UMIN-CTR Clinical Trial

Basic information
Public title	Establishment of disease specific iPS cells and analysis of the pathology
Acronym	disease specific iPS cells
Scientific Title	Establishment of disease specific iPS cells and analysis of the pathology
Scientific Title:Acronym	disease specific iPS cells
Region	Japan

Condition
Condition	Spinal Muscular Atrophy, Muscular dystrophy, Normal tension glaucoma, Primary immunodeficiency disease, Li-Fraumeni syndrome, Shwachman-Diamond syndrome, Drug-induced acute pancreatitis, Lung-disease
Classification by specialty	Hepato-biliary-pancreatic medicine Pneumology Hematology and clinical oncology Neurology Pediatrics Ophthalmology Orthopedics Adult Child
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To establish disease specific iPS cells and its disease model systems. To evaluate the pathological mechanisms or exploring therapeutic agents.
Basic objectives2	Others
Basic objectives -Others	Pathology
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Analysis of the phenotype of differentiated cells derived from patients
Key secondary outcomes

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Diagnosis
Type of intervention	Maneuver
Interventions/Control_1	harvest skin sample from patients
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	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	Patients who have been confirmed clinically and molecularly biology with refractory disease. Patients who have established disease-specific iPS cells after informed consent at an external research institution.
Key exclusion criteria	Patients who are judged inappropriate by the doctor's judgment. Patients infected with human immunodeficiency virus, human hepatitis B virus, and human hepatitis C virus.
Target sample size	40

Research contact person
Name of lead principal investigator	1st name Michinori Middle name Last name Funato
Organization	National Hospital Organization, Nagara Medical Center
Division name	Department of Clinical Research
Zip code	502-8558
Address	1300-7, Nagara, Gifu
TEL	058-232-7755
Email	mfunato@mac.com

Public contact
Name of contact person	1st name Michinori Middle name Last name Funato
Organization	National Hospital Organization, Nagara Medical Center
Division name	Department of Clinical Research
Zip code	502-8558
Address	1300-7, Nagara, Gifu
TEL	058-232-7755
Homepage URL
Email	mfunato@mac.com

Sponsor
Institute	National Hospital Organization, Nagara Medical Center
Institute
Department

Funding Source
Organization	National Hospital Organization, Nagara Medical Center
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

Other related organizations
Co-sponsor	Gifu Pharmaceutical University, Osaka Toneyama Medical Center, Tokyo Medical Center, Kobe University, Kanazawa University, Kyoto University, Gifu University
Name of secondary funder(s)

IRB Contact (For public release)
Organization	National Hospital Organization, Nagara Medical Center
Address	1300-7, Nagara, Gifu
Tel	058-232-7755
Email	mfunato@mac.com

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	2020 Year 01 Month 20 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	2019 Year 10 Month 01 Day
Date of IRB	2019 Year 11 Month 18 Day
Anticipated trial start date	2019 Year 11 Month 18 Day
Last follow-up date	2025 Year 09 Month 30 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other

Other related information

In this study, somatic cells will be collected after obtaining consent from the patient or his / her sponsor. At that time, as much as possible, somatic cells use blood or milk teeth after natural tooth extraction, but due to problems such as establishment efficiency, skin fragments may be collected according to the skin biopsy method. After that, in the culture room of the research building, reprogramming factors are introduced into patient somatic cells (skin fibroblasts, blood cells, milk teeth, etc.) to establish patient disease-specific iPS cells and induce differentiation into diseased cells. Analyze pathological conditions and search for therapeutic drugs.

Management information
Registered date	2020 Year 01 Month 12 Day
Last modified on	2020 Year 01 Month 12 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000044635