UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Amniotic membrane-derived mesenchymal stromal cells for the treatment of steroid-resistant acute GVHD
Title of the study (Brief title)	Amnion MSC for GVHD
Region	Japan

Condition
Condition	steroid resistant GVHD
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	YES

Objectives
Narrative objectives1	Allogeneic hematopoietic stem cell transplantation is the curative therapeutic option for hematopoietic malignancies and insufficiencies. Although there has been nearly 40 years of clinical experience in treatment of graft versus host disease (GVHD) as a major burden following allogeneic hematopoietic stem cell transplantation, it remains to be improved. Various immunosuppression agents have been developed for clinical use. The number of immunosuppression agents that can suppress the natural immune system, which is thought to be a trigger of GVHD onset, remains small. Recent studies have shown that mesenchymal stem cells (MSC) are present in various types of tissue, and a strong modulator of both acquired and natural immune systems. Thus, MSC may show promise for the treatment of excess immune responses. Amniotic membrane derived MSCs can be an alternative MSC source. Fetal accessory tissue derived cells are the youngest cells that can be obtained clinically. They are minimally affected by environment and life history, have active proliferative activity ready for expansion, and have stable quality. The steroid resistant GVHD, which was selected as the target for treatment this time, has a refractory nature, and the mortality rate of severe cases remains quite high. Thus, development of new therapeutic agents is desired.
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Pragmatic
Developmental phase	Phase I

Assessment
Primary outcomes	Safety of up to 52 weeks after
Key secondary outcomes	CR to be continued 4 weeks or more CR/PR at the time of 4weeks after the first dose

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	Medicine
Interventions/Control_1	infusion of Amnion derived MSC
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	15 years-old <=
Age-upper limit	80 years-old >
Gender	Male and Female
Key inclusion criteria	Steroid Resistant GVHD grade II or over Steroid Resistant GVHD grade I or over with high risk transplantation for GVHD e.g. GVHD in HLA Haploidentical Transplantation
Key exclusion criteria	1 patients with Liver damage other than GVHD (Total bililubin >2.0mg/dl or AST and/or ALT>200U/L 2 patients with Serum creatinine >2mg/dl 3 patients with allergy to bovine or porcine products 4 patienst with relapsed at MSC infusion 5 patients with uncontrolled infection 6 blood O2 saturation <94% even 3L/min Oxygen supply 7 Patients attending physician has determined ineligible.
Target sample size	5

Research contact person
Name of lead principal investigator	Hiroyasu Ogawa
Organization	Hyogo College of Medicine
Division name	Hematology
Address	1-1 mukogawa-cho, Nishinomiya, Hyogo,Japan
TEL	+81-798-45-6886
Email	ogawah@hyo-med.ac.jp

Public contact
Name of contact person	Toshihiro Soma
Organization	Hyogo college of Medicine
Division name	Hematology
Address	1-1 mukogawa-cho, Nishinomiya, Hyogo,Japan
TEL	+81-798-45-6886
Homepage URL
Email	somat@hyo-med.ac.jp

Sponsor
Institute	Hematology Division, Internal Medicine, Hyogo college of Medicine
Institute
Department

Funding Source
Organization	Japanese Government
Organization
Division
Category of Funding Organization
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	National cereberal and cardiovascukar Center Faundation for biomedical Research and innovation Hokkaido Graduate 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	兵庫医科大学病院 The Hospital of Hyogo College of Medicine

Other administrative information
Date of disclosure of the study information	2014 Year 09 Month 08 Day

Progress
Recruitment status	Terminated
Date of protocol fixation	2014 Year 08 Month 15 Day
Anticipated trial start date	2014 Year 09 Month 16 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	2014 Year 09 Month 02 Day
Last modified on	2019 Year 03 Month 07 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017416