UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	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
Title of the study (Brief title)	Phase I/II trial of AM01 (amnion-derived mesenchymal stem cells) for steroid-refractory acute GVHD
Region	Japan

Condition
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

Objectives
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

Assessment
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

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	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

Eligibility
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

Research contact person
Name of lead principal investigator	Kenichi Yamahara
Organization	Hyogo College of Medicine
Division name	Department of Transfusion Medicine and Cellular Therapy
Address	1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501 Japan
TEL	0798-45-6398
Email	yamahara@hyo-med.ac.jp

Public contact
Name of contact person	Kenichi Yamahara
Organization	Hyogo College of Medicine
Division name	Department of Transfusion Medicine and Cellular Therapy
Address	1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501 Japan
TEL	0798-45-6398
Homepage URL
Email	yamahara@hyo-med.ac.jp

Sponsor
Institute	Hyogo College of Medicine
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Government offices of other countries
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	Hokkaido University Hospital
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（兵庫県）、北海道大学病院（北海道）

Other administrative information
Date of disclosure of the study information	2017 Year 11 Month 13 Day

Progress
Recruitment status	Preinitiation
Date of protocol fixation	2017 Year 08 Month 22 Day
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

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2017 Year 11 Month 13 Day
Last modified on	2017 Year 11 Month 13 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034175