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
10
JNT-517: 75 mg BID
Placebo Tablet: BID
JNT-517: 150 mg BID
University of Texas Southwestern Medical Center
Dallas, Texas, United States
RECRUITINGNumber 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)
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