ANZCTR search results

This study is designed as a 3-part study in healthy participants to test the drug-to –drug interaction of experimental drug, JNT-517 with itraconazole, midazolam and pravastatin, respectively. JNT-517 is believed to reduce levels of phenylalanine in the by increasing its removal with urine and reducing uptake of phenylalanine in the gut.

This will be a phase 1, first-in-human, open-label, 3-period, 3-treatment crossover design study to evaluate the food effects and relative bioavailability of 2 formulations of JNT-517 in 12 healthy participants.

This will be a Phase 1 first-in-human (FIH) study in 2 parts to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of JNT-517 at various incremental doses administered as single and multiple doses in healthy volunteers. The study will begin with a single ascending dose portion (Part A) followed by the multiple ascending dose portion (Part B) in healthy volunteers in Australia to evaluate the safety, tolerability, PK, and PD of JNT-517 at various incremental doses.

The study is intended to assess the relative bioavailability of the analytes BH4 and sepiapterin following a single oral dose of 2 formulations of PTC923 (PTC923 Phase 3 powder for oral use in water [Formulation B] vs. PTC923 Phase 1/2 powder for oral suspension in Medisca oral mix [Formulation A]) under fed conditions in male and female adult healthy volunteers. Medisca oral mix is used for Formulation A to be consistent with the way in which the Phase 1 and Phase 2 studies were conducted previously. The current powder for oral use PTC923 formulation has been used in the Phase 1/2 studies in healthy volunteers and PKU and/or PBD patients.

Phenylketonuria (PKU) is a rare Inborn Error of Metabolism (IEM) with an incidence of approximately 1 in 10 - 15,000. Prior to the advent of newborn screening and effective dietary treatments, PKU caused a severe neurodevelopmental phenotype marked by global developmental delay, severe intellectual impairment, and seizures. Early diagnosis from newborn screening and introductions of dietary interventions prevent these severe neurological manifestations. Despite this, neuropsychological complications of PKU are observed when control is suboptimal, which include reduced executive function, attention deficit, decreased processing speed, cognitive impairment and disturbances in emotion and behaviour. Neuro-disabilities are thus a key phenotype amongst the PKU cohort when their metabolic control is suboptimal. This project is aimed at exploring practical tools to improve our management outcomes for children and families living with this rare chronic disease. We hypothesise that participating in a brief evidence-based parenting intervention (Triple P), when compared to baseline, will improve progress towards child behaviour/parenting goals, improve children’s quality of life, decrease parent-reported use of ineffective parenting practices, increase parent self-efficacy, reduce parent-reported child behavioural problems, reduce parenting stress, and improve children’s blood Phe levels.

CNSA-001 represents the first viable formulation of sepiapterin intended for the treatment of hyperphenylalaninemia (HPA) in PKU patients. This Phase 2 study will assess 2 doses of CNSA-001 in comparison to the maximum recommended dose of Kuvan. This is a proof-of-concept (PoC) study that will help to establish dose selection and support the design of future Phase 2/3 studies in PKU patients.

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.