An Open-Label Study to Evaluate the Long-Term Safety of JNT-517 in Participants With Phenylketonuria
Otsuka Pharmaceutical Development & Commercialization, Inc.
Summary
The goal of this Phase 3, open-label study is to evaluate the long-term safety of JNT-517 in pediatric and adult participants with Phenylketonuria (PKU) after completion of either Study JNT517-101 (NCT05781399) or JNT517-201 (NCT06637514) as well as participants who have not participated in a prior JNT-517 study. In this trial, all participants will receive JNT-517 using age- and weight-banded dosing as outlined in the protocol, regardless of any dose received in a previous study.
Eligibility
- Age range
- 4+ years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Diagnosis of phenylketonuria (ie, PAH deficiency) by either molecular testing or biochemical criteria consistent with the applicable regional guidelines. 2. Participants 4 years of age and older, inclusive, at time of Screening. 3. Not on pegvaliase within 4 weeks of Screening. 4. Not on sepiapterin within 2 weeks of Screening. 5. If on sapropterin or large neutral amino acids at Screening, must be on a stable dose for 4 weeks prior to Screening. 6. Willing and able to maintain a diet consistent in Phe content from the Screening period through the duration of the st…
Interventions
- DrugJNT-517
JNT-517 administered orally twice daily using age- and weight-banded dosing.
Locations (12)
- University of Florida (UF) Health Shands HospitalGainesville, Florida
- University of South FloridaTampa, Florida
- Oregon Health and Science UniversityPortland, Oregon
- University of Pittsburgh Medical Center (UPMC) - Children's Hospital of PittsburghPittsburgh, Pennsylvania
- Children's Medical Center DallasDallas, Texas
- University of Texas Southwestern Medical CenterDallas, Texas