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Clinical Study

PTC923-MD-003: A Study Drug for Patients with Phenylketonuria (PKU)

PKU (Phenylketonuria) is caused by a defective gene. When a person with PKU eats foods that contain protein, or eats aspartame, an artificial sweetener, a dangerous buildup of phenylalanine can occur. PTC923 is a study medication to reduce blood Phenylalanine (Phe) levels. This study will assess the safety and how effective this medication is for those of any age with a PKU diagnosis.


For more information contact:

Carrie Bailey

IRB#: IRB_00150374 | PI: Nicola Longo | Department: PEDIATRIC GENETICS | Approval Date: 2022-07-27 06:00:00
Specialties: Pediatric Genetics

Who can participate?

 Gender: All

 Age: All Ages

 Volunteers: Volunteers with special conditions

 Location: In Person

Inclusion Criteria:

  • Ages 0 years and older
  • Diagnosis of PKU
  • Participants of childbearing age must use specific birth control

Exclusion Criteria:

  • Problems with your stomach or intestines
  • History of stomach surgery
  • Pregnant

Will I be paid for my time?


Last Updated: 3/20/23