Source: University of Southern California
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: May 2010
• Est. primary completion date: March 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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 Criteria:

• 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.

Study Design

Observational Model: Case Control, Time Perspective: Prospective

Related Conditions & MeSH terms

• Phenylketonuria
• Phenylketonurias

Locations

Country	Name	City	State
United States	LAC+USC Medical Center	Los Angeles	California

Sponsors (2)

Lead Sponsor	Collaborator
Shoji Yano	BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Pattern of change from baseline to 6 and 12 months in Vineland scale standard scores, evaluated with a repeated measure ANOVA approach.		One year	No
Secondary	Correlation and regression methods (non-linear if appropriate) to examine the relationship between changes in behavior and in amino acid profiles.		one year	No