Testing JNT-517 in healthy individuals and those with Phenylketonuria

A Phase 1, First-In-Human, Multiple Part, Single Ascending and Multiple Dose Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

This study is testing a new drug called JNT-517 to see how safe it is and how it works in both healthy people and those with Phenylketonuria (PKU).

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 112 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Jnana Therapeutics (industry)
• Locations: 15 sites (Gainesville, Florida and 14 other locations)
• Trial ID: NCT05781399 on ClinicalTrials.gov

What this trial studies

This Phase 1 clinical study evaluates the safety, tolerability, and pharmacokinetics of JNT-517 through multiple parts involving healthy participants and individuals diagnosed with Phenylketonuria (PKU). The study includes single and multiple ascending doses of JNT-517 in healthy volunteers, as well as a comparison of different formulations and their effects when taken with food. In the final part, the study assesses the impact of JNT-517 on urinary phenylalanine and other amino acids in PKU patients, with participants receiving either the drug or a placebo in a blinded manner.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 to 65 with a confirmed diagnosis of Phenylketonuria and specific plasma phenylalanine levels.

Not a fit: Patients with acute or chronic medical conditions that could complicate participation or those with a history of hepatitis B or C will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to a new treatment option for individuals with Phenylketonuria, potentially improving their metabolic control.

How similar studies have performed: While this approach is novel in its specific application to PKU, similar studies evaluating pharmacokinetics and safety of new treatments have shown promise.

Eligibility criteria

Key Inclusion Criteria:

Parts A, B, and C:

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:
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.

Where this trial is running

Gainesville, Florida and 14 other locations

• University of Florida College of Medicine — Gainesville, Florida, United States (RECRUITING)
• University of South Florida — Tampa, Florida, United States (RECRUITING)
• Rare Disease Research — Atlanta, Georgia, United States (RECRUITING)
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (RECRUITING)
• University of Nebraska Medical Center — Omaha, Nebraska, United States (RECRUITING)
• Oregon Health & Sciences University — Portland, Oregon, United States (RECRUITING)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (RECRUITING)
• UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (RECRUITING)
• UT Southwestern Medical Center — Dallas, Texas, United States (RECRUITING)
• University of Texas Health Science Center at Houston — Houston, Texas, United States (RECRUITING)
• Utah Health - The University of Utah Hospital — Salt Lake City, Utah, United States (RECRUITING)
• Nucleus Network Melbourne — Melbourne, Melbourne VIC, Australia (RECRUITING)
• Westmead Hospital — Westmead, New South Wales, Australia (RECRUITING)
• Mater Misericordia Ltd — South Brisbane, Queensland, Australia (RECRUITING)
• Royal Adelaide Hospital — Adelaide, South Australia, Australia (RECRUITING)

Study contacts

• Study coordinator: Toby Vaughn

Conditions: Phenylketonuria, PKU