First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

Launched by OTSUKA PHARMACEUTICAL DEVELOPMENT & COMMERCIALIZATION, INC. · Mar 10, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new medication called JNT-517 to see how safe it is and how well it works for people with a condition called phenylketonuria (PKU), as well as for healthy volunteers. The trial has four parts: the first two parts involve giving different doses of the medication to healthy participants to see how well they tolerate it; the third part compares two forms of the medication—tablet and liquid—and examines how food affects its absorption. Finally, the fourth part focuses specifically on participants with PKU to assess the medication's safety and effects on their amino acid levels.

To participate, individuals usually need to be between 18 to 65 years old, healthy or diagnosed with PKU, and meet certain health criteria, such as having a body mass index (BMI) within a specific range. Participants will be monitored throughout the study, which includes a screening phase, a treatment period, and follow-up checks to ensure their safety. This trial is currently recruiting participants, and it’s important for those interested to discuss their eligibility with the study team.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• Parts A, B, C, and F:
• • 1. Males and females 18 to 55 years of age.
• • 2. Medically healthy with no clinically significant medical history.
• • 3. Body mass index (BMI) of 18-40 kg/m2 and total body weight \>50 kg (110 lbs).
• • 4. Non-smoker for at least 2 weeks prior to dosing and willing to abstain during the study.
• Part D and E:
• • 5. Males and females 18 to 65 years of age, inclusive.
• • 6. Diagnosis of PKU with a confirmed genotype.
• • 7. At least 2 plasma Phe levels \>600 μM over the past 12 months.
• • 8. BMI of 18-40 kg/m2.
• All Parts:
• • 9. Females of childbearing potential must agree to use 2 highly effective contraceptive methods.
• • 10. Capable of giving signed informed consent and able to comply with study procedures.
• Key Exclusion Criteria:
• All Parts:
• • 1. 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.
• • 2. Positive for hepatitis B or C or human immunodeficiency virus.
• • 3. Any history of malignancy in the last 5 years, excluding non-melanoma skin cancer.
• • 4. Any history of liver disease.
• • 5. Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion.
• • 6. Participation in another investigational drug trial within 30 days or, if known, 5 half-lives of the investigational drug (whichever is longer).
• • 7. History of drug/alcohol abuse in the last year.
• • 8. Current, recent, or suspected infection within 4 weeks of Screening of SARS-CoV-2/COVID-19.
• • 9. Received a vaccine for SARS-CoV-2/COVID-19 within 14 days of Screening.
• • 10. Unable to tolerate oral medication.
• • 11. Allergy to JNT-517 or any component of the investigational product.
• • 12. Received \>50 mL of blood or plasma within 30 days of Screening or \>500 mL of blood or plasma within 60 days of Screening.