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New treatment option for Phenylketonuria (PKU)
Official title A Long-Term Study of JNT-517 in Participants With Phenylketonuria
ClinicalTrials.gov ID: NCT06628128
What this study is testing
What is JNT-517?
JNT-517 is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for phenylketonuria (pku).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The goal of this Phase 3, open-label study is to evaluate the long-term safety of JNT-517 in pediatric and adult participants with Phenylketonuria (PKU) after completion of either Study JNT517-101 (NCT05781399) or JNT517-201 (NCT06637514) as well as participants who have not participated in a prior JNT-517 study. In this trial, all participants will receive JNT-517 using age- and weight-banded dosing as outlined in the protocol, regardless of any dose received in a previous study.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- Diagnosis of phenylketonuria (ie, PAH deficiency) by either molecular testing or biochemical criteria consistent with the applicable regional...
- Participants 4 years of age and older, inclusive, at time of Screening.
- Not on pegvaliase within 4 weeks of Screening.
- Not on sepiapterin within 2 weeks of Screening.
- If on sapropterin or large neutral amino acids at Screening, must be on a stable dose for 4 weeks prior to Screening.
You likely can't join if
- Participation in this study is not considered safe and/or feasible in the opinion of the Investigator.
- Participants have not completed a previous JNT-517 study and are eligible for another active JNT-517 trial at the site, unless approval is obtained...
- Any acute or chronic medical condition that would prevent the participant from complying with the procedures or place the participant at risk if they...
- Positive for hepatitis B or C or human immunodeficiency virus.
- Any history of significant liver disease.
- Any history of cataracts or more than minimal cataracts observed during the Screening ophthalmologic examination.
See the full eligibility criteria
Who can join
- Diagnosis of phenylketonuria (ie, PAH deficiency) by either molecular testing or biochemical criteria consistent with the applicable regional guidelines.
- Participants 4 years of age and older, inclusive, at time of Screening.
- Not on pegvaliase within 4 weeks of Screening.
- Not on sepiapterin within 2 weeks of Screening.
- If on sapropterin or large neutral amino acids at Screening, must be on a stable dose for 4 weeks prior to Screening.
- Willing and able to maintain a diet consistent in Phe content from the Screening period through the duration of the study, unless otherwise directed by a dietician as allowed in the protocol.
- Body weight ≥ 12.5 kg.
- If female of childbearing potential:
- Must have a negative serum pregnancy test at Screening and a negative urine pregnancy test by Day 1.
- Must practice sexual abstinence, or if involved in any sexual intercourse that could lead to pregnancy, must agree to use 2 different contraceptive methods, where at least 1 method must be highly effective, from...
- Must refrain from donating ova during the course of the study and for 30 days after the last dose of the study drug.
- Is a female not of childbearing potential or postmenopausal, defined as follows:
- Has had surgical sterilization (hysterectomy, bilateral oophorectomy, or bilateral salpingectomy).
- Has had amenorrhea for minimum of 1 year with confirmation by levels of follicle stimulating hormone testing.
- Has not achieved menarche (has not had first menstrual period). If a female achieves menarche during the study, she will need to follow the contraception requirement for females of childbearing potential.
- If male, must practice sexual abstinence, or if involved in any sexual intercourse that could lead to pregnancy, must agree to use 2 different contraceptive methods, where at least 1 method must be highly effective...
- Capable of giving signed informed consent, or parent/legal guardian to provide informed consent and pediatric participant to give assent, and be able to comply with study procedures.
- Participants with psychiatric illness must be well-controlled for the last 3 months prior to screening visit and, if on medication, on stable medications for the last 3 months. Key
What rules you out
- Participation in this study is not considered safe and/or feasible in the opinion of the Investigator.
- Participants have not completed a previous JNT-517 study and are eligible for another active JNT-517 trial at the site, unless approval is obtained from the medical monitor.
- 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 significant liver disease.
- Any history of cataracts or more than minimal cataracts observed during the Screening ophthalmologic examination.
- Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion.
- Estimated glomerular filtration rate \< 60 milliliters per minute per 1.73 square meters (mL/min/1.73 m\^2) by 2021 Chronic Kidney Disease Epidemiology Collaboration formula (participants aged 17 years or greater) or by...
- History of drug or alcohol abuse in the last year.
- Use of any medication that are inhibitors or inducers of cytochrome P450 (CYP)3A4 or inhibitors of the transporter P glycoprotein (P-gp) within 4 weeks prior to the first dose of study drug and unwilling and/or unable...
- Use of any medications that are a substrate of breast cancer resistance protein (BCRP), multidrug and toxin extrusion (MATE)1, MATE2-K, organic anion transporter 3 (OAT3), or CYP3A4 within 4 weeks prior to the first...
- Current, recent, or suspected infection within 2 weeks of Screening of Severe Acute Respiratory Syndrome Coronavirus 2/Coronavirus Disease 2019 (SARS-CoV-2/COVID-19).
- Unable to tolerate oral medication or inability to swallow tablets.
- Allergy to JNT-517 or any component of the investigational product.
- Any of the following laboratory values at the Screening visit:
- Alanine aminotransferase or aspartate aminotransferase values ˃ 1.5 x the upper limit of normal (ULN).
- Total bilirubin ˃ULN unless history of Gilbert Syndrome and then total bilirubin \>4 milligrams per deciliter [mg/dL] is exclusionary.
- Hemoglobin ˂10.0 g/dL (˂100.0 grams per liter [g/L])
- White blood cell count ˃1 x ULN
- Platelet count ˂150 × 109/L (˂150,000/cubic millimeters[mm\^3])
- Participation in another investigational drug trial within 30 days (other than for JNT-517) or, if known 5 half-lives of investigational drug (whichever is longer).
The study team makes the final eligibility decision.
Where it's taking place
- Gainesville, Florida, United States
- Tampa, Florida, United States
- Portland, Oregon, United States
- Pittsburgh, Pennsylvania, United States
- Dallas, Texas, United States
- Houston, Texas, United States
- Salt Lake City, Utah, United States
- South Brisbane, Queensland, Australia
- Adelaide, South Australia, Australia
- Parkville, Victoria, Australia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Gainesville, Florida, United States; Tampa, Florida, United States; Portland, Oregon, United States; Pittsburgh, Pennsylvania, United States; Dallas, Texas, United States; Houston, Texas, United States and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.