NCT00827762

Brief Summary

The purpose of this study is to determine whether improvements in behavior occur in children with phenylketonuria (PKU) who are taking Kuvan.

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
2

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jan 2009

Shorter than P25 for all trials

Geographic Reach
1 country

4 active sites

Status
terminated

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

Study Start

First participant enrolled

January 1, 2009

Completed
20 days until next milestone

First Submitted

Initial submission to the registry

January 21, 2009

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 23, 2009

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2010

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2010

Completed
Last Updated

December 5, 2013

Status Verified

December 1, 2013

Enrollment Period

1 year

First QC Date

January 21, 2009

Last Update Submit

December 3, 2013

Conditions

Phenylketonuria

Keywords

phenylketonuriaKuvansapropterinbehaviorcognitionexecutive abilitiesattention

Outcome Measures

Primary Outcomes (1)

  • Behavior Rating Inventory of Executive Function (BRIEF)

    baseline, 4-week follow-up, 24-week follow-up

Secondary Outcomes (4)

  • Behavior Assessment System for Children - Second Edition (BASC-2)

    baseline, 4-week follow-up, 24-week follow-up

  • Conners 3rd Edition (Conners 3)

    baseline, 4-week follow-up, 24-week follow-up

  • Conners Continuous Performance Test II Version 5 (CCPT-II Version 5)

    baseline, 4-week follow-up, 24-week follow-up

  • Matrix Reasoning subtest of the Wechsler Abbreviated Scale of Intelligence (WASI)

    baseline, 4-week follow-up, 24-week follow-up

Study Arms (1)

Phenylketonuria

Individuals with mild phenylketonuria/hyperphenylalanemia who are beginning treatment with Kuvan.

Drug: Kuvan

Interventions

KuvanDRUG

20/mg/kg/day taken once daily or as otherwise prescribed by physician as standard care.

Also known as: Sapropterin
Phenylketonuria

Eligibility Criteria

Age6 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Primary care clinic for phenylketonuria.

You may qualify if:

  • Willing and able to provide informed consent and/or assent.
  • Willing and able to comply with study procedures.
  • Between 6 and 18 years of age, inclusive.
  • Intention of physician to prescribe Kuvan.
  • Phenylalanine levels between 360μmol/L and 600μmol/L, inclusive, when untreated with dietary restrictions.
  • Negative pregnancy test if of childbearing potential.
  • Willing to use contraception if sexually active.

You may not qualify if:

  • Treatment with Kuvan within the past 6 months.
  • Pregnant, breastfeeding, or planning to become pregnant during study.
  • Use of investigational product less than 30 days prior to or during study.
  • Concurrent condition that could interfere with participation or safety.
  • Any condition creating high risk of poor compliance with study.
  • History of major medical disorder unrelated to phenylketonuria.
  • Perceived to be unreliable or unavailable for study.
  • Use of L-Dopa, methotrexate, or other drugs that inhibit folate metabolism.
  • Known hypersensitivity to sapropterin or excipients.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Northwestern University/Children's Memorial Hospital

Chicago, Illinois, 60614, United States

Location

University of Missouri

Columbia, Missouri, 65211, United States

Location

Washington University

St Louis, Missouri, 63130, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Related Publications (3)

  • Christ SE, Steiner RD, Grange DK, Abrams RA, White DA. Inhibitory control in children with phenylketonuria. Dev Neuropsychol. 2006;30(3):845-64. doi: 10.1207/s15326942dn3003_5.

    PMID: 17083296BACKGROUND

  • White DA, Nortz MJ, Mandernach T, Huntington K, Steiner RD. Age-related working memory impairments in children with prefrontal dysfunction associated with phenylketonuria. J Int Neuropsychol Soc. 2002 Jan;8(1):1-11.

    PMID: 11843066BACKGROUND

  • White DA, Nortz MJ, Mandernach T, Huntington K, Steiner RD. Deficits in memory strategy use related to prefrontal dysfunction during early development: evidence from children with phenylketonuria. Neuropsychology. 2001 Apr;15(2):221-9. doi: 10.1037//0894-4105.15.2.221.

    PMID: 11324865BACKGROUND

MeSH Terms

Conditions

PhenylketonuriasBehavior

Interventions

sapropterin

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

  • Desiree White, Ph.D.

    Washington University School of Medicine

    PRINCIPAL INVESTIGATOR

  • Dorothy K. Grange, M.D.

    Washington University School of Medicine

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 21, 2009

First Posted

January 23, 2009

Study Start

January 1, 2009

Primary Completion

January 1, 2010

Study Completion

January 1, 2010

Last Updated

December 5, 2013

Record last verified: 2013-12

Locations

US(4)