JNT-517 for Phenylketonuria
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the safety and effects of an oral treatment, JNT-517, for adolescents with phenylketonuria (PKU), a condition where the body can't break down a certain amino acid, leading to its harmful buildup. Participants will receive either JNT-517 or a placebo twice a day for 28 days, with an 80% chance of receiving the actual medication. Those diagnosed with PKU who can swallow tablets and have certain levels of phenylalanine in their blood might qualify. The study includes regular checkups, some at home, and requires keeping a food diary. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, contributing to important advancements in PKU treatment.
Do I need to stop my current medications for the trial?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that JNT-517 is likely to be safe for humans?
A previous study found JNT-517 to be safe and well-tolerated, with no serious side effects reported. Both the 75 mg and 150 mg doses showed similar safety results. Participants taking JNT-517 experienced no major health problems. The treatment also increased the removal of a substance linked to phenylketonuria, a condition affecting protein processing. These findings suggest that JNT-517 is generally safe for use in this context.12345
Why do researchers think this study treatment might be promising for PKU?
Most treatments for phenylketonuria (PKU) focus on dietary management or enzyme replacement therapies. However, JNT-517 stands out because it targets phenylalanine hydroxylase, the enzyme crucial for breaking down phenylalanine, a substance that can accumulate and cause problems in PKU patients. This direct approach could offer a more efficient way to manage phenylalanine levels compared to existing methods. Researchers are particularly excited about JNT-517 due to its potential to significantly improve the quality of life for those with PKU with fewer dietary restrictions.
What evidence suggests that JNT-517 might be an effective treatment for phenylketonuria?
Research has shown that JNT-517 may help treat Phenylketonuria (PKU). In earlier studies, 57% of patients taking 75 mg twice a day, and 80% of those taking 150 mg twice a day, reached the recommended levels of phenylalanine (Phe) in their blood after 28 days. This trial will test JNT-517 at both 75 mg and 150 mg doses, as well as a placebo. The higher dose of 150 mg also resulted in a 60% average drop in blood Phe levels compared to a placebo. JNT-517 blocks a protein that retains Phe in the body, aiding in its removal through urine. Overall, these findings suggest JNT-517 could effectively manage PKU.12345
Are You a Good Fit for This Trial?
Adolescents aged 12-17 with Phenylketonuria (PKU), weighing at least 45 kg and a BMI under 40, who can swallow tablets. They must have consistent plasma Phe levels above a certain threshold. Girls able to have children must use reliable birth control or practice abstinence.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive 75 mg JNT-517 or placebo BID for 28 days
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- JNT-517
Find a Clinic Near You
Who Is Running the Clinical Trial?
Jnana Therapeutics
Lead Sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Lead Sponsor
John Kraus
Otsuka Pharmaceutical Development & Commercialization, Inc.
Chief Medical Officer since 2023
MD, PhD
Tarek Rabah
Otsuka Pharmaceutical Development & Commercialization, Inc.
Chief Executive Officer since 2022
BS in Biology and BA in Business from the American University of Beirut, MBA from McGill University