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Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria

NCT01412437 · Status: WITHDRAWN · Phase: NA · Type: INTERVENTIONAL

Last updated 2014-12-17

No results posted yet for this study

Summary

The investigators will use different types of brain imaging (MRI) in patients with Phenylketonuria (PKU) who are currently not on a strict diet to test the hypothesis that there is improvement in brain circuitry and biochemistry after return to diet and/or sapropterin dihydrochloride (Kuvan).

Conditions

  • PKU

Interventions

DIETARY_SUPPLEMENT

diet

12 participants will be randomized to diet. Phe levels will be followed by blood levels. A dietician will analyze diet for phe content and advise

DRUG

sapropterin dihydrochloride

20 mg/kg for 4 months

DRUG

sapropterin dihydrochloride

Sponsors & Collaborators

  • Georgetown University

    collaborator OTHER

  • Children's National Research Institute

    lead OTHER

Principal Investigators

  • Andrea L Gropman, M.D. · Children's National Research Institute

Study Design

Allocation
RANDOMIZED
Purpose
DIAGNOSTIC
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
40 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-04-30
Primary Completion
2012-03-31
Completion
2012-12-31

Countries

  • United States

Study Locations

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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 NCT01412437 on ClinicalTrials.gov