Natural History Clinical Study in Adult PKU

Recruiting in Palo Alto (17 mi)

+10 other locations

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Waitlist Available

Sponsor: Homology Medicines, Inc

No Placebo Group

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

Inclusion Criteria

Aged 18-55 years at the time of informed consent

Diagnosis of PKU due to PAH deficiency

One plasma Phe value with a concentration of ≥ 600 μmol/L drawn at Screening and at least 1 historical Phe value ≥ 600 μmol/L in the preceding 12 months

Treatment Details

Interventions

• None ()

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

University of Pittsburgh Medical Center- CHOP Pittsburgh, PA

Community Health Clinic Topeka, IN

University of Utah Health Salt Lake City, UT

Children's Hospital of Orange County Orange, CA

More Trial Locations

Who Is Running the Clinical Trial?

Homology Medicines, Inc Lead Sponsor

References