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Nutritional and Neurotransmitter Changes in PKU Subjects on BH4

NCT00688844 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 58

Last updated 2015-06-08

Study results available
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Summary

HYPOTHESIS: The investigators hypothesize that KuvanTM therapy could influence nutritional and body composition parameters and neurotransmitter concentrations in pediatric and adult PKU subjects.

SUMMARY: Though the investigators know that KuvanTM lowers blood Phe levels and improves tolerance for natural protein in at least half of the PKU (Phenylketonuria) patient population, investigators do not know the full effects this medicine will have on the patient's diet, or what impact the medicine or diet changes will have on the body composition or nutrient status of PKU patients. Since KuvanTM may also help the body produce neurotransmitters, investigators also want to find out if taking KuvanTM changes neurotransmitter levels in PKU patients, and if PKU patients who are benefitting from KuvanTM feel less stigmatized and have a better outlook on life as a result of the treatment.

Therefore, the research study has several objectives. These are to investigate the impact KuvanTM therapy has on (1) body composition parameters of PKU patients: such as lean body mass, percent body fat, bone density, weight gain, and growth (2) dietary changes, and the effect of those changes, on intake of calories and essential nutrients (3) changes in blood biomarkers of certain nutrients (4) blood and urine neurotransmitter levels, since these changes could indicate improved neurological functioning, (5) and quality of life of PKU patients, who may feel less burdened due to the dietary freedom KuvanTM provides.

Conditions

Interventions

DRUG

KuvanTM Therapy

BH4 treatment was prescribed by each participant's medical provider, not an intervention that was assigned as part of the current study.

Sponsors & Collaborators

  • BioMarin Pharmaceutical

    collaborator INDUSTRY

  • Atlanta Clinical and Translational Science Institute

    collaborator OTHER

  • Emory University

    lead OTHER

Principal Investigators

  • Rani H Singh, PhD, RD · Emory University Department of Human Genetics

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2008-10-31
Primary Completion
2010-10-31
Completion
2010-10-31

Countries

  • United States

Study Locations

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Diseases
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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00688844 on ClinicalTrials.gov