A Phase I Study in Healthy Volunteers and Parkinson's Disease (PD) Patients.
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.
調査の概要
研究の種類
入学 (推定)
段階
- フェーズ 1
連絡先と場所
研究連絡先
- 名前:Sarah J Fritchley, PhD
- メール:sfritchley@missiontherapeutics.com
研究場所
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Cambridge、イギリス
- 募集
- Cambridge Biomedical Research Centre, Cambridge
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主任研究者:
- Caroline Williams-Gray, Dr
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Glasgow、イギリス
- まだ募集していません
- Neuroclnx, Glasgow
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主任研究者:
- David Anderson, Dr
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Liverpool、イギリス
- 募集
- Royal Liverpool University Hospital
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主任研究者:
- Antonella Macherello, Dr
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London、イギリス
- 完了
- Parexel
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London、イギリス
- 完了
- Perceptive
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Merthyr Tydfil、イギリス
- 完了
- Simbec-Orion
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Newcastle、イギリス
- 募集
- Freeman Hospital, Newcastle Upon Tyne
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主任研究者:
- Dr David Ledingham, Dr
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Plymouth、イギリス
- 募集
- University Hospitals Plymouth NHS Trust, Derriford Hospital
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主任研究者:
- Stephen Mullin, Dr
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Salford、イギリス
- 募集
- Salford Hospital (Manchester)
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主任研究者:
- Monty Silverdale, Professor
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Southampton、イギリス
- 募集
- Southampton General Hospital, Southampton
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主任研究者:
- Boyd Ghosh, Dr
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Adelaide、オーストラリア
- まだ募集していません
- The Royal Adelaide Hospital
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主任研究者:
- Thomas E Kimber, Professor
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Melbourne、オーストラリア
- まだ募集していません
- Monash Health, Kingston Centre
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主任研究者:
- Niro Vijiaratnam, Dr
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Melbourne、オーストラリア
- 募集
- Doherty Clinical Trials
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主任研究者:
- Thomas Frisby, Dr
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Dresden、ドイツ
- まだ募集していません
- Technische Universitaet Dresden, Dresden
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主任研究者:
- Bjoern Falkenburger, Dr
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Lille、フランス
- 募集
- Centre Hospitalier Universitaire de Lille, Institut Cœur Poumon
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主任研究者:
- Prof David Davos, Professor
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Paris、フランス
- まだ募集していません
- Hôpital Henri-Mondor (Créteil, AP-HP), Paris
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主任研究者:
- Philippe Remy, Dr
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Paris、フランス
- まだ募集していません
- Pitie-Salpetriere Hospital, Paris
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主任研究者:
- Louise-Laure Mariani, Professor
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Toulouse、フランス
- まだ募集していません
- Centre Hospitalier Universitaire (CHU) de Toulouse, Toulouse
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主任研究者:
- Olivier Rascol, Professor
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参加基準
適格基準
就学可能な年齢
- 大人
- 高齢者
健康ボランティアの受け入れ
説明
PART 5 - Currently recruiting in Parkinson's Disease Patients
Inclusion Criteria
- Male and female participants aged ≥ 40 to ≤ 75 years.
- 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):
- Body mass index (BMI) between 18 and 34.0 kg/m2, inclusive.
- 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.
- Must have had other causes of Parkinsonism excluded
- No clinically significant history of previous allergy/ sensitivity to MTX325 or any of the excipients contained within the IMP.
- Participant with a negative urinary drugs of abuse (DOA) screen (excluding alcohol).
- Participant with negative human immunodeficiency virus (HIV), hepatitis B surface antigen [HbsAg]) and hepatitis C virus antibody (HCV Ab) test results at Screening.
- 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.
- No clinically significant abnormalities in vital signs during the screening period.
- Able to perform all protocol assessments and comply with the study visit schedule.
- Able and willing to provide informed consent.
- Clinically established PD as per MDS Criteria[
- Absence of dementia as shown by a baseline Montreal Cognitive Assessment (MoCA®)
Exclusion Criteria
- 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.
- 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.
- Those with known PD risk genes (per medical history).
- Reside in a nursing home or assisted care facility.
- No more than 2 PD related freezing episodes or falls in the past 6 months.
- Any condition that may predispose participant to complications or technical difficulty with lumbar puncture, in the opinion of the Investigator.
- Participants who have conditions that would preclude a lumbar puncture (LP), such as a local infection at the site
- 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.
- Reports having experienced suicidal ideation (Type 4 on 5 on the Columbia-Suicide Severity Rating Scale [C-SSRS©]
- Participants should not be in receipt of any known MATE2k substrates
- 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.
- Clinically significant abnormal test results for serum biochemistry, haematology, coagulation and/or urine analyses as determined within 45 days before first dose of IMP.
- A clinically significant history of gastrointestinal disorder likely to influence IMP absorption.
- 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.
- Inability to communicate well with the Investigators.
- Participation (dosed) in a NCE clinical study within the previous 3 months or five half lives
- Donation of 450 mL or more blood within the 3 months before the first dose of IMP.
- Female participants who are pregnant, breastfeeding or lactating.
- Clinically significant abnormalities in 12-lead electrocardiogram (ECG)
- Participants with recent COVID-19 infection without resolution of symptoms
- Participants who have received a COVID-19 vaccine injection from the Screening visit up to first dose of IMP
- A clinically significant history of drug or alcohol abuse within the past 2 years.
- Currently prescribed treatment for Parkinson's Disease symptoms.
- Any history of allergy/atopy including drug-induced allergy history of severe cutaneous adverse reaction or other type 4/delayed-type hypersensitivity.
- Current or previous history of eosinophilia with either unknown cause, or where known, the inciting factor is not removed or avoided.
- Previous history of Erythema Multiforme and/or identified current risk factor(s) for Erythema Multiforme
- 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.
- Clinically significant history of previous allergy/ sensitivity to [18F] FDG.
- Participants who have had previous exposure to ionizing radiation
- 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.
- 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.
- Patients with uncontrolled type I or type II diabetes (insulin or non-insulin dependent).
- Current symptomatic Hay fever or any history of hay fever involving more than nose and eyes.
PART 1, 2 ,4 - COMPLETED
Inclusion Criteria:
- Healthy male and female (Part 1-2 only) participant, aged ≥ 18 to ≤ 55 years.
- Female participant of childbearing potential (Part 1-2 only)
- Female participant of non-childbearing potential (Part 1-2 only).
- Female participant (Part 1-2 only) with a negative pregnancy test at Screening visit.
- 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.
- Male participant (and partner of childbearing potential) willing to use a highly effective method of contraception
- 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.
- No clinically significant history of previous allergy / sensitivity to MTX325 or any of the excipients contained within the IMP.
- No clinically significant abnormal test results for serum biochemistry, haematology, coagulation
- 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.
- Participant with negative human immunodeficiency virus (HIV), hepatitis B surface antigen [HbsAg]) and hepatitis C virus antibody (HCV Ab) test results at Screening.
- No clinically significant abnormalities in 12-lead electrocardiogram (ECG)
- No clinically significant abnormalities in vital signs
- Participant must be available to complete the study (including all follow-up visits).
- Participant must satisfy an Investigator about his/her fitness to participate in the study.
- Participant must provide written informed consent to participate in the study.
- Participants with a negative COVID-19 test on admission (if required).
- Part 4 only: Participant with normal MRI performed within 3 months of dosing, as judged by the investigator.
Part 4 - COMPLETED
- Participants who have had previous exposure to ionizing radiation from research studies
- Participant has any contraindication to arterial line insertion
- Inability to lie supine for up to 120 mins for PET procedures.
- Participant has any contra-indication to MRI as determined by screening procedures and MRI safety questionnaire
- Participant suffers from claustrophobia or needle phobia.
- Any history of allergy/atopy including drug-induced allergy or any history of severe cutaneous adverse reaction or other type 4/delayed-type hypersensitivity.
- Current or previous history of eosinophilia with either unknown cause, or where known, the inciting factor is not removed or avoided.
- Previous history of Erythema Multiforme and/or identified current risk factor(s) for Erythema
Part 3 - COMPLETED
- Healthy male and female participant, ≥ 65 years of age.
- Female participant of non-childbearing potential.
- 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.
- Male participant (and partner of childbearing potential) willing to use a highly effective method of contraception
- Participant with a body weight of at least 50.0 kg and BMI of 18-32 kg/m2.
- No clinically significant history of previous allergy / sensitivity to MTX325 or any of the excipients contained within the IMP.
- No clinically significant abnormal test results for serum biochemistry, haematology and/or urine analyses determined within 35 days before first dose of IMP.
- 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.
- Participant with a negative urinary drugs of abuse (DOA)
- Participant with negative human immunodeficiency virus (HIV), hepatitis B surface antigen [HbsAg]) and hepatitis C virus antibody (HCV Ab) test results at Screening.
- No clinically significant abnormalities in 12-lead electrocardiogram (ECG)
- No clinically significant abnormalities in vital signs
- Participant must be available to complete the study (including all follow-up visits).
- Participant must satisfy an Investigator about his/her fitness to participate in the study.
- Participant must provide written informed consent to participate in the study.
Exclusion Criteria (Part 1, 2, 4) - COMPLETED
- A clinically significant history of gastrointestinal disorder likely to influence IMP absorption.
- Use of prescription or non-prescription drugs, excluding allowable drugs and contraception
- 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.
- Evidence of renal, hepatic, central nervous system, respiratory, cardiovascular or metabolic dysfunction.
- A clinically significant history of drug or alcohol abuse (defined as the consumption of more than 14 units
- Inability to communicate well with the Investigators
- Participation (dosed) in a New Chemical Entity (NCE) clinical study within the previous 3 months or five half-lives
- Donation of 450 mL or more blood within the 3 months before the first dose of IMP.
- Vegans, vegetarians or other dietary restrictions (Part 1 food effect evaluation, only).
- Users of nicotine products
- Participants with an excessive habitual daily intake of caffeine
- Female participants who are pregnant, breastfeeding or lactating (Part 1-2 only).
- Participants with veins unsuitable for venepuncture and cannulation.
- Participants with recent COVID-19 infection without resolution of symptoms
- Participants who have received a COVID-19 vaccine injection
Part 1 Treatment Period 2a (CSF sampling) Cohort(s) only:
- Participants who have criteria that would preclude a lumbar puncture (LP)
- Participant who has a history of clinically significant hypersensitivity to local anaesthesia
- Participant who has a history of clinically significant or major back pathology (lumbar) surgery
Part 4 - COMPLETED
- 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.
- Participant has any contraindication to arterial line insertion
- Inability to lie supine for up to 120 mins for PET procedures.
- Participant has any contra-indication to MRI
- Participant suffers from claustrophobia or needle phobia.
- Any history of allergy/atopy
- Current or previous history of eosinophilia with either unknown cause, or where known, the inciting factor is not removed or avoided.
- Previous history of Erythema Multiforme and/or identified current risk factor(s) for Erythema Multiforme
Part 3 - COMPLETED
- A clinically significant history of gastrointestinal disorder likely to influence IMP absorption.
- Evidence of febrile illness within 1 week of first dose of IMP.
- 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
- Evidence of clinically significant renal, hepatic, central nervous system, respiratory, cardiovascular or metabolic dysfunction.
- History of torsade de pointes, heart failure, hypokalaemia, long QT syndrome or any other additional cardiac risk factors.
- A clinically significant history of drug or alcohol abuse
- Participants with an excess habitual daily intake of caffeine
- Inability to communicate well with the Investigators
- Participation in a NCE clinical study within the previous 3 months or five half-lives
- Donation of 450 mL or more blood within the 3 months before the first dose of IMP.
- Participants who have received a COVID-19 vaccine injection within 35 days prior to first dose of IMP.
- Users of nicotine products
- Participants with veins unsuitable for venepuncture and cannulation.
- Participants with recent COVID-19 infection with resolution of symptoms
研究計画
研究はどのように設計されていますか?
デザインの詳細
- 主な目的:処理
- 割り当て:ランダム化
- 介入モデル:並列代入
- マスキング:なし(オープンラベル)
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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アクティブコンパレータ:Parts 1 - 4
MTX325 Active Oral Capsules - 1.5mg - 100mg These 4 parts are SAD, MAD, Elderly and PET biodistribution.
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MTX325
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プラセボコンパレーター: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)
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プラセボ
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アクティブコンパレータ:Part 4
MTX325 Capsules - 20mg.
Radiolabelled MTX325 solution for injection.
This is a healthy volunteer PET study.
Not placebo controlled.
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MTX325
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アクティブコンパレータ:Part 5
MTX325 - 20 mg Capsules
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MTX325
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この研究は何を測定していますか?
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Safety and Tolerability
時間枠:up to 28 days post dose
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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
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Brain Biodistribution
時間枠:MRI performed during screening period and PET performed on Day 1
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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.
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MRI performed during screening period and PET performed on Day 1
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Safety and Tolerability
時間枠:up to 28 days post dose
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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
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協力者と研究者
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- MTX325-101
- 2024-517310-15-01 (Ctis)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
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