ACTIVE_NOT_RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The goal of Parts A and B of this Phase 1/2, 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 6 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 2 in participants with PKU (4 weeks)-randomized, double-blind, placebo-controlled * Part E: Phase 2 in participants with PKU (4 weeks) open label * Part F: SAD Phase 1 in healthy participants, randomized, double-blind, placebo-controlled In each part, participants will complete a Screening Period, a Treatment Period, and a Follow-up Period for safety.

Official Title

A Phase 1/2, First-In-Human, Multiple Part, Single Ascending and Multiple Dose Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

Quick Facts

Study Start:2022-10-31
Study Completion:2025-07-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05781399

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.

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

Contacts and Locations

Study Locations (Sites)

University of Florida College of Medicine
Gainesville, Florida, 32610
United States
University of South Florida
Tampa, Florida, 33620
United States
Rare Disease Research
Atlanta, Georgia, 30329
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
University of Nebraska Medical Center
Omaha, Nebraska, 68198
United States
Oregon Health & Sciences University
Portland, Oregon, 97239
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
United States
UT Southwestern Medical Center
Dallas, Texas, 75390
United States
University of Texas Health Science Center at Houston
Houston, Texas, 77030
United States
Utah Health - The University of Utah Hospital
Salt Lake City, Utah, 84112
United States

Collaborators and Investigators

Sponsor: Jnana Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-10-31
Study Completion Date2025-07-31

Study Record Updates

Study Start Date2022-10-31
Study Completion Date2025-07-31

Terms related to this study

Keywords Provided by Researchers

  • PKU

Additional Relevant MeSH Terms

  • Phenylketonuria