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New treatment option for Phenylketonuria
Official title A Study of JNT-517 in Participants With Phenylketonuria (PKU)
ClinicalTrials.gov ID: NCT06971731
What this study is testing
What is JNT-517 Tablet?
JNT-517 Tablet is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for phenylketonuria.
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, randomized study is to assess the safety, efficacy, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adults (18 years of age or older) with PKU. Participants will receive either JNT-517 or placebo and will be blinded to their treatment assignment.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Males and females ≥18 years of age on Day 1
- Clinical diagnosis of PKU
- Average of at least 3 plasma Phe levels (after \>4-hour fast) during Screening period of ≥360 μmol/L
- Not on pegvaliase within 4 weeks prior to Screening
- If on sapropterin or large neutral amino acids, such as PheBloc®, NeoPhe®, and PreKunil® at Screening, must be on a stable dose 4 weeks prior to...
You likely can't join if
- Exclusion Criteria Participants will be excluded from the study if any of the following criteria are met:
- Any acute or uncontrolled chronic medical condition that would prevent the participant from complying with the procedures or place the participant at...
- Positive for hepatitis B or C or human immunodeficiency virus
- Any history of malignancy of any organ system (other than non-melanoma skin cancer or in situ cervical cancer), treated or untreated, within the past...
- Any history of significant liver disease
- Any history of cataracts or more than minimal cataracts observed during the Screening ophthalmologic examination. Minimal cataracts are defined as...
See the full eligibility criteria
Who can join
- Males and females ≥18 years of age on Day 1
- Clinical diagnosis of PKU
- Average of at least 3 plasma Phe levels (after \>4-hour fast) during Screening period of ≥360 μmol/L
- Not on pegvaliase within 4 weeks prior to Screening
- If on sapropterin or large neutral amino acids, such as PheBloc®, NeoPhe®, and PreKunil® at Screening, must be on a stable dose 4 weeks prior to Screening and for the entire study duration.
- Willing and able to maintain a stable diet in Phe and total protein (intact protein and medical food protein) and able to adjust diet through the duration of the study according to the Dietary Management Guidelines
- Body weight \>40 kilograms (kg)
- If biologically 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 highly effective contraceptive methods from Screening until at least 30 days after the last...
- If taking estrogen- or progesterone-based oral contraceptives, must agree to use 2 other highly effective methods of contraception or must agree to sexual abstinence during the study
- Must refrain from donating ova during the course of the study and for 30 days after the last dose of the study drug.
- If a biologically 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 biologically male, must practice sexual abstinence, or if involved in any sexual intercourse that could lead to pregnancy, must agree to use highly effective contraceptive methods from Day 1 until at least 30 days...
- Participants with psychiatric illness must be well-controlled for the last 6 months prior to the Screening visit and if on medication, on stable medications for the last 3 months.
- Capable of giving signed informed consent or parent/legal guardian to provide informed consent and the participant to give assent and confirm able to comply with study procedures
What rules you out
- Exclusion Criteria Participants will be excluded from the study if any of the following criteria are met:
- Any acute or uncontrolled 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 malignancy of any organ system (other than non-melanoma skin cancer or in situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence...
- Any history of significant liver disease
- Any history of cataracts or more than minimal cataracts observed during the Screening ophthalmologic examination. Minimal cataracts are defined as changes similar to lens opacities classification system III (LOCS III)...
- Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion
- Estimated glomerular filtration rate (eGFR) \<60 mL/min/1.73 m2 by 2021 Chronic Kidney Disease Epidemiology Collaboration formula
- Participation in another investigational drug trial within 30 days or, if known, 5 half-lives of the investigational drug (whichever is longer). For gene therapy or editing trials, participants must have received the...
- Alcohol consumption within 5 days of randomization and/or unwilling to limit to 1 alcoholic drink per day until after the 6-month study visit
- History of drug/alcohol abuse in the last year
- Use of any medications that are inhibitors or inducers of cytochrome P450 (CYP3A4) or inhibitors of the transporter P-glycoprotein (P-gp) within 4 weeks prior to randomization and unwilling and/or unable to avoid these...
- Use of any medications that are substrates of breast cancer resistance protein (BCRP), multidrug and toxin extrusion (MATE)1, or MATE2-K within 4 weeks prior to randomization and unwilling and/or unable to avoid these...
- Current, recent, or suspected active viral or bacterial infection within 2 weeks prior to and during the Screening Period
- Unable to tolerate oral medication or have a condition that would interfere with the absorption of JNT-517
- 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× the upper limit of normal (ULN)-Total bilirubin ˃ULN unless history of Gilbert...
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Gainesville, Florida, United States
- Tampa, Florida, United States
- Chicago, Illinois, United States
- Portland, Oregon, United States
- Pittsburgh, Pennsylvania, United States
- Nashville, Tennessee, United States
- Dallas, Texas, United States
- Houston, Texas, United States
- Salt Lake City, Utah, United States
- Adelaide, Australia
- Parkville, Australia
- South Brisbane, Australia
- Calgary, Alberta, Canada
- Prague, Czechia
- Tours, France
- Mainz, Germany
- Münster, Germany
- Chiyoda-ku, Japan
- Kutsukake-cho, Japan
+ 6 more site(s).
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, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Gainesville, Florida, United States; Tampa, Florida, United States; Chicago, Illinois, United States; Portland, Oregon, United States; Pittsburgh, Pennsylvania, United States and 20 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.