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

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

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000029945

Enrollment

Registered

2017-11-13

Start date

2017-12-01

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Interventions

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

Eligibility

Sex/Gender

All

Inclusion criteria

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.

Exclusion criteria

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

Design outcomes

Primary

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

Secondary

Measure	Time frame
<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	—

Measure

Time frame

<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

—

Countries

Japan

Contacts

Public ContactKenichi Yamahara

Hyogo College of Medicine Department of Transfusion Medicine and Cellular Therapy

yamahara@hyo-med.ac.jp0798-45-6398

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026