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Treatment for Phenylketonuria

N/A
Waitlist Available
Research Sponsored by Homology Medicines, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to week 52
Awards & highlights

Summary

The objective of this study is to characterize the natural history of phenylketonuria (PKU) due to phenylalanine hydroxylase (PAH) deficiency in adults through prospective collection of clinical, cognitive, and quality of life assessments.

Eligible Conditions
  • Phenylketonuria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to week 52
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 52 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Plasma phenylalanine (Phe) concentrations
Plasma tyrosine (Tyr) concentrations
Quality of life (QOL), as assessed using the PKU-QOL questionnaire measures

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Who is running the clinical trial?

Homology Medicines, IncLead Sponsor
5 Previous Clinical Trials
12,034 Total Patients Enrolled
~2 spots leftby Sep 2025
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