UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Molecular and therapeutic apploaches of autosomal recessive familial Alzheimer's disease using APPE693dlta-iPS cells.
Title of the study (Brief title)	Analysis of disease-causing mutant APP (E693delta) iPS cells
Region	Japan

Condition
Condition	Familial Alzheimer's disease(APP E693delta).
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	Establishment of the iPS cells from a patient with familial Alzheimer's disease(APP E693delta).
Basic objectives2	Others
Basic objectives -Others	Screening of small-molecule inhibitors preventing familial Alzheimer's disease(APP E693delta).
Trial characteristics_1	Exploratory
Trial characteristics_2	Pragmatic
Developmental phase

Assessment
Primary outcomes	Establishment of the iPS cells from a patient with familial Alzheimer's disease(APP E693delta).
Key secondary outcomes	Screening of small-molecule inhibitors preventing familial Alzheimer's disease(APP E693delta).

In outcomes field, the entry of just a few words such as "safety" or "efficiency" will not be accepted. Specify the name of outcome measures, including the time when you plan to measure. Usually, only one primary outcome is accepted. Write the other outcomes in "secondary outcomes" field.

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	Treatment
Type of intervention	Gene
Interventions/Control_1	Introduction of Yamanaka's factor into patient-derived somatic cells.
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

In interventions field, include the details of interventions, such as duration, amount, and frequency. If the intervention includes prescription or use of medical devices, duration is required.

Eligibility
Age-lower limit	50 years-old <=
Age-upper limit	60 years-old >=
Gender	Female
Key inclusion criteria	Patients havoring APP-E693delta
Key exclusion criteria	none
Target sample size	1

Research contact person
Name of lead principal investigator	Yoshihide Sunada
Organization	Kawasaki Medical School
Division name	Neurology
Address	577 Matsushima, Kurashiki, Okayama
TEL	0864621111
Email	ysunada@med.kawasaki-m.ac.jp

Public contact
Name of contact person	Yoshihide Sunada
Organization	Kawasaki Medical School
Division name	Neurology
Address	577 Matsushima, Kurashiki, Okayama
TEL	0864621111
Homepage URL
Email	ysunada@med.kawasaki-m.ac.jp

Sponsor
Institute	Kawasaki Medical School
Institute
Department

Sponsor means an organization that is responsible for plan, deployment and report of the research including funding management. It doesn't mean funding agency". Therefore, all clinical trial should have the one.

Funding Source
Organization	Kawasaki Medical School
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

Other related organizations
Co-sponsor
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

Other administrative information
Date of disclosure of the study information	2015 Year 06 Month 08 Day

Progress
Recruitment status	No longer recruiting
Date of protocol fixation	2015 Year 06 Month 08 Day
Anticipated trial start date	2015 Year 06 Month 08 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	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2015 Year 06 Month 08 Day
Last modified on	2016 Year 05 Month 19 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000020666