A Clinical Study to Evaluate the Safety of MF1, a New Treatment for Parkinson's Disease-related Disorders (MF1 Study)
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-85
SponsorUniversity of Shizuoka
About this trial
This is a Phase I, investigator-initiated, first-in-human study to evaluate the safety, tolerability, and pharmacokinetics of MF1, a novel agent that is expected to inhibit α-synuclein related pathogenesis in α-synucleinopathies, primarily Parkinson's disease (PD). MF1 aims to address the unmet medical need in PD, which affects about 1% of individuals aged 60 years and older in Japan and is projected to reach 43 million patients worldwide by 2050.
The trial consists of three parts: Part A (single ascending dose) and Part B (multiple ascending dose) in healthy Japanese male adults, and Part C (multiple dose) in patients with idiopathic PD. Part A is a randomized, double-blind, placebo-controlled, single-center study assessing single oral doses , including a food-effect evaluation. Part B is a randomized, double-blind, placebo-controlled, single-center study with once-daily dosing for 7 days. Part C is an open-label, multicenter study in 4-8 PD patients (MDS 2015 criteria, Hoehn \& Yahr stage ≤3) receiving once daily for 14 days, with or without stable background antiparkinsonian therapy.
The primary objective is to assess safety and tolerability; secondary objectives include characterization of plasma, urine, and cerebrospinal fluid pharmacokinetics and assessment of food effect. Exploratory pharmacodynamic endpoints include biomarkers such as α-synuclein, neurofilament light chain, UCHL-1, FABP3, GFAP, and other neurodegeneration markers.
Key exclusion criteria include clinically significant systemic diseases, seizure history, serious infections (HBV, HCV, HIV, syphilis), recent suicidal ideation or attempts, and recent use of other investigational products.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
1)Healthy Japanese male adults aged >=18 and <45 years at the time of informed consent.
2) Subjects with a body mass index (BMI) of >=18.5 and <25.0 kg/m2 at screening.
3) Subjects who have received sufficient explanation regarding the study from the principal investigator or subinvestigator, have understood the objectives of the study, voluntarily agreed to participate, and have provided written informed consent of their own free will.
1) Patients diagnosed with idiopathic Parkinson's disease according to the International Parkinson and Movement Disorder Society (MDS) Clinical Diagnostic Criteria (2015).
Disqualifiers
1) Subjects with clinically significant cardiovascular, neurological, pulmonary, hepatic, renal, metabolic, gastrointestinal, urological, immunological, endocrine, or psychiatric disorders, or any other abnormalities that may affect safety, increase seizure risk, lower seizure threshold, or confound study results.
2) Subjects with current or past diseases or surgical histories involving the gastrointestinal tract, liver, kidneys, or other organs that may affect drug absorption, metabolism, or excretion.
3) Subjects who used any medication, including over-the-counter drugs, within 7 days prior to the day before the first administration of the investigational product.
4) Subjects with seizure disorders such as epilepsy, or a history thereof.
Trial design
Design model
Parallel
Treatments tested in this trial
MF-1
DrugOral administration of MF1
Placebo
DrugIndistinguishable from MF1
Treatment groups
58 Participants
are divided into 5 treatment groupsGroup A: MF1 single ascending dose (healthy)Experimental treatment 1 intervention
Group B: Placebo single dose (healthy)Placebo comparator 1 intervention
Group C: MF1 multiple ascending doses (healthy)Experimental treatment 1 intervention
Group D: Placebo multiple doses (healthy)Placebo comparator 1 intervention
Group E: MF1 multiple doses (Parkinson's disease)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Number of participants with treatment-emergent adverse events and serious adverse events
The number and percentage of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) will be summarized by treatment group and study part (Part A, Part B, Part C), including events leading to permanent discontinuation of study drug and clinically significant changes in vital signs, clinical laboratory tests, and 12-lead ECGs.
Time frame
12 days from last dosing2
Maximum plasma concentration (Cmax) of MF1
Cmax will be determined from plasma concentration-time data following single and multiple oral doses of MF1 in healthy subjects (Parts A and B) and patients with Parkinson's disease (Part C)
Time frame
5 days after last dosing3
Area under the plasma concentration-time curve from time zero to last measurable concentration (AUC0-t) of MF1
AUC0-t will be calculated using the linear-log trapezoidal method from plasma concentration-time data following single and multiple oral doses of MF1
Time frame
Time Frame: Pre-dose through 5 days after last dosing4
Terminal elimination half-life (t1/2) of MF1
t1/2 will be calculated from the terminal slope of the plasma concentration-time profile following single and multiple oral doses of MF1
Time frame
Pre-dose through 5 days after last dosingLocations
1Map coordinates are unavailable for these locations. Locations are shown below instead.
Sumida HospitalRecruiting130-0004, Sumida-ku, Tokyo
Japan
JapanSponsors and contacts
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University of Shizuoka
Lead sponsor
Tohoku University
Collaborator
Source ClinicalTrials.gov