RecruitingPhase 1ACTRN12625000230459

A Phase 1 Randomized, Double-Blind, Placebo-Controlled Study With A Food Effect Assessment To Evaluate The Safety, Tolerability, And Pharmacokinetics Of Single Ascending Doses And Multiple Ascending Doses Of PX578 In Healthy Adult Participants (Part A & B)

Sponsor

Pretzel Therapeutics Inc.

Enrollment

112 participants

Start Date

Apr 22, 2025

Study Type

Interventional

Conditions

Mitochondrial disease

Summary

This is a double-blind, placebo-controlled study to assess the safety of PX578 and how this drug acts in the body in healthy volunteers. PX578 may be indicated for use in patients with Mitochondrial disease, but a trial of the drug in healthy volunteers is needed before trials in Mitochondrial disease patients can proceed. Who is it for? You may be eligible for this study if you are aged 18 to 55 years and are in good general health without a clinically significant medical history. Study details All healthy volunteer participants who choose to enrol in this study will be assigned by chance to receive single or multiple doses of PX578 or placebo. All participants will have their vital signs checked (heart rate, blood pressure, temperature, etc), and will provide blood and urine samples for testing. It is hoped this research will determine the maximum dose of PX578 that can be administered safely without causing severe reactions. Once the dose of PX578 has been determined in healthy volunteers, a trial investigating the efficacy of PX578 as a treatment for patients with Mitochondrial disease may proceed.

Eligibility

Sex: Both males and femalesMin Age: 18 YearssMax Age: 55 Yearss

Plain Language Summary

Simplified for easier understanding

Mitochondrial disease is a rare but serious condition where the mitochondria — the energy-producing parts of cells — don't work properly. This affects organs and tissues throughout the body and can cause muscle weakness, fatigue, neurological problems, and more. There are currently very few effective treatments available. PX578 is a new experimental medication that may support mitochondrial function. Before it can be tested in patients, it needs to be evaluated in healthy volunteers first to establish its safety, how well it is tolerated, and how the body handles it. This Phase 1 study will test single and then multiple escalating doses to determine a safe dosing range. You may be eligible if you are a healthy adult aged 18 to 55 with a normal BMI (18–32) and no significant medical history. You must be willing to abstain from alcohol, smoking, and certain foods during the study. Participants who are pregnant, have liver disease, HIV, active hepatitis B or C, or take prescription medications are not eligible.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

This Phase I healthy volunteer study will assess the safety, tolerability and pharmacokinetics (PK) of PX578 in healthy participants and will be conducted in 2 parts: Part A: Single ascending dos

This Phase I healthy volunteer study will assess the safety, tolerability and pharmacokinetics (PK) of PX578 in healthy participants and will be conducted in 2 parts: Part A: Single ascending dose (SAD) evaluation of PX578 oral capsule or placebo will be administered a single dose on Day 1 at up to 6 fixed dose levels (in a total of 6 cohorts) in the range from 100 mg for Cohort 1, 250 mg for Cohort 2 and 500 mg for Cohort 3. For Cohorts 4, 5 and 6 the dose will be determined by the Safety Review Committee (SRC) based on safety, tolerability and PK data from previous cohorts up to a maximum dose of 1500 mg. Part B: Multiple ascending dose (MAD) evaluation PX578 oral capsule or placebo will be administered twice daily for 10 days at up to 6 fixed dose levels (in a total of 6 cohorts) . The dose for all cohorts will be determined by the SRC based on safety, tolerability and PK data from previous cohorts up to a maximum dose of 1500 mg. Participants will be dosed at the site and will receive study drug directly from the Investigator or designee, under medical supervision. Drug administration will be recorded and any discrepancies with the dosing regimen will be documented and explained in the eCRF and the source documents. The dose of study drug and study participant identification will be confirmed at the time of dosing by a member of the study site staff other than the person administering the study drug.

Locations(1)

Christchurch, New Zealand

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