The goal of this Phase 2, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adolescents (12 to less than 18 years of age) with PKU. Participants will receive either JNT-517 or placebo and will be blinded to their treatment assignment. Participants will have a 4 in 5 (or 80%) chance of receiving JNT-517. The study will last for up to 63 days including a Screening period, Treatment period and Follow-up period for safety. Participants will: * Take 75 mg JNT-517 or a placebo BID (2x per day) for 28 days * Visit the clinic or have a mobile health nurse visit your home for checkups and tests * Collect urine sample at home and bring to clinic on specified days * Keep a food diary 3 days before each study visit
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
14
JNT-517: 75 mg BID
Placebo Tablet: BID
JNT-517: 150 mg BID
University of Florida (UF) Health Shands Hospital
Gainesville, Florida, United States
University of South Florida
Tampa, Florida, United States
Oregon Health and Science University
Portland, Oregon, United States
Children's Medical Center Dallas
Dallas, Texas, United States
University of Texas Health (UTHealth) Science Center at Houston
Houston, Texas, United States
Utah Health - The University of Utah Hospital
Salt Lake City, Utah, United States
Murdoch Children's Research Institute
Parkville, Melbourne, Victoria, Australia
Number of participants with treatment-emergent adverse events (TEAEs)
Reported based on results of 12-lead electrocardiograms (ECGs), vital signs and clinical laboratory tests.
Time frame: Screening to Study Completion (Average of 9 weeks)
Plasma AUC (area under the concentration-time curve)
Time frame: Screening to Study Completion (Average of 9 weeks)
Cmax (maximum observed concentration)
Time frame: Screening to Study Completion (Average of 9 weeks)
Tmax (time to Cmax)
Time frame: Screening to Study Completion (Average of 9 weeks)
Change over time in plasma phenylalanine (Phe) and other amino acids
Time frame: Screening to Study Completion (Average of 9 weeks)
Change over time in urinary phenylalanine (Phe) and other amino acids
Time frame: Screening to Study Completion (Average of 9 weeks)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.