Phenylketonuria Clinical Trial
Official title:
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
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.
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT05099640 -
A Study of PTC923 in Participants With Phenylketonuria
|
Phase 3 | |
| Completed |
NCT01924026 -
Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
|
N/A | |
| Completed |
NCT01428258 -
Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria
|
N/A | |
| Completed |
NCT00925054 -
Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
|
Phase 2 | |
| Completed |
NCT00778206 -
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
|
||
| Recruiting |
NCT05948020 -
Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria
|
N/A | |
| Completed |
NCT02555579 -
Simplified Diet Approach in Phenylketonuria
|
N/A | |
| Completed |
NCT03097250 -
MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria
|
||
| Completed |
NCT01965691 -
Protein Requirements in Children With Phenylketonuria (PKU)
|
N/A | |
| Completed |
NCT01965912 -
Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
|
Phase 4 | |
| Completed |
NCT00688844 -
Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
|
N/A | |
| Completed |
NCT00789568 -
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
|
Phase 1 | |
| Terminated |
NCT01465100 -
Liver Cell Transplant for Phenylketonuria
|
Phase 1/Phase 2 | |
| Completed |
NCT01732471 -
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
|
Phase 3 | |
| Completed |
NCT04879277 -
Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria
|
N/A | |
| Completed |
NCT02176603 -
Observational Study of Endothelial Dysfunction in Phenylketonuria
|
N/A | |
| Completed |
NCT01819727 -
An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
|
Phase 3 | |
| Completed |
NCT01869972 -
Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
|
N/A | |
| Terminated |
NCT01904708 -
Moderate Intensity Exercise and Phenylketonuria
|
N/A | |
| Completed |
NCT01560286 -
A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
|
Phase 2 |