UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Development of human cellular models for motor neuron diseases using disease-specific induced pluripotent stem (iPS) cells
Title of the study (Brief title)	Development of cellular models for motor neuron diseases from iPS cells
Region	Japan

Condition
Condition	Adult-onset motor neuron diseases (MND) including sporadic or familial amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA) type IV
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	In order to clarify pathomechanism underlying ALS/MND, patient-derived disease-specific iPS cells are generated and used to develop human cellular models reproducing the disease phenotype.
Basic objectives2	Others
Basic objectives -Others	Biological property of neural cells differentiated from ALS/MND-specific iPS cells
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Biological property of cultured neural cells differentiated from ALS/MND-specific iPS cells, such as cellular morphology, neurite elongation, synaptogenesis, and action potentials
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	20 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	- Clinically diagnosed as sporadic/familial (hereditary) ALS or spinal muscular atrophy type IV, based on the guide to diagnosis and medical care by the Ministry of Health, Labour and Welfare, Japan - Confirmed gene mutations in case of familial (hereditary) ALS - Obtained informed consent in writing
Key exclusion criteria	- Presence of dementia - Difficulty in sampling
Target sample size	20

Research contact person
Name of lead principal investigator	Masashi Aoki, MD, PhD
Organization	Tohoku University Graduate School of Medicine
Division name	Neurology
Address	1-1 Seiryo-machi, Aoba-ku, Sendai 980-8574, Japan
TEL	+81-22-717-7000
Email	admin@neurol.med.tohoku.ac.jp

Public contact
Name of contact person	Hitoshi Warita, MD, PhD
Organization	Tohoku University Graduate School of Medicine
Division name	Neurology
Address	1-1 Seiryo-machi, Aoba-ku, Sendai 980-8574, Japan
TEL	+81-22-717-7000
Homepage URL
Email	warita-ns@umin.net

Sponsor
Institute	Tohoku University
Institute
Department

Funding Source
Organization	Ministry of Education, Culture, Sports, Science and Technology (MEXT) of Japan
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	National Institute of Neuroscience, National Center of Neurology and Psychiatry (NCNP) and Department of Physiology, Keio University School of Medicine
Name of secondary funｄer(s)

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	東北大学病院（宮城県）

Other administrative information
Date of disclosure of the study information	2013 Year 09 Month 01 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2010 Year 10 Month 19 Day
Anticipated trial start date	2010 Year 11 Month 10 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Partially published
URL releasing results	https://www.ncbi.nlm.nih.gov/pubmed/26997647
Results
Other related information	- observational study - prospective study

Management information
Registered date	2013 Year 08 Month 21 Day
Last modified on	2018 Year 03 Month 06 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000013490