First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria
Phenylketonuria
About this trial
This is an interventional treatment trial for Phenylketonuria focused on measuring PKU
Eligibility Criteria
Key Inclusion Criteria: Parts A, B, and C: Males and females 18 to 55 years of age. Medically healthy with no clinically significant medical history. Body mass index (BMI) of 18-40 kg/m2 and total body weight >50 kg (110 lbs). Non-smoker for at least 2 weeks prior to dosing and willing to abstain during the study. Part D: Males and females 18 to 65 years of age, inclusive. Diagnosis of PKU with a confirmed genotype. At least 2 plasma Phe levels >600 μM over the past 12 months. BMI of 18-40 kg/m2. All Parts: Females of childbearing potential must agree to use 2 highly effective contraceptive methods. Capable of giving signed informed consent and able to comply with study procedures. Key Exclusion Criteria: All Parts: Any acute or chronic medical condition that would prevent the participant from complying with the procedures or place the participant at risk if they participate in the study. Positive for hepatitis B or C or human immunodeficiency virus. Any history of malignancy in the last 5 years, excluding non-melanoma skin cancer. Any history of liver disease. Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion. Participation in another investigational drug trial within 30 days or, if known, 5 half-lives of the investigational drug (whichever is longer). History of drug/alcohol abuse in the last year. Current, recent, or suspected infection within 4 weeks of Screening of SARS-CoV-2/COVID-19. Received a vaccine for SARS-CoV-2/COVID-19 within 14 days of Screening. Unable to tolerate oral medication. Allergy to JNT-517 or any component of the investigational product. Received >50 mL of blood or plasma within 30 days of Screening or >500 mL of blood or plasma within 60 days of Screening.
Sites / Locations
- University of Florida College of MedicineRecruiting
- University of South FloridaRecruiting
- Rare Disease ResearchRecruiting
- Ann & Robert H. Lurie Children's Hospital of ChicagoRecruiting
- Oregon Health & Sciences UniversityRecruiting
- Children's Hospital of PhiladelphiaRecruiting
- UPMC Children's Hospital of PittsburghRecruiting
- UT Southwestern Medical CenterRecruiting
- Nucleus Network MelbourneRecruiting
- Westmead HospitalRecruiting
- Mater Misericordia LtdRecruiting
- Royal Adelaide HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
JNT-517 SAD (Part A)
JNT-517 MAD (Part B)
JNT-517 Suspension Then Tablet Fasted Then Tablet Fed (Part C)
JNT-517 Tablet Fasted Then Tablet Fed Then Suspension (Part C)
JNT-517 Tablet Fed Then Suspension Then Tablet Fasted (Part C)
JNT-517 PKU (Part D)
Single dose of JNT-517 or placebo in fasted state.
JNT-517 or placebo once or twice daily for 14 days, with first daily dose given after an overnight fast.
Single dose of JNT-517 suspension, JNT-517 tablet in a fasted state, and JNT-517 tablet in a fed state in a sequential, open-label manner. Each treatment is separated by a minimum of 5 half-lives.
Single dose of JNT-517 tablet in a fasted state, JNT-517 tablet in a fed state, and JNT-517 suspension in a sequential, open-label manner. Each treatment is separated by a minimum of 5 half-lives.
Single dose of JNT-517 tablet in a fed state, JNT-517 suspension, and JNT-517 tablet in a fasted state in a sequential, open-label manner. Each treatment is separated by a minimum of 5 half-lives.
JNT-517 or placebo daily for 4 weeks. Dose is based on data from Parts A, B, and C.