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

2026년 6월 18일 업데이트: University of Shizuoka

A Phase I Investigator-initiated First-in-human Study to Evaluate the Safety and Pharmacokinetics of MF1 in Healthy Adults and Patients With Parkinson's Disease (MF1-FIH)

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.

연구 개요

상세 설명

  • α-Synucleinopathies are a group of progressive neurodegenerative diseases caused by the pathological aggregation and prion-like propagation of α-synuclein protein, culminating in selective neuronal loss. Parkinson's disease (PD) represents the most prevalent form, affecting approximately 1% of individuals aged 60 years and older in Japan, with the global burden projected to reach 43 million patients by 2050. Despite decades of research, currently approved therapies remain restricted to symptomatic management targeting dopaminergic deficits and do not address the underlying mechanisms of α-synuclein misfolding, oligomerization, or cell-to-cell spread. No disease-modifying, neuroprotective, or neurorestorative therapy has been approved to date, representing a critical unmet medical need.
  • MF1 is a novel investigational agent designed to target the pathophysiological mechanisms underlying α-synucleinopathy. Preclinical studies have demonstrated that MF1 inhibits α-synuclein aggregation and reduces neuroinflammatory cascades associated with dopaminergic neurodegeneration. Based on its mechanism of action, MF1 is expected to be a novel therapeutic agent targeting these pathological mechanisms, with the potential to confer both neuroprotective effects and clinically meaningful symptomatic benefits, thereby addressing this unmet medical need.
  • This First-in-Human (FIH) Phase I trial employs a stepwise dose-escalation approach across three sequential parts to characterize the safety, tolerability, and pharmacokinetic (PK) profile of MF1. The three-part sequential dose-escalation design, incorporating single ascending doses (Part A) and multiple ascending doses (Part B) in healthy adult volunteers, followed by evaluation in a patient cohort (Part C), is consistent with established first-in-human (FIH) best practices and ensures systematic safety data review prior to advancement to each subsequent study part.
  • The restriction to healthy Japanese males aged 18-44 years with BMI 18.5 to <25.0 kg/m² in Parts A and B is intended to minimize confounding variables during initial safety and PK characterization. Given that MF1 is intended for a patient population with a higher average age and potential co-morbidities, Part C enrolls patients with idiopathic PD under a more inclusive age range (40-84 years) and permits concomitant use of select stable antiparkinsonian medications to reflect real-world treatment conditions. The restriction to Hoehn & Yahr stage ≤3 ensures that participants retain sufficient functional capacity for protocol compliance and limits confounding from advanced disease-related physiological changes.
  • Food effect assessment in Part A is incorporated to characterize the influence of food intake on oral bioavailability of MF1, which will inform dosing recommendations for subsequent studies. CSF sampling in a subset of participants is planned to assess CNS penetration of MF1, a critical pharmacological prerequisite for a CNS-targeted agent. Exploratory pharmacodynamic assessments include established fluid biomarkers of neuronal injury and glial activation (α-synuclein, Nf-L, UCHL-1, FABP3, GFAP), which will provide mechanistic evidence of target engagement and serve as early signals for neuroprotective activity.
  • The primary objectives of Part C are to assess the safety and tolerability profile in a small number of elderly subjects and to obtain pharmacokinetic data in this patient population, which will serve to support rational dose selection for subsequent phases of clinical development.

연구 유형

중재적

등록 (추정된)

58

단계

  • 1단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

연구 장소

    • Tokyo
      • Sumida-ku, Tokyo, 일본, 130-0004
        • 모병
        • Sumida Hospital
        • 연락하다:
        • 연락하다:
          • Masanoir Fujiwara
        • 수석 연구원:
          • Rie Yazawa, MD

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

예

설명

Inclusion Criteria:

(Parts A and B)

  • 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.

(Part C)

  • 1) Patients diagnosed with idiopathic Parkinson's disease according to the International Parkinson and Movement Disorder Society (MDS) Clinical Diagnostic Criteria (2015).
  • 2) Patients with Parkinson's disease classified as Stage 3 or below according to the modified Hoehn and Yahr staging scale.
  • 3) 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.
  • 4) 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.

  • 5) Male or female patients aged >=40 and <85 years at the time of informed consent.
  • 6) Patients with a BMI of >=18.5 and <32.0 kg/m2 at screening.
  • 7) Female patients who are postmenopausal for at least one year at the time of informed consent, including menopause resulting from hysterectomy or oophorectomy.
  • 8) 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.

Exclusion Criteria:

(Parts A and B)

  • 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.
  • 5) Subjects with allergies or a history of allergies to drugs or foods.
  • 6) Subjects with allergic predisposition who are considered unsuitable for participation by the principal investigator or subinvestigator.
  • 7) Subjects with current or past alcohol or drug dependence.
  • 8) 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.
  • 9) Subjects who tested positive at screening for HBs antigen, HCV antibody, HIV antigen/antibody, or syphilis serology (TP antibody test or RPR test).
  • 10) Subjects unwilling to use appropriate contraception from the time of informed consent until the final study visit.
  • 11) 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.
  • 12) Subjects who received investigational treatment in another clinical trial within 4 months prior to investigational product administration.
  • 13) 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)

  • 1) Patients with drug-induced parkinsonism, metabolic neurogenetic disorders, encephalitis, Parkinson-plus syndromes, or other atypical parkinsonian syndromes.
  • 2) Patients with freezing of gait.
  • 3) Patients with a history of stereotactic brain surgery for Parkinson's disease (e.g., pallidotomy, deep brain stimulation, or fetal tissue transplantation).
  • 4) Patients with clinically significant cardiovascular, neurological, pulmonary, hepatic, renal, metabolic, gastrointestinal, urological, immunological, endocrine, or psychiatric disorders other than Parkinson's disease, or any other abnormalities that may affect safety, increase seizure risk, lower seizure threshold, or confound study results.
  • 5) Patients 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.
  • 6) Patients with seizure disorders such as epilepsy, or a history thereof.
  • 7) Patients currently receiving antiplatelet agents or anticoagulants.
  • 8) Patients with allergies or a history of allergies to drugs or foods.
  • 9) Patients with allergic predisposition who are considered unsuitable for participation by the principal investigator or subinvestigator.
  • 10) Patients with current or past alcohol or drug dependence.
  • 11) Patients who donated >=400 mL of whole blood within 16 weeks, >=200 mL of whole blood within 4 weeks, or blood components within 2 weeks prior to investigational product administration.
  • 12) Patients who tested positive at screening for HBs antigen, HCV antibody, HIV antigen/antibody, or syphilis serology (TP antibody test or RPR test).
  • 13) Patients unwilling to use appropriate contraception from the time of informed consent until the final study visit.
  • 14) Patients 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.
  • 15) Patients who received investigational treatment in another clinical trial within 4 months prior to investigational product administration.
  • 16) Patients judged unsuitable for study participation by the principal investigator or subinvestigator based on findings from screening or admission assessments, observations, or examinations.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 상영
  • 할당: 무작위
  • 중재 모델: 병렬 할당
  • 마스킹: 삼루타

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: MF1 single ascending dose (healthy)
Single oral dose of MF1 in healthy male adults (Part A).
Oral administration of MF1
위약 비교기: Placebo single dose (healthy)
Single oral dose of placebo in healthy male adults (Part A).
Indistinguishable from MF1
실험적: MF1 multiple ascending doses (healthy)
Once-daily oral doses of MF1 for 7 days in healthy male adults (Part B).
Oral administration of MF1
위약 비교기: Placebo multiple doses (healthy)
Once-daily oral doses of placebo for 7 days in healthy male adults (Part B).
Indistinguishable from MF1
실험적: MF1 multiple doses (Parkinson's disease)
Once-daily oral doses of MF1 for 14 days in patients with Parkinson's disease (Part C).
Oral administration of MF1

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Number of participants with treatment-emergent adverse events and serious adverse events
기간: 12 days from last dosing
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
기간: 5 days after last dosing
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
기간: Time Frame: Pre-dose through 5 days after last dosing
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
기간: Pre-dose through 5 days after last dosing
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
기간: Pre-dose through 24 hours after last dosing
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

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2026년 6월 1일

기본 완료 (추정된)

2028년 9월 30일

연구 완료 (추정된)

2028년 10월 31일

연구 등록 날짜

최초 제출

2026년 6월 9일

QC 기준을 충족하는 최초 제출

2026년 6월 18일

처음 게시됨 (실제)

2026년 6월 24일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 6월 24일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 6월 18일

마지막으로 확인됨

2026년 6월 1일

추가 정보

이 연구와 관련된 용어

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

예

IPD 계획 설명

De-identified individual participant data (IPD) underlying the primary and key secondary outcome analyses may be shared with qualified researchers and collaborating pharmaceutical companies for the purpose of further development of the investigational product and related scientific research. IPD will be made available after completion of the primary analysis and publication of the main results of this trial, subject to approval by the investigator. Data will be provided in a de-identified format in which direct identifiers are removed and indirect identifiers are processed according to our institutional data protection policy, so that individual participants cannot reasonably be re-identified. Access to IPD will require a written research proposal, a data use agreement specifying the scope of use, data security measures, and prohibition of attempts at re-identification or unauthorized sharing of the data.

IPD 공유 지원 정보 유형

  • 연구_프로토콜
  • 수액
  • ICF
  • ANALYTIC_CODE
  • CSR

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

아니

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

구독하다