RecruitingPhase 1ACTRN12623001117606

A Phase 1 single and multiple ascending dose study of YA-101 in Healthy Subjects

A Double-Blind, Placebo-Controlled, Single Dose and Multiple Ascending Dose, Phase I Study to Evaluate the Safety, Tolerability and Pharmacokinetics of YA-101 in Healthy Subjects

Sponsor

Yoda Pharmaceuticals Aus Pty Ltd

Enrollment

60 participants

Start Date

Oct 12, 2023

Study Type

Interventional

Conditions

Multiple System Atrophy

Summary

Multiple system atrophy (MSA) is a rare, severe and life-threatening adult-onset neurodegenerative disease, characterized by a combination of parkinsonism, cerebellar ataxia, and autonomic dysfunction. At present, only symptomatic therapy is available for the treatment of MSA, and most of the prescribed medications are being used in an off-label manner. YA-101 is being developed for the treatment of subjects with MSA, a serious and life-threatening condition with a high, unmet clinical need. The safety, tolerability, PK, and food effect of YA-101 will be evaluated in HV in Parts 1 and 2 of this study. The study will be conducted in healthy volunters aged between 18-65 years . Besides the one food effect (FE) cohort, each cohort in Part 1~Part 2 is randomized, placebo-controlled, double-blinded to establish safety and tolerability in healthy volunteers (HV) following single ascending dose (SAD) and multiple ascending dose (MAD). A total of 60 participants will be enrolled in Australia. Duration of the study Part 1 HV SAD: Total duration of study is up to 8 days with 1-day treatment Part 2 HV MAD: Total duration of study is up to 21 days, 14-day treatment Part 2 HV FE: Total duration of study is up to 11 days with 1-day treatment

Eligibility

Sex: Both males and femalesMin Age: 18 YearssMax Age: 65 Yearss

Plain Language Summary

Simplified for easier understanding

This Phase 1 study is testing the safety and tolerability of a new investigational drug called YA-101 in healthy volunteers. YA-101 is being developed as a potential future treatment for Multiple System Atrophy (MSA) — a rare and progressive brain disease that causes problems with movement, balance, and automatic body functions like blood pressure and bladder control. Currently, there is no cure or disease-modifying treatment for MSA, making new drug development critically important. Before YA-101 can be tested in patients with MSA, researchers need to confirm it is safe and understand how the body processes it. This trial will enrol 60 healthy volunteers in Australia who will receive either the drug or a placebo, with doses gradually increasing to establish the highest safe dose. Some participants will also test how food affects how the drug is absorbed. You may be eligible if you are a healthy adult aged 18 to 65 with no history of significant medical conditions, drug or alcohol issues, or recent participation in other trials. The study involves short stays in a clinical facility, multiple blood tests, and regular health monitoring. Your contribution helps pave the way for future trials in people actually living with MSA.

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 is a phase 1 study consisting of Part 1~Part 2. Besides the one food effect (FE) cohort, each cohort in Part 1~Part 2 is randomized, placebo-controlled, double-blinded to establish safety and tolerability in healthy volunteers (HV) following single ascending dose (SAD) and multiple ascending dose (MAD). FE is a single cohort, cross-over, 2-period study planned to evaluate the effect of food on the PK profile of open-label YA-101. - Part 1 will assess the safety, tolerability, and PK of YA-101 administered to healthy adult volunteers as a single-ascending dose (HV SAD) - Part 2 will assess the safety, tolerability, and PK of YA-101 administered to healthy adult volunteers as multiple-ascending doses (HV MAD), and as a single dose administered with and without food to assess food effect (HV FE). Investigational Product: YA-101 Dose form: Suspension Route of Administration: Oral Dose (mg/day): Starting dose for HV SAD Cohort 1 is based on the human equivalent dose (HED) calculated from the NOAEL reported in the 28-day GLP rat toxicity study, with application of an appropriate safety factor. SAD: Starting dose for Cohort 1 is starting at 900mg and escalating to a maximum of 9600mg of YA-101. Subsequent dose levels for Cohort 2 and Cohort 3 will be determined based on the emerging safety, tolerability, and PK data in the preceding cohort(s).Dose frequency: once per day FE: HV FE doses will be determined based on the emerging safety, tolerability, and PK data from SAD cohorts. In the fed state, a high-fat (approximately 50% of total caloric content of the meal) and high-calorie (approximately 800 to 1000 calories) meal is provided before dosing. YA-101 suspension will be administered within 30 minutes after the completion of meal. (Dose frequency: once per day on Day 1 and Day 4) MAD: HV MAD doses will be determined based on the emerging safety, tolerability, and PK data from SAD cohorts. The dosing time of Part 1 is separated from the first dosing time of Part 2 by greater than 1 week. Participants from Part 1 may also participate in Part 2. Dose frequency: twice daily for 14 days. On Day 14, subjects will only take the morning dose. Placebo: YA-101 matching placebo will be provided for each cohort in SAD and MAD. Study drug administration will take place on-site with oversight by study staff. Furthermore, the study drug must be dispensed under the supervision of the investigator or an authorized designee, and it should be dispensed exclusively to study subjects. The designated site staff or pharmacist will maintain records of the study drug received, dispensed to subjects, and any returned quantities.