A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria
Otsuka Pharmaceutical Development & Commercialization, Inc.
Summary
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
Eligibility
- Age range
- 12–17 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Males and females 12 to less than 18 years of age, inclusive on Day 1. 2. Clinical diagnosis of PKU. 3. Ability to swallow tablets. 4. Average of 2 plasma Phe levels during the Screening period greater than 360 μM and no plasma Phe level less than 300 μM. 5. Body weight equal or greater than 45 kg and body mass index less than 40 kg/m2. 6. Females of childbearing potential must practice sexual abstinence or agree to use 2 highly effective contraceptive methods. 7. Capable of giving signed informed consent (emancipated minors) or parent/legal guardian to provide info…
Interventions
- DrugJNT-517 Tablet
JNT-517: 75 mg BID
- OtherPlacebo
Placebo Tablet: BID
- DrugJNT-517 Tablet
JNT-517: 150 mg BID
Locations (7)
- University of Florida (UF) Health Shands HospitalGainesville, Florida
- University of South FloridaTampa, Florida
- Oregon Health and Science UniversityPortland, Oregon
- Children's Medical Center DallasDallas, Texas
- University of Texas Health (UTHealth) Science Center at HoustonHouston, Texas
- Utah Health - The University of Utah HospitalSalt Lake City, Utah