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

Description

The goal of Parts A and B of this Phase 1, first-in-human, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of single (SAD) and multiple (MAD) ascending doses of oral JNT-517 in healthy participants. In Part C, the goal is to evaluate the differences in bioavailability between a tablet and suspension formulation of JNT-517 and the food effect in healthy volunteers. All participants in Part C will receive JNT-517. The goal of Part D is to assess the safety, tolerability, PK, and effect on urinary Phe and other amino acids of JNT-517 in participants with phenylketonuria (PKU). Participants in Part D will receive either JNT-517 or placebo and will be blinded to their treatment assignment. The study consists of 4 parts: * Part A: SAD in healthy participants -randomized, double-blind, placebo-controlled * Part B: MAD in healthy participants (14 days)-randomized, double-blind, placebo-controlled * Part C: Relative bioavailability of 2 formulations and food effect in healthy participants-randomized, open-label * Part D: Phase 1b in participants with PKU (4 weeks)-randomized, double-blind, placebo-controlled In each part, participants will complete a Screening Period, a Treatment Period, and a Follow-up Period for safety.

Conditions

Phenylketonuria

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Study Overview

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Study Details

Study overview

The goal of Parts A and B of this Phase 1, first-in-human, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of single (SAD) and multiple (MAD) ascending doses of oral JNT-517 in healthy participants. In Part C, the goal is to evaluate the differences in bioavailability between a tablet and suspension formulation of JNT-517 and the food effect in healthy volunteers. All participants in Part C will receive JNT-517. The goal of Part D is to assess the safety, tolerability, PK, and effect on urinary Phe and other amino acids of JNT-517 in participants with phenylketonuria (PKU). Participants in Part D will receive either JNT-517 or placebo and will be blinded to their treatment assignment. The study consists of 4 parts: * Part A: SAD in healthy participants -randomized, double-blind, placebo-controlled * Part B: MAD in healthy participants (14 days)-randomized, double-blind, placebo-controlled * Part C: Relative bioavailability of 2 formulations and food effect in healthy participants-randomized, open-label * Part D: Phase 1b in participants with PKU (4 weeks)-randomized, double-blind, placebo-controlled In each part, participants will complete a Screening Period, a Treatment Period, and a Follow-up Period for safety.

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

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

Condition
Phenylketonuria
Intervention / Treatment

-

Contacts and Locations

Gainesville

University of Florida College of Medicine, Gainesville, Florida, United States, 32610

Tampa

University of South Florida, Tampa, Florida, United States, 33620

Atlanta

Rare Disease Research, Atlanta, Georgia, United States, 30329

Chicago

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Omaha

University of Nebraska Medical Center, Omaha, Nebraska, United States, 68198

Portland

Oregon Health & Sciences University, Portland, Oregon, United States, 97239

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Pittsburgh

UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States, 15224

Dallas

UT Southwestern Medical Center, Dallas, Texas, United States, 75390

Houston

University of Texas Health Science Center at Houston, Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 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.
  • 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.
  • 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.
  • 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.

Ages Eligible for Study

18 Years to 65 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

Yes

Collaborators and Investigators

Jnana Therapeutics,

Study Record Dates

2025-03