hAESCs Prevent Acute Graft-versus-host Disease After Hematopoietic Stem Cell Transplantation

Clinical Study of hAESCs on Prevention of Acute Graft-versus-host Disease After Hematopoietic Stem Cell Transplantation

This is a parallel controlled clinical study evaluating the safety and efficacy of hAECs in preventing aGVHD after HSCT.

Study Overview

• Status: Withdrawn
• Conditions: Acute Graft Versus Host Disease
• Intervention / Treatment: Biological: Human amniotic epithelial stem cells（hAESCs）; Biological: placebo (cell preservation solution)

Detailed Description

This study is a controlled trial, with 18 subjects enrolled in the experimental group or the control group. The study will consist of four phases, including screening phase, preparation phase, hAESCs treatment phase and observational follow-up period. The cell dose of the experimental group was 1x10^6 cells/kg and the control group is infused placebo (the composition was the same as hAESCs injection excipients, but did not contain hAESCs). The infusion of hAESCs/placebo at the day before HSCT and 7th days after HSCT.

• Study Type: Interventional
• Phase: Phase 1

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Standard risk GVHD patients with hematological malignancies older than 18 years;
• High risk GVHD patients with hematological malignancies: haplotype hematopoietic stem cell transplantation patients, donor is female or more than 30 years old;
• Well informed about this study and signed a consent form before the trial;
• Left ventricular ejection fraction (LVEF) ≧ 50%, no evidence of pericardial effusion;
• No evidence of lung infection by X-rays examination;
• Eastern cooperative oncology group (ECOG) performance status of 0 or 1, Hematopoietic cell transplantation - specific comorbidity index (HCT-CI) of 0, 1, 2;
• Normal liver and kidney function: Serum bilirubin≤35µmol/L, AST/ALT was less than 2 times the upper limit of normal value, and serum creatinine ≤130µmol/L

Exclusion Criteria:

• Reduce pretreatment dose or secondary transplantation;
• Participate other clinical trials within 2 months before this study;
• Female, 1) pregnant/nursing period, or 2) have a pregnancy plan during the study period, or 3) have fertility and cannot take effective contraception;
• History of severe allergic disease or is allergic to one or more drugs;
• Patients who are considered unsuitable for the study by the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: hAESCs; Intravenous infusion of hAESCs to 9 subjects at the day before HSCT and 7th days after HSCT. The dose is 1×10^6 cell/kg.	Biological: Human amniotic epithelial stem cells（hAESCs）; Human Amniotic Epithelial Cells Prevent Acute Graft-versus-host Disease After Hematopoietic Stem Cell Transplantation (hAECs-GVHD)
Placebo Comparator: placebo (cell preservation solution); Intravenous infusion of placebo (cell preservation solution with no hAESCs) to other 9 subjects at the day before HSCT and 7th days after HSCT.	Biological: placebo (cell preservation solution); Same dose placebo (cell preservation solution) injections as control group

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Follow up closely after intravenous hAESCs therapy to monitor Adverse Event/Serious Adverse Event(AE/SAE)	AE occurring throughout the study period will be evaluated using the CTCAE V5.0 standard. AE/SAE and AESI were evaluated after first hAESCs infusion by laboratory examination, measurement of vital signs, physical examination, and subjects' symptoms to detect new abnormalities and/or deterioration of previous conditions and evaluate the safety of the study. hAESCs intravenous infusion is followed up and monitored closely, mainly including vital signs, physical examination, ECOG, electrocardiogram, blood biochemistry, blood RT, urine RT, coagulation laboratory test, imaging examination, type, frequency and severity of AE.	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of acute graft-versus-host disease in patients	Occurrence and severity (grade) of Graft-versus-host disease after hAECs infusion	1 year
Immune reconstitution 6 months before and after hematopoietic stem cell transplantation	10 days(d) before hematopoietic stem cell transplantation and 1d, 7d±2, 14d±2, 21d±2, 28d±2, 3 month(m) ± 2 days , 6 m ± 2 days, testing expression of immune cells	6 months
Hematopoietic stem cell transplantation was performed 10 days before and 1 d, 7 d ±2, 14 d ±2, 21 d±2, 28 d ±2 after transplantation The amount of cytokines in plasma was measured	The amount of cytokines in plasma was measured: TGF-b	1 month
Incidence of CMV and EBV infection	Hematopoietic stem cell transplantation after 7 days ±2, 14 days ±2, 21 days±2, 28d±2, 2M±2 days, 3M±2 days, 6M±2 days, 12M±2 days;	1 year

Collaborators and Investigators

• Sponsor: Nanfang Hospital, Southern Medical University
• Collaborators: Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China
• Investigators: Study Chair: Xiaojun Huang, Nanfang Hospital, Southern Medical University

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): June 30, 2028

Study Registration Dates

• First Submitted: July 8, 2022
• First Submitted That Met QC Criteria: May 29, 2024
• First Posted (Actual): June 5, 2024

Study Record Updates

• Last Update Posted (Actual): April 16, 2026
• Last Update Submitted That Met QC Criteria: April 13, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: hAESCs
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: NFEC-2019-128

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No