NCT00728676

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Aug 2008

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 30, 2008

Completed
2 days until next milestone

Study Start

First participant enrolled

August 1, 2008

Completed
5 days until next milestone

First Posted

Study publicly available on registry

August 6, 2008

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2010

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2010

Completed
Last Updated

January 18, 2013

Status Verified

January 1, 2013

Enrollment Period

1.6 years

First QC Date

July 30, 2008

Last Update Submit

January 17, 2013

Conditions

Phenylketonuria

Keywords

PKU,Phe,PAH,Kuvan,Trp,Tyr

Outcome Measures

Primary Outcomes (1)

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

    One year

Secondary Outcomes (1)

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

    one year

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Individuals with PKU followed at our clinic

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

LAC+USC Medical Center

Los Angeles, California, 90033, United States

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Shoji Yano, MD, Ph.D.

    University of Southern California

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Director, Genetics Division,

Study Record Dates

First Submitted

July 30, 2008

First Posted

August 6, 2008

Study Start

August 1, 2008

Primary Completion

March 1, 2010

Study Completion

May 1, 2010

Last Updated

January 18, 2013

Record last verified: 2013-01

Locations

US(1)