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Phenylketonuria for Phenylketonuria

N/A
Waitlist Available
Led By Desiree White, Ph.D.
Research Sponsored by Washington University School of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
All Individual Drugs Already Approved
No Placebo-Only Group

Summary

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

Eligible Conditions
  • Phenylketonuria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: PhenylketonuriaExperimental Treatment1 Intervention
Individuals with mild phenylketonuria/hyperphenylalanemia who are beginning treatment with Kuvan.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sapropterin
FDA approved

Find a Location

Who is running the clinical trial?

Washington University School of MedicineLead Sponsor
1,991 Previous Clinical Trials
2,295,882 Total Patients Enrolled
BioMarin PharmaceuticalIndustry Sponsor
159 Previous Clinical Trials
115,072 Total Patients Enrolled
University of Missouri-ColumbiaOTHER
379 Previous Clinical Trials
628,492 Total Patients Enrolled
~0 spots leftby Nov 2025
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