UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000051823 |
|---|---|
| Receipt number | R000059103 |
| Scientific Title | Clinical studies to elucidate the molecular mechanisms associated with the pathogenesis of amyotrophic lateral sclerosis (ALS) and to identify effective drug targets |
| Date of disclosure of the study information | 2023/08/07 |
| Last modified on | 2023/08/04 12:18:18 |
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Basic information
Public title
Clinical studies to elucidate the molecular mechanisms associated with the pathogenesis of amyotrophic lateral sclerosis (ALS) and to identify effective drug targets
Acronym
Clinical research to elucidate ALS pathogenesis and to identify drug targets
Scientific Title
Clinical studies to elucidate the molecular mechanisms associated with the pathogenesis of amyotrophic lateral sclerosis (ALS) and to identify effective drug targets
Scientific Title:Acronym
Clinical research to elucidate ALS pathogenesis and to identify drug targets
Region
| Japan |
Condition
Condition
Amyotrophic lateral sclerosis
Classification by specialty
| Medicine in general | Neurology |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
The goal of this project is to identify the causes of ALS and to develop diagnostic and effective treatment methods for ALS and other neurological diseases similar to ALS by using blood, cerebrospinal fluid, urine, saliva, and feces of ALS patients.
Basic objectives2
Others
Basic objectives -Others
We aim to identify pathological changes that link acute cerebrospinal stress to RNA-related dynamics, and to identify trigger factors in the transition from early ALS disease to late cell death by using blood, cerebrospinal fluid, urine, saliva, and feces of ALS and control patients. This project is a step toward developing biomarkers and effective treatments for ALS.
Trial characteristics_1
Trial characteristics_2
Developmental phase
Not applicable
Assessment
Primary outcomes
1) Clinical symptoms
Clinical information (vital signs, i.e., height, weight, blood pressure, pulse, consciousness, and respiratory status, as well as personality and mood assessment) obtained during outpatient and inpatient examinations at departments including the Department of Neurology at Keio University Hospital. The information is obtained by interview about decision making regarding life extension and treatment options and family history, visual examination, palpation, percussion, and neurological findings including manual muscle strength test.
2) ALS Severity Scale (revised ALSFRS), Muscle Strength Rating Scale, Higher Functioning Rating Scale, Quality of Life Rating Scale, ADL Rating Scale
3) Related images that are judged to be necessary for normal medical care including CT images of the head and whole body, screening imaging tests for related diseases using isotopes, head MRI and other images including MRA, MRS, and SPECT images to evaluate cerebral blood flow. Clinical examination results commonly performed during medical care and examination process including blood sampling and cerebrospinal fluid sampling. Results of electrophysiological tests including nerve conduction studies, needle electromyography, and other tests of evoked potentials induced by electrical and magnetic stimulation methods, and results of other ALS-related physiological function tests including evaluations of respiratory, cardiac, and swallowing functions.
4) Sample analysis data
Various data obtained by molecular biological methods
Key secondary outcomes
Base
Study type
Observational
Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10
Eligibility
Age-lower limit
| 18 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
Subjects will be who visit Keio University Hospital from the date the study is approved to September 30, 2032 and who are willing to actively participate in this study, consisting of ALS patients (including suspected cases), patients with other neurodegenerative diseases including spinal muscular atrophy, primary lateral sclerosis, spinal and bulbar muscular atrophy(SBMA), Hirayama disease, spinocerebellar degeneration, Alzheimer's disease, dementia with Lewy bodies, Parkinson's disease, basal ganglia syndrome, frontotemporal dementia, Parkinson's disease, basal ganglia syndrome and frontotemporal dementia, patients with other diseases including multiple sclerosis, cerebrovascular disease, myasthenia gravis, neuropathy, myositis, paraneoplastic syndrome, hydrocephalus, traumatic brain and spinal cord injury, spinal cord diseases including cervical myelopathy, encephalomyelitis, brain tumor, spinal tumor, myelopathy, non-spinal cord tumors, and cavernous sinus disease), patients with non-neurological diseases, and healthy subjects. The age of eligibility is limited to 18 years of age or older. If it is difficult to obtain consent from the research subject him/herself due to physical or mental disability at the time of obtaining consent, consent may be obtained from a substitute who is objectively judged to be able to represent the subject's will and interests, and the subject will be enrolled after obtaining written consent on behalf of the subject by the substitute. Even if the research subject is capable of decision-making and normal judgment and has indicated his/her willingness to consent to this research, but the subject has difficulty giving a signature on the consent form due to physical or mental disability, he/she will be selected and enrolled as a research subject by obtaining a written consent form from a surrogate.
Key exclusion criteria
The case when the subject does not agree to participate in this study.
Target sample size
600
Research contact person
Name of lead principal investigator
| 1st name | Yoshinori |
| Middle name | |
| Last name | Nishimoto |
Organization
Keio University, School of Medicine
Division name
Department of Neurology
Zip code
160-8582
Address
35 Shinanomachi, Shinjuku-ku, Tokyo 160-8582, Japan
TEL
+81-3-3353-1211
ynishimo@keio.jp
Public contact
Name of contact person
| 1st name | Yoshinori |
| Middle name | |
| Last name | Nishimoto |
Organization
Keio University School of Medicine
Division name
Department of Neurology
Zip code
160-8582
Address
35 Shinanomachi, Shinjuku-ku, Tokyo 160-8582, Japan
TEL
+81-3-3353-1211
Homepage URL
ynishimo@keio.jp
Sponsor or person
Institute
Keio University, School of Medicine
Institute
Department
Personal name
Yoshinori Nishimoto
Funding Source
Organization
Ministry of Education, Culture, Sports, Science and Technology (MEXT)
Organization
Division
Category of Funding Organization
Japanese Governmental office
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
Name of secondary funder(s)
Japan Agency for Medical Research and Development (AMED)
Takeda Science Foundation
IRB Contact (For public release)
Organization
Keio University, School of Medicine
Address
35 Shinanomachi, Shinjuku-ku, Tokyo 160-8582, Japan
Tel
+81-3-3353-1211
med-rinri-jimu@adst.keio.ac.jp
Secondary IDs
Secondary IDs
NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW
Institutions
Institutions
Other administrative information
Date of disclosure of the study information
| 2023 | Year | 08 | Month | 07 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Open public recruiting
Date of protocol fixation
| 2022 | Year | 12 | Month | 02 | Day |
Date of IRB
| 2023 | Year | 01 | Month | 12 | Day |
Anticipated trial start date
| 2023 | Year | 01 | Month | 12 | Day |
Last follow-up date
| 2032 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Omics and transcriptome analysis for gene expression profiles, proteins and metabolites using PCR, qPCR, microarray and various molecular biological detection methods will be performed as molecular biological examinations of blood, cerebrospinal fluid, urine, saliva and stool collected from the subjects.
Genome analysis and profile analysis will be performed for detecting methylation and other epigenetics. Whole genome or specific genomic regions will also be analyzed. Genome analysis will be conducted in consideration of the "Ethical Guidelines for Human Genome/Genetic Analysis Research" of the Ministry of Education, Culture, Sports, Science and Technology, the Ministry of Health, Labor and Welfare, and the Ministry of Economy, Trade and Industry.
Management information
Registered date
| 2023 | Year | 08 | Month | 04 | Day |
Last modified on
| 2023 | Year | 08 | Month | 04 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000059103
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| Registered date | File name |
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| Research case data | |
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