UMIN-CTR Clinical Trial

Public title

Phase I/II trial of AM01 (amnion-derived mesenchymal stem cells) for steroid-refractory acute graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation

Acronym

Phase I/II trial of AM01 (amnion-derived mesenchymal stem cells) for steroid-refractory acute GVHD

Scientific Title

Phase I/II trial of AM01 (amnion-derived mesenchymal stem cells) for steroid-refractory acute graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation

Scientific Title:Acronym

Phase I/II trial of AM01 (amnion-derived mesenchymal stem cells) for steroid-refractory acute GVHD

Region

Japan

Condition

Steroid-refractory acute graft-versus-host disease (GVHD)

Classification by specialty

Hematology and clinical oncology

Blood transfusion

Classification by malignancy

Others

Genomic information

NO

Narrative objectives1

To evaluate the safety and efficacy of intravenous administration of amnion-derived mesenchymal stem cells (AM01) on steroid-refractory acute GVHD after allogeneic hematopoietic stem cell transplantation

Basic objectives2

Safety,Efficacy

Basic objectives -Others

Trial characteristics_1

Exploratory

Trial characteristics_2

Explanatory

Developmental phase

Phase I,II

Primary outcomes

<Safety>
Adverse events related to infusion toxicity within 24 hours after administration of AM01

Key secondary outcomes

<Safety>
Adverse events within 52 weeks after first administration of AM01
<Efficacy>
1) Maintain a complete response (CR) more than 28 days
2) A CR or partial response (PR) at 4 weeks after first administration
3) The following points in 8, 12, 16, 20 and 24 weeks after first administration
1. Severity of acute GVHD
2. Onset/severity of chronic GVHD
3. Overall survival
4. Recurrence of primary disease
5. Outbreak of severe infection
6. Dose of steroid

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

Intravenous infusion of AM01 at the following days
Low dose group: 1.0x10^6 cells/kg (day 0, 7, 14 and 21)
High dose group: 4.0x10^6 cells/kg (day 0, 7, 14 and 21)
Safety in high dose group will be evaluated after a safety confirmation in at least 3 cases of low dose group.

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

1. Diagnosis of grades II to IV steroid-refractory acute GVHD
2. Aged 15 to 80
3. Written informed consent from patient, parent or guardian.

Key exclusion criteria

1. Has received more than one treatment for acute GVHD other than steroid
2. Presence of liver dysfunction other than GVHD (serum total bilirubin >2.0mg/dl or serum AST/ALT >3 times upper limit of normal)
3. Presence of kidney dysfunction (serum creatinine >2.0mg/dl)
4. Percutaneous oxygen saturation is less than 94% even under oxygen administration
5. Presence of an uncontrolled severe infection
6. Presence of severe hypersensitivity to bovine-derived constituents, human serum albumin and gentamicin
7. History of hypersensitivity to iodine or iodine-containing contrast agent
8. Previous participation in a study of any treatment with cell therapy product
9. Previous participation in a study of any investigational treatment within 12 weeks of agreement for this study
10. In pregnancy or breast-feeding
11. Considered as ineligible to this study

Target sample size

12

Name of lead principal investigator

1st name
Middle name
Last name	Kenichi Yamahara

Organization

Hyogo College of Medicine

Division name

Department of Transfusion Medicine and Cellular Therapy

Zip code

Address

1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501 Japan

TEL

0798-45-6398

Email

yamahara@hyo-med.ac.jp

Name of contact person

1st name
Middle name
Last name	Kenichi Yamahara

Organization

Hyogo College of Medicine

Division name

Department of Transfusion Medicine and Cellular Therapy

Zip code

Address

1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501 Japan

TEL

0798-45-6398

Homepage URL

Email

yamahara@hyo-med.ac.jp

Institute

Hyogo College of Medicine

Institute

Department

Personal name

Organization

Japan Agency for Medical Research and Development

Organization

Division

Category of Funding Organization

Government offices of other countries

Nationality of Funding Organization

Japan

Co-sponsor

Hokkaido University Hospital

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（兵庫県）、北海道大学病院（北海道）

Date of disclosure of the study information

2017

Year

11

Month

13

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

Preinitiation

Date of protocol fixation

2017

Year

08

Month

22

Day

Date of IRB

Anticipated trial start date

2017

Year

12

Month

01

Day

Last follow-up date

2019

Year

12

Month

31

Day

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2017

Year

11

Month

13

Day

Last modified on

2017

Year

11

Month

13

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034175