UMIN-CTR Clinical Trial

Public title

Amniotic membrane-derived mesenchymal stromal cells for the treatment of
steroid-resistant acute GVHD

Acronym

Amnion MSC for GVHD

Scientific Title

Amniotic membrane-derived mesenchymal stromal cells for the treatment of
steroid-resistant acute GVHD

Scientific Title:Acronym

Amnion MSC for GVHD

Region

Japan

Condition

steroid resistant GVHD

Classification by specialty

Hematology and clinical oncology

Classification by malignancy

Malignancy

Genomic information

YES

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

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

Study type

Interventional

Basic design

Single arm

Randomization

Non-randomized

Randomization unit

Blinding

Open -no one is blinded

Control

Uncontrolled

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

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

Name of lead principal investigator

1st name
Middle name
Last name	Hiroyasu Ogawa

Organization

Hyogo College of Medicine

Division name

Hematology

Zip code

Address

1-1 mukogawa-cho, Nishinomiya, Hyogo,Japan

TEL

+81-798-45-6886

Email

ogawah@hyo-med.ac.jp

Name of contact person

1st name
Middle name
Last name	Toshihiro Soma

Organization

Hyogo college of Medicine

Division name

Hematology

Zip code

Address

1-1 mukogawa-cho, Nishinomiya, Hyogo,Japan

TEL

+81-798-45-6886

Homepage URL

Email

somat@hyo-med.ac.jp

Institute

Hematology Division, Internal Medicine, Hyogo college of Medicine

Institute

Department

Personal name

Organization

Japanese Government

Organization

Division

Category of Funding Organization

Nationality of Funding Organization

Japan

Co-sponsor

National cereberal and cardiovascukar Center
Faundation for biomedical Research and innovation
Hokkaido Graduate school of medicine

Name of secondary funder(s)

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

兵庫医科大学病院
The Hospital of Hyogo College of Medicine

Date of disclosure of the study information

2014

Year

09

Month

08

Day

URL releasing protocol

Publication of results

Unpublished

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

Recruitment status

Terminated

Date of protocol fixation

2014

Year

08

Month

15

Day

Date of IRB

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

Other related information

Registered date

2014

Year

09

Month

02

Day

Last modified on

2019

Year

03

Month

07

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017416