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A Phase I Study in Healthy Volunteers and Parkinson's Disease (PD) Patients.

14. september 2026 opdateret af: Mission Therapeutics

A Multi-part, Adaptive Phase 1, First Time in Human Study in Healthy Volunteers to Assess Safety, Tolerability and Pharmacokinetics (PK) Following Single Ascending Dose (SAD) and Multiple Ascending Doses (MAD) of MTX325, Including Option to Assess: a Single Dose in Elderly Participants; Multiple Doses in Patients With Parkinson's Disease (PD); Central Nervous System (CNS) Penetration, Biodistribution, and Biomarkers; and the Effect of Food on PK.

The goal of this trial is to learn if MTX325 can be developed as a potential disease-modifying treatment for Parkinson's Disease. Parts 1-4 complete, Part 5 (multiple doses in patients with Parkinson's Disease) in progress.

Studieoversigt

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

106

Fase

  • Fase 1

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

      • Adelaide, Australien
        • Ikke rekrutterer endnu
        • The Royal Adelaide Hospital
        • Ledende efterforsker:
          • Thomas E Kimber, Professor
      • Melbourne, Australien
        • Ikke rekrutterer endnu
        • Monash Health, Kingston Centre
        • Ledende efterforsker:
          • Niro Vijiaratnam, Dr
      • Melbourne, Australien
        • Rekruttering
        • Doherty Clinical Trials
        • Ledende efterforsker:
          • Thomas Frisby, Dr
      • Cambridge, Det Forenede Kongerige
        • Rekruttering
        • Cambridge Biomedical Research Centre, Cambridge
        • Ledende efterforsker:
          • Caroline Williams-Gray, Dr
      • Glasgow, Det Forenede Kongerige
        • Ikke rekrutterer endnu
        • Neuroclnx, Glasgow
        • Ledende efterforsker:
          • David Anderson, Dr
      • Liverpool, Det Forenede Kongerige
        • Rekruttering
        • Royal Liverpool University Hospital
        • Ledende efterforsker:
          • Antonella Macherello, Dr
      • London, Det Forenede Kongerige
        • Afsluttet
        • Parexel
      • London, Det Forenede Kongerige
        • Afsluttet
        • Perceptive
      • Merthyr Tydfil, Det Forenede Kongerige
        • Afsluttet
        • Simbec-Orion
      • Newcastle, Det Forenede Kongerige
        • Rekruttering
        • Freeman Hospital, Newcastle Upon Tyne
        • Ledende efterforsker:
          • Dr David Ledingham, Dr
      • Plymouth, Det Forenede Kongerige
        • Rekruttering
        • University Hospitals Plymouth NHS Trust, Derriford Hospital
        • Ledende efterforsker:
          • Stephen Mullin, Dr
      • Salford, Det Forenede Kongerige
        • Rekruttering
        • Salford Hospital (Manchester)
        • Ledende efterforsker:
          • Monty Silverdale, Professor
      • Southampton, Det Forenede Kongerige
        • Rekruttering
        • Southampton General Hospital, Southampton
        • Ledende efterforsker:
          • Boyd Ghosh, Dr
      • Lille, Frankrig
        • Rekruttering
        • Centre Hospitalier Universitaire de Lille, Institut Cœur Poumon
        • Ledende efterforsker:
          • Prof David Davos, Professor
      • Paris, Frankrig
        • Ikke rekrutterer endnu
        • Hôpital Henri-Mondor (Créteil, AP-HP), Paris
        • Ledende efterforsker:
          • Philippe Remy, Dr
      • Paris, Frankrig
        • Ikke rekrutterer endnu
        • Pitie-Salpetriere Hospital, Paris
        • Ledende efterforsker:
          • Louise-Laure Mariani, Professor
      • Toulouse, Frankrig
        • Ikke rekrutterer endnu
        • Centre Hospitalier Universitaire (CHU) de Toulouse, Toulouse
        • Ledende efterforsker:
          • Olivier Rascol, Professor
      • Dresden, Tyskland
        • Ikke rekrutterer endnu
        • Technische Universitaet Dresden, Dresden
        • Ledende efterforsker:
          • Bjoern Falkenburger, Dr

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ja

Beskrivelse

PART 5 - Currently recruiting in Parkinson's Disease Patients

Inclusion Criteria

  1. Male and female participants aged ≥ 40 to ≤ 75 years.
  2. Male or female participants. Female participants of childbearing potential must be willing to use highly effective forms of contraception (refer to Section 9.7.1 for details on highly effective methods of contraception and definitions of women of childbearing potential and of fertile men):
  3. Body mass index (BMI) between 18 and 34.0 kg/m2, inclusive.
  4. If being treated with selective serotonin reuptake inhibitors (SSRIs) for anxiety/ depression, must be on a stable dose for 60 days prior to Day -1 and must remain on that dose for the remainder of the study.
  5. Must have had other causes of Parkinsonism excluded
  6. No clinically significant history of previous allergy/ sensitivity to MTX325 or any of the excipients contained within the IMP.
  7. Participant with a negative urinary drugs of abuse (DOA) screen (excluding alcohol).
  8. Participant with negative human immunodeficiency virus (HIV), hepatitis B surface antigen [HbsAg]) and hepatitis C virus antibody (HCV Ab) test results at Screening.
  9. No history of torsade de pointes or long QT syndrome and no heart failure, hypokalaemia, or any other additional cardiac risk factors if judged clinically significant in the opinion of the Investigator.
  10. No clinically significant abnormalities in vital signs during the screening period.
  11. Able to perform all protocol assessments and comply with the study visit schedule.
  12. Able and willing to provide informed consent.
  13. Clinically established PD as per MDS Criteria[
  14. Absence of dementia as shown by a baseline Montreal Cognitive Assessment (MoCA®)

Exclusion Criteria

  1. A clinically significant history of gastrointestinal disorders or any significant medical conditions that would be likely to influence IMP absorption, or evidence of clinically significant central nervous system, respiratory, cardiovascular or metabolic dysfunction or other significant medical conditions with potential to impact participant safety or hinder interpretation of study results.
  2. Clinically significant neurologic disorder other than PD, including history of stroke within 12 months of Screening, seizure within 5 years of Screening, or head trauma with loss of consciousness within 6 months of Screening.
  3. Those with known PD risk genes (per medical history).
  4. Reside in a nursing home or assisted care facility.
  5. No more than 2 PD related freezing episodes or falls in the past 6 months.
  6. Any condition that may predispose participant to complications or technical difficulty with lumbar puncture, in the opinion of the Investigator.
  7. Participants who have conditions that would preclude a lumbar puncture (LP), such as a local infection at the site
  8. Participant who has a history of clinically significant hypersensitivity to local anaesthesia, or its derivatives used during CSF collection or to any medication used to prepare the area of LP.
  9. Reports having experienced suicidal ideation (Type 4 on 5 on the Columbia-Suicide Severity Rating Scale [C-SSRS©]
  10. Participants should not be in receipt of any known MATE2k substrates
  11. Plan to receive or administration of rotigotine, catechol-o-methyltransferase (COMT) inhibitors (e.g., entacapone, tolcapone or opicapone), neuroleptics, venlafaxine, NMN, nicotinamide riboside or non-selective Monoamine oxidase [MAO] inhibitors within 7 days or 5 half-lives (whichever is longer) prior to first dose and during the study conduct.
  12. Clinically significant abnormal test results for serum biochemistry, haematology, coagulation and/or urine analyses as determined within 45 days before first dose of IMP.
  13. A clinically significant history of gastrointestinal disorder likely to influence IMP absorption.
  14. Participant has any condition that, in the opinion of the investigator, is clinically significant and may put the participant at greater safety risk, influence response to study product, or interfere with study assessment.
  15. Inability to communicate well with the Investigators.
  16. Participation (dosed) in a NCE clinical study within the previous 3 months or five half lives
  17. Donation of 450 mL or more blood within the 3 months before the first dose of IMP.
  18. Female participants who are pregnant, breastfeeding or lactating.
  19. Clinically significant abnormalities in 12-lead electrocardiogram (ECG)
  20. Participants with recent COVID-19 infection without resolution of symptoms
  21. Participants who have received a COVID-19 vaccine injection from the Screening visit up to first dose of IMP
  22. A clinically significant history of drug or alcohol abuse within the past 2 years.
  23. Currently prescribed treatment for Parkinson's Disease symptoms.
  24. Any history of allergy/atopy including drug-induced allergy history of severe cutaneous adverse reaction or other type 4/delayed-type hypersensitivity.
  25. Current or previous history of eosinophilia with either unknown cause, or where known, the inciting factor is not removed or avoided.
  26. Previous history of Erythema Multiforme and/or identified current risk factor(s) for Erythema Multiforme
  27. Participant has any contra-indication to PET or MRI as determined by screening procedures and PET and MRI safety questionnaires as conducted by the site.
  28. Clinically significant history of previous allergy/ sensitivity to [18F] FDG.
  29. Participants who have had previous exposure to ionizing radiation
  30. Chronic kidney disease defined as glomerular filtration rate (GFR) 1.5 × the upper limit of normal (ULN) or ALT or AST >3 × ULN or if participant has Child-Pugh Class C cirrhosis or equivalent severe hepatic impairment.
  31. Hepatic disease or altered liver function as defined by total bilirubin >1.5 × the upper limit of normal (ULN) or ALT or AST >3 × ULN or if participant has Child-Pugh Class C cirrhosis or equivalent severe hepatic impairment.
  32. Patients with uncontrolled type I or type II diabetes (insulin or non-insulin dependent).
  33. Current symptomatic Hay fever or any history of hay fever involving more than nose and eyes.

PART 1, 2 ,4 - COMPLETED

Inclusion Criteria:

  1. Healthy male and female (Part 1-2 only) participant, aged ≥ 18 to ≤ 55 years.
  2. Female participant of childbearing potential (Part 1-2 only)
  3. Female participant of non-childbearing potential (Part 1-2 only).
  4. Female participant (Part 1-2 only) with a negative pregnancy test at Screening visit.
  5. Female participant of menopausal status (Part 1-2 only) confirmed by demonstrating at Screening that the serum level of the follicle stimulating hormone (FSH) falls within the respective pathology reference range.
  6. Male participant (and partner of childbearing potential) willing to use a highly effective method of contraception
  7. Participant with a body weight of at least 50.0 kg and body mass index (BMI) of 1832 kg/m2. BMI = body weight (kg) / [height (m)]2.
  8. No clinically significant history of previous allergy / sensitivity to MTX325 or any of the excipients contained within the IMP.
  9. No clinically significant abnormal test results for serum biochemistry, haematology, coagulation
  10. Participant with a negative urinary drugs of abuse (DOA) screen (including alcohol) test results, determined within 35 days (45 days Part 4 only) before first dose of IMP.
  11. Participant with negative human immunodeficiency virus (HIV), hepatitis B surface antigen [HbsAg]) and hepatitis C virus antibody (HCV Ab) test results at Screening.
  12. No clinically significant abnormalities in 12-lead electrocardiogram (ECG)
  13. No clinically significant abnormalities in vital signs
  14. Participant must be available to complete the study (including all follow-up visits).
  15. Participant must satisfy an Investigator about his/her fitness to participate in the study.
  16. Participant must provide written informed consent to participate in the study.
  17. Participants with a negative COVID-19 test on admission (if required).
  18. Part 4 only: Participant with normal MRI performed within 3 months of dosing, as judged by the investigator.

Part 4 - COMPLETED

  1. Participants who have had previous exposure to ionizing radiation from research studies
  2. Participant has any contraindication to arterial line insertion
  3. Inability to lie supine for up to 120 mins for PET procedures.
  4. Participant has any contra-indication to MRI as determined by screening procedures and MRI safety questionnaire
  5. Participant suffers from claustrophobia or needle phobia.
  6. Any history of allergy/atopy including drug-induced allergy or any history of severe cutaneous adverse reaction or other type 4/delayed-type hypersensitivity.
  7. Current or previous history of eosinophilia with either unknown cause, or where known, the inciting factor is not removed or avoided.
  8. Previous history of Erythema Multiforme and/or identified current risk factor(s) for Erythema

Part 3 - COMPLETED

  1. Healthy male and female participant, ≥ 65 years of age.
  2. Female participant of non-childbearing potential.
  3. Female participant of menopausal status confirmed by demonstrating at Screening that the serum level of the follicle stimulating hormone (FSH) falls within the respective pathology reference range.
  4. Male participant (and partner of childbearing potential) willing to use a highly effective method of contraception
  5. Participant with a body weight of at least 50.0 kg and BMI of 18-32 kg/m2.
  6. No clinically significant history of previous allergy / sensitivity to MTX325 or any of the excipients contained within the IMP.
  7. No clinically significant abnormal test results for serum biochemistry, haematology and/or urine analyses determined within 35 days before first dose of IMP.
  8. Participant with an estimated glomerular filtration rate (eGFR) calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation >60 mL/min/1.73 m2.
  9. Participant with a negative urinary drugs of abuse (DOA)
  10. Participant with negative human immunodeficiency virus (HIV), hepatitis B surface antigen [HbsAg]) and hepatitis C virus antibody (HCV Ab) test results at Screening.
  11. No clinically significant abnormalities in 12-lead electrocardiogram (ECG)
  12. No clinically significant abnormalities in vital signs
  13. Participant must be available to complete the study (including all follow-up visits).
  14. Participant must satisfy an Investigator about his/her fitness to participate in the study.
  15. Participant must provide written informed consent to participate in the study.

Exclusion Criteria (Part 1, 2, 4) - COMPLETED

  1. A clinically significant history of gastrointestinal disorder likely to influence IMP absorption.
  2. Use of prescription or non-prescription drugs, excluding allowable drugs and contraception
  3. Reports having experienced suicidal ideation (Type 4 on 5 on the Columbia-Suicide Severity Rating Scale [C-SSRS©] him/herself or others - Part 2 only.
  4. Evidence of renal, hepatic, central nervous system, respiratory, cardiovascular or metabolic dysfunction.
  5. A clinically significant history of drug or alcohol abuse (defined as the consumption of more than 14 units
  6. Inability to communicate well with the Investigators
  7. Participation (dosed) in a New Chemical Entity (NCE) clinical study within the previous 3 months or five half-lives
  8. Donation of 450 mL or more blood within the 3 months before the first dose of IMP.
  9. Vegans, vegetarians or other dietary restrictions (Part 1 food effect evaluation, only).
  10. Users of nicotine products
  11. Participants with an excessive habitual daily intake of caffeine
  12. Female participants who are pregnant, breastfeeding or lactating (Part 1-2 only).
  13. Participants with veins unsuitable for venepuncture and cannulation.
  14. Participants with recent COVID-19 infection without resolution of symptoms
  15. Participants who have received a COVID-19 vaccine injection

Part 1 Treatment Period 2a (CSF sampling) Cohort(s) only:

  1. Participants who have criteria that would preclude a lumbar puncture (LP)
  2. Participant who has a history of clinically significant hypersensitivity to local anaesthesia
  3. Participant who has a history of clinically significant or major back pathology (lumbar) surgery

Part 4 - COMPLETED

  1. Participants who have had previous exposure to ionizing radiation from research studies, such that, in combination with the exposure from this study, their exposure will be >10 mSv for the previous 12 months.
  2. Participant has any contraindication to arterial line insertion
  3. Inability to lie supine for up to 120 mins for PET procedures.
  4. Participant has any contra-indication to MRI
  5. Participant suffers from claustrophobia or needle phobia.
  6. Any history of allergy/atopy
  7. Current or previous history of eosinophilia with either unknown cause, or where known, the inciting factor is not removed or avoided.
  8. Previous history of Erythema Multiforme and/or identified current risk factor(s) for Erythema Multiforme

Part 3 - COMPLETED

  1. A clinically significant history of gastrointestinal disorder likely to influence IMP absorption.
  2. Evidence of febrile illness within 1 week of first dose of IMP.
  3. Use of prescription or non-prescription drugs, including vitamins, herbal and dietary supplements or any medication known to prolong the QT/QTc interval within 35 days or 5 half-lives
  4. Evidence of clinically significant renal, hepatic, central nervous system, respiratory, cardiovascular or metabolic dysfunction.
  5. History of torsade de pointes, heart failure, hypokalaemia, long QT syndrome or any other additional cardiac risk factors.
  6. A clinically significant history of drug or alcohol abuse
  7. Participants with an excess habitual daily intake of caffeine
  8. Inability to communicate well with the Investigators
  9. Participation in a NCE clinical study within the previous 3 months or five half-lives
  10. Donation of 450 mL or more blood within the 3 months before the first dose of IMP.
  11. Participants who have received a COVID-19 vaccine injection within 35 days prior to first dose of IMP.
  12. Users of nicotine products
  13. Participants with veins unsuitable for venepuncture and cannulation.
  14. Participants with recent COVID-19 infection with resolution of symptoms

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Aktiv komparator: Parts 1 - 4
MTX325 Active Oral Capsules - 1.5mg - 100mg These 4 parts are SAD, MAD, Elderly and PET biodistribution.
MTX325
Placebo komparator: Parts 1 - 3, and 5
MTX325 Placebo Capsules - 50mg. Parts 1-3 are SAD, MAD and Elderly cohorts. Matched strength placebo capsules in Part 5 (PD participants)
Placebo
Aktiv komparator: Part 4
MTX325 Capsules - 20mg. Radiolabelled MTX325 solution for injection. This is a healthy volunteer PET study. Not placebo controlled.
MTX325
Aktiv komparator: Part 5
MTX325 - 20 mg Capsules
MTX325

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Safety and Tolerability
Tidsramme: up to 28 days post dose

To assess the safety and tolerability of single ascending doses of MTX325, when administered to participants. Adverse Events will be collected. The following vital signs will be measured to form the safety and tolerability outcome- blood pressure, heart rate, oral body temperature, respiratory rate. Laboratory Safety analyses will include biochemistry, haematology, and urinalysis. 12-lead ECG will be performed to collect heart rate, RR interval, PR interval, QRS width, QT interval, and QTcF interval.

All participants who receive at least 1 dose of IMP will be included in the safety analysis.

up to 28 days post dose
Brain Biodistribution
Tidsramme: MRI performed during screening period and PET performed on Day 1
To investigate the brain biodistribution in MTX325. This part of the protocol is intended to ascertain the biodistribution of MTX325 in the brain. Radiolabelled MTX325 will be injected and PET images taken to determine the whole brain and regions of interest distribution volume. MRI images will be performed to be able to co-register regions of interest.
MRI performed during screening period and PET performed on Day 1
Safety and Tolerability
Tidsramme: up to 28 days post dose

To evaluate the safety and tolerability of MTX325 in untreated participants with mild to moderate idiopathic PD. Adverse Events will be collected. The following vital signs will be measured to form the safety and tolerability outcome - blood pressure, heart rate, oral body temperature, respiratory rate. Laboratory Safety analyses will include biochemistry, haematology, and urinalysis. 12-lead ECG will be performed to collect heart rate, RR interval, PR interval, QRS width, QT interval, and QTcF interval.

All participants who receive at least 1 dose of IMP will be included in the safety analysis.

up to 28 days post dose

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

30. november 2023

Primær færdiggørelse (Anslået)

30. juni 2027

Studieafslutning (Anslået)

27. december 2027

Datoer for studieregistrering

Først indsendt

6. maj 2026

Først indsendt, der opfyldte QC-kriterier

1. juni 2026

Først opslået (Faktiske)

5. juni 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

15. september 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

14. september 2026

Sidst verificeret

1. juli 2026

Mere information

Begreber relateret til denne undersøgelse

Plan for individuelle deltagerdata (IPD)

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