UMIN-CTR Clinical Trial

Basic information
Public title	Clinical research of bone marrow-derived mesenchymal stem cell transplantation for the patients with epidermolysis bullosa.
Acronym	Clinical research of bone marrow-derived mesenchymal stem cell transplantation for the patients with epidermolysis bullosa.
Scientific Title	Clinical research of bone marrow-derived mesenchymal stem cell transplantation for the patients with epidermolysis bullosa.
Scientific Title:Acronym	Clinical research of bone marrow-derived mesenchymal stem cell transplantation for the patients with epidermolysis bullosa.
Region	Japan

Condition
Condition	Epidermolysis bullosa (junctional, dystrophic)
Classification by specialty	Hematology and clinical oncology Dermatology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Evaluation of safety and efficacy of family donor-derived bone marrow mesenchymal stem cell transplantation to the patients with junctional and dystrophic epidermolysis bullosa.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Safety evaluation (Adverse events)
Key secondary outcomes	Efficacy evaluation (macroscopic and histological examination of the skin lesions)

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	Maneuver
Interventions/Control_1	Subcutaneous transplantation of bone marrow mesenchymal stem 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

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	60 years-old >
Gender	Male and Female
Key inclusion criteria	1) Patients diagnosed with epidermolysis bullosa (junctional or dystrophic type). 2) Patients aged 20-60 years old. 3) Patients with intractable skin ulcer which present for more than 6 weeks. 4) Patients who have opposite-sex parent or sibling with donor agreement. 5) Patients who agreed with written concent
Key exclusion criteria	1) Patients with severe psychotic disorders 2) Patients with alcoholic addiction 3) Patients with disturbance of consciousness 4) Patients with history of malignancy within the last 5 years 5) Patients with pregnancy 6) Patients with diabetes Other patients who are evaluated not to be suitable for this research
Target sample size	6

Research contact person
Name of lead principal investigator	1st name Middle name Last name Katsuto Tamai
Organization	Osaka University Graduate School of Medicine
Division name	Department of Stem Cell Therapy Science
Zip code
Address	2-2, Yamadaoka, Suita-shi, Osaka 565-0871, Japan
TEL	06-6879-3902
Email

Public contact
Name of contact person	1st name Middle name Last name
Organization	Osaka University Hospital
Division name	Medical Center for Translational Research
Zip code
Address	2-15, Yamadaoka, Suita-shi, Osaka 565-0871, Japan
TEL
Homepage URL
Email

Sponsor
Institute	Osaka University Graduate School of Medicine
Institute
Department

Funding Source
Organization	None
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization
Address
Tel
Email

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	大阪大学医学部附属病院（大阪府）/Osaka University Hospital (Osaka)

Other administrative information
Date of disclosure of the study information	2011 Year 11 Month 14 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	Completed
Date of protocol fixation	2011 Year 10 Month 14 Day
Date of IRB	2013 Year 04 Month 03 Day
Anticipated trial start date	2013 Year 07 Month 26 Day
Last follow-up date	2015 Year 05 Month 01 Day
Date of closure to data entry	2015 Year 05 Month 01 Day
Date trial data considered complete	2017 Year 02 Month 02 Day
Date analysis concluded	2017 Year 02 Month 02 Day

Other
Other related information

Management information
Registered date	2011 Year 11 Month 14 Day
Last modified on	2019 Year 11 Month 20 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000007940