Evaluating Safety and Pharmacokinetics of MF1 in Healthy Adults and Parkinsons Disease Patients in a Phase I Study

What this Trial Is About

Researchers are evaluating MF1, a new drug aimed at targeting the underlying pathological mechanisms of alpha-synucleinopathies, primarily Parkinsons disease PD. This Phase I first-in-human study seeks to assess the safety, tolerability, and pharmacokinetics of MF1, addressing a critical unmet medical need as current therapies only treat symptoms without modifying disease progression. The study involves healthy Japanese male adults and patients with idiopathic PD, reflecting different stages of clinical development. The trial has three parts Part A tests single ascending oral doses of MF1 and includes a food-effect assessment in healthy males aged 18 to under 45 years. Part B administers once-daily MF1 or placebo for 7 days in the same healthy population. Part C is an open-label study giving once-daily MF1 for 14 days to patients aged 40 to under 85 years with idiopathic PD Hoehn & Yahr stage 3 or below, with or without stable background antiparkinsonian treatments. The study also evaluates cerebrospinal fluid penetration and exploratory biomarkers related to neurodegeneration. Participants will undergo safety monitoring including adverse event assessments and pharmacokinetic sampling of plasma, urine, and cerebrospinal fluid. Biomarkers such as alpha-synuclein and others will be measured to explore pharmacodynamic effects. The study includes randomized, placebo-controlled parts in healthy volunteers and an open-label part in PD patients, with follow-up for up to 12 days after dosing. The total participation duration varies by study part, with careful review before progressing between stages.

A Clinical Study to Evaluate the Safety of MF1, a New Treatment for Parkinson's Disease-related Disorders (MF1 Study)