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

NCT07666022 | Recruiting Phase 1

• 1 other identifier: C-72
• Study Type: interventional
• Target: 58
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Healthy Adult Male; PARKINSON DISEASE (Disorder)

Keywords

FIH

Outcome Measures

Primary Outcomes (5)

• 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.

  12 days from last dosing

• 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)

  5 days after last dosing

• 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: Pre-dose through 5 days after last dosing

• 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

  Pre-dose through 5 days after last dosing

• Cerebrospinal fluid (CSF) concentration of MF1

  CSF concentrations of MF1 will be determined at a predefined time point in healthy participants in single ascending last 2 doses（Part A） and patients with Parkinson's disease (Part C) to assess central nervous system penetration.

  Pre-dose through 24 hours after last dosing

Study Arms (5)

MF1 single ascending dose (healthy)

EXPERIMENTAL

Single oral dose of MF1 in healthy male adults (Part A).

Drug: MF-1

Placebo single dose (healthy)

PLACEBO COMPARATOR

Single oral dose of placebo in healthy male adults (Part A).

Drug: Placebo

MF1 multiple ascending doses (healthy)

EXPERIMENTAL

Once-daily oral doses of MF1 for 7 days in healthy male adults (Part B).

Drug: MF-1

Placebo multiple doses (healthy)

PLACEBO COMPARATOR

Once-daily oral doses of placebo for 7 days in healthy male adults (Part B).

Drug: Placebo

MF1 multiple doses (Parkinson's disease)

EXPERIMENTAL

Once-daily oral doses of MF1 for 14 days in patients with Parkinson's disease (Part C).

Drug: MF-1

Interventions

MF-1 DRUG

Oral administration of MF1

MF1 multiple ascending doses (healthy); MF1 multiple doses (Parkinson's disease); MF1 single ascending dose (healthy)

Placebo DRUG

Indistinguishable from MF1

Placebo multiple doses (healthy); Placebo single dose (healthy)

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• (Parts A and B)
• )Healthy Japanese male adults aged \>=18 and \<45 years at the time of informed consent.
• \) Subjects with a body mass index (BMI) of \>=18.5 and \<25.0 kg/m2 at screening.
• \) 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.
• (Part C)
• \) Patients diagnosed with idiopathic Parkinson's disease according to the International Parkinson and Movement Disorder Society (MDS) Clinical Diagnostic Criteria (2015).
• \) Patients with Parkinson's disease classified as Stage 3 or below according to the modified Hoehn and Yahr staging scale.
• \) Patients who are either untreated or have been receiving one of the following treatments at a stable dosage regimen for at least 8 weeks prior to screening, with no planned changes during the study period: selegiline up to 5 mg twice daily, rasagiline up to 1 mg once daily, or immediate-release carbidopa/levodopa up to 25/100 mg three times daily.
• \) Patients with an average Bristol Stool Scale score of \<=3 from the date of informed consent to eligibility assessment, or patients with fewer than two bowel movements per week.
• If the period between informed consent and eligibility assessment is less than one week, information prior to informed consent will also be collected to assess bowel conditions for at least one week in total.
• \) Male or female patients aged \>=40 and \<85 years at the time of informed consent.
• \) Patients with a BMI of \>=18.5 and \<32.0 kg/m2 at screening.
• \) Female patients who are postmenopausal for at least one year at the time of informed consent, including menopause resulting from hysterectomy or oophorectomy.
• \) Patients 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.

You may not qualify if:

• (Parts A and B)
• \) 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.
• \) 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.
• \) 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.
• \) Subjects with seizure disorders such as epilepsy, or a history thereof.
• \) Subjects with allergies or a history of allergies to drugs or foods.
• \) Subjects with allergic predisposition who are considered unsuitable for participation by the principal investigator or subinvestigator.
• \) Subjects with current or past alcohol or drug dependence.
• \) Subjects who donated \>=400 mL of whole blood within 12 weeks, \>=200 mL of whole blood within 4 weeks, or blood components within 2 weeks prior to investigational product administration.
• \) Subjects who tested positive at screening for HBs antigen, HCV antibody, HIV antigen/antibody, or syphilis serology (TP antibody test or RPR test).
• \) Subjects unwilling to use appropriate contraception from the time of informed consent until the final study visit.
• \) Subjects who answered "Yes" to Question 4 or 5 regarding suicidal ideation on the Columbia-Suicide Severity Rating Scale (C-SSRS) at screening, or who had a history of suicidal behavior within 6 months prior to screening.
• \) Subjects who received investigational treatment in another clinical trial within 4 months prior to investigational product administration.
• \) Subjects judged unsuitable for study participation by the principal investigator or subinvestigator based on findings from screening or admission assessments, observations, or examinations.
• (Part C)

Sponsors & Collaborators

• University of Shizuoka: lead
• Tohoku University: collaborator

Study Sites (1)

Sumida Hospital

Sumida-ku, Tokyo, 130-0004, Japan

RECRUITING

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Central Study Contacts

MASANORI FUJIWARA

CONTACT

+81 22-717-7136; masanori.fujiwara.a5@tohoku.ac.jp

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: SCREENING
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 9, 2026
• First Posted: June 24, 2026
• Study Start: June 1, 2026
• Primary Completion (Estimated): September 30, 2028
• Study Completion (Estimated): October 31, 2028
• Last Updated: June 24, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE

Locations

JP (1)