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

Shoji Yano10 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Phenylketonuria

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

LAC+USC Medical Center

Los Angeles, California, United States

Outcomes

Primary Outcomes

Pattern of change from baseline to 6 and 12 months in Vineland scale standard scores, evaluated with a repeated measure ANOVA approach.

Time frame: One year

Secondary Outcomes

Correlation and regression methods (non-linear if appropriate) to examine the relationship between changes in behavior and in amino acid profiles.

Time frame: one year

Data from ClinicalTrials.gov

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