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

NCT ID: NCT00728676

Last Updated: 2013-01-18

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 10 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2008-08-31
• Study Completion Date: 2010-05-31

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.

Conditions

Phenylketonuria

Study Design

• Observational Model Type: CASE_CONTROL
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

BioMarin Pharmaceutical

INDUSTRY

Sponsor Role: collaborator

Shoji Yano

OTHER

Sponsor Role: lead

Responsible Party

Shoji Yano

Director, Genetics Division,

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Shoji Yano, MD, Ph.D.

Role: PRINCIPAL_INVESTIGATOR

University of Southern California

Locations

LAC+USC Medical Center

Los Angeles, California, United States

Countries

United States

Other Identifiers

BioMarin-300

Identifier Type: -

Identifier Source: org_study_id