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GVHD,AcutePhase 1aGVHD，hAESCsNCT06164288

Safety and Efficacy Study of hAESCs Therapy for aGVHD

A Phase 1 study testing Human Amniotic Epithelial Stem Cell Injection in people with GVHD,Acute and aGVHD, run by Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Allogeneic hematopoietic stem cell transplantation (allo-HSCT), a process in which hematopoietic stem cells from a donor are injected into the recipient's body, are the treatment of choice for many hematologic malignancies. Graft-versus-host disease (GVHD) is a common and important complication after allogeneic HSCT. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
GVHD,Acute, aGVHD
What is being tested?
Human Amniotic Epithelial Stem Cell Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteChina
Study dates
19 Feb 2024to 22 Mar 2025
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • A subject will be eligible for inclusion in this study only if ALL of the following criteria are met:
  • The subject or his/her legal guardian has agreed to participate in this clinical trial and has signed the Informed Consent Form (ICF), indicating that the subject understands the purpose and procedures of this clinical trial and is willing to participate in the study.
  • Aged ≥ 18 years; male or female.
  • First-episode refractory acute graft-versus-host disease (aGVHD) with an overall clinical grade of III to IV as determined by the Mount Sinai Acute GVHD International Consortium (MAGIC) grading criteria. If other etiologies can be excluded by clinical diagnosis, biopsy confirmation is not required. Subjects must also meet ALL of the following conditions:
  • Patients with hematologic malignancies who have received hematopoietic stem cell transplantation from any donor type and any stem cell source (bone marrow, peripheral blood, or umbilical cord blood), with myeloablative conditioning prior to transplantation (patients with acute lymphoblastic leukemia or chronic myeloid leukemia will be given priority for enrollment);
  • aGVHD refractory to second-line therapy: subjects with steroid-resistant aGVHD who have subsequently received at least one second-line agent (e.g., anti-CD25 monoclonal antibody, ruxolitinib), with progression or no improvement of aGVHD symptoms.
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  • Note: Steroid-resistant aGVHD is defined as disease progression (PD) assessed within 3-5 days of treatment with intravenous methylprednisolone at a starting dose of 1-2 mg/kg/day or an equivalent corticosteroid dose (worsening of aGVHD in at least one target organ by at least one grade, with or without improvement of aGVHD in other organs), or no response (NR) assessed within 5-7 days of treatment (no improvement and no worsening of aGVHD severity in any target organ), or failure to achieve complete response (CR) by Day 14 (all involved organs achieving grade 0).
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
  • Adequate function of major organs, as defined by the following criteria:
  • Cardiac function: left ventricular ejection fraction (LVEF) ≥ 50%;
  • Hepatic function: aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≤ 3 × upper limit of normal (ULN); if the elevation of AST or ALT is related to aGVHD, AST or ALT ≤ 5 × ULN;
  • Renal function: serum creatinine (Cr) ≤ 1.5 × ULN, or creatinine clearance (Ccr) ≥ 30 mL/min (calculated by the Cockcroft-Gault formula);
  • Peripheral oxygen saturation (SpO₂) ≥ 92%.
  • If the subject or the subject's spouse is a woman of childbearing potential, the subject agrees to use effective contraception during the trial period (non-pharmacological contraceptive measures will be used by the subject).

Who cannot join

  • A subject will be excluded from this study if he/she meets ANY of the following criteria:
  • Receiving treatment in another therapeutic clinical trial within 4 weeks prior to infusion of the investigational treatment.
  • Subjects with a history of other solid organ transplantation.
  • Subjects who have received mesenchymal stem cell (MSC) or human amniotic epithelial stem cell (hAESC) therapy, or any other stem cell therapy (other than hematopoietic stem cells), within 6 months prior to infusion of the investigational treatment.
  • Receipt of a live or attenuated virus vaccine within 4 weeks prior to infusion of the investigational treatment.
  • Positive for hepatitis B surface antigen (HBsAg) and/or positive for hepatitis B core antibody (HBcAb) with HBV DNA above the upper limit of normal (if HBsAg, HBeAg, and HBcAb are all positive, HBV DNA testing is not required and the subject will be directly excluded); positive for hepatitis C virus (HCV) antibody; positive for human immunodeficiency virus (HIV) antibody; positive for syphilis antibody.
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  • Note: Laboratory results obtained within 1 month prior to enrollment are acceptable for the above virologic examinations.
  • Known hypersensitivity to any component of the treatment used in this study (including DMSO and BAT).
  • Known uncontrolled pulmonary diseases, including but not limited to pulmonary fibrosis, interstitial lung disease, acute exacerbation of chronic obstructive pulmonary disease (COPD), and active pulmonary infection.
  • Severe hepatic veno-occlusive disease (VOD).
  • Pregnant or lactating women.
  • The investigator determines that the subject is unlikely to complete all protocol-required visits or procedures (including the follow-up period), or has insufficient compliance for participation in this study.
  • Any underlying or current medical or psychiatric condition that, in the investigator's opinion, would interfere with the treatment and evaluation of the subject, including but not limited to: poorly controlled hypertension (systolic blood pressure > 160 mmHg and/or diastolic blood pressure > 100 mmHg), uncontrolled infection, pulmonary hypertension, clinically significant (e.g., active) cardiovascular or cerebrovascular disease, such as cerebrovascular accident (within 6 months prior to signing the ICF), myocardial infarction (within 6 months prior to signing the ICF), unstable angina, congestive heart failure of New York Heart Association (NYHA) class III or IV, or severe arrhythmia that cannot be controlled by medication or that may potentially affect the investigational treatment; clinically significant abnormalities on electrocardiogram (ECG), or QTcF ≥ 480 ms.

Trial locations and contacts

1 site in 1 country.

China1 site

  • Peking University People's Hospital

    Beijing, Beijing Municipality, 100044

Other trials in China

About this study

Official title: A Phase I Study to Evaluate the Safety and Efficacy of Human Amniotic Epithelial Stem Cell（hAESCs）Injection Treatment for the Patients With Refractory aGVHD After Allogeneic Hematopoietic Cell Transplantation (HCT)

Allogeneic hematopoietic stem cell transplantation (allo-HSCT), a process in which hematopoietic stem cells from a donor are injected into the recipient's body, are the treatment of choice for many hematologic malignancies. Graft-versus-host disease (GVHD) is a common and important complication after allogeneic HSCT. GVHD is a major obstacle to the success of HSCT treatment and a leading cause of death after HSCT treatment.

Hormone therapy is currently the standard treatment for aGVHD, i.e., the first-line treatment. However, 40%~50% of aGVHD cannot be controlled by hormone therapy, and additional therapeutic intervention is required. According to the National Comprehensive Cancer Network (NCCN) clinical practice guidelines for hematopoietic stem cell transplantation - pre-transplant recipient evaluation and management of GVHD (2021.V3), the recommended drugs for second-line treatment of grade II~IV aGVHD include: alemtuzumab, α-1 antitrypsin, antithymocyte globulin, basiliximab, calcineurin inhibitors, etanercept, extracorporeal photopheresis replacement therapy, infliximab, mammalian rapamycin target protein inhibitors, mycophenolate mofetil, Pentostatin, ruxolitinib, tocilizumab. Second-line treatment is based on retrospective data and there is no standard salvage therapy, which is reflected in the inconsistent treatment strategy for aGVHD across transplant centers.

Read more

One of the biological functions of hAESCs in amniotic membranes in vivo is to exert reproductive immunomodulatory effects and protect the fetus from rejection by the maternal immune system, so hAESCs have natural immunomodulatory functions. hAESCs have significant inhibitory effects on T cells, antigen-presenting cells (APCs), natural killer (NK) cells, macrophages, neutrophils, B cells and other immune cells associated with organ damage during the pathogenesis of aGVHD, and hAESCs have great potential in the treatment of aGVHD. Therefore, the sponsor developed hAESCs injections intended for the treatment of aGVHD.

The experimental drug in this study is hAESCs injection, which is intended to be used for the treatment of adult patients with grade III.~IV. refractory aGVHD after hematopoietic stem cell transplantation, and to explore the safety and preliminary efficacy of its treatment.

Conditions
GVHD,Acute, aGVHD
Being tested
Human Amniotic Epithelial Stem Cell Injection
Keywords
aGVHD, hAESCs, hAECs, stem cells

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Human Amniotic Epithelial Stem Cell InjectionDrug

    The dose escalation is carried out according to the "3+3" ascending principle, and a total of 3 dose levels are set:

    1. Dose Level 1: 1×10^6 cells/kg;
    2. Dose Level 2: 2×10^6 cells/kg;
    3. Dose Level 3: 3×10^6 cells/kg; Note: In this study, if the maximum tolerated dose (MTD) is not explored at dose level 3 (3×10^6 cells/kg), it will be discussed by the Safety Review Committee (SRC) to decide whether to proceed with further dose escalation or expansion to 6 patients.
Study groups in NCT06164288
Study groupRoleWhat they receive
hAESCs injectionExperimentalIntravenous infusion of human amniotic epithelial stem cell injection, with one infusion frequency.
Receives: Drug: Human Amniotic Epithelial Stem Cell Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Subjects with DLT at a Given Dose Level

    Number of Subjects with DLT at a Given Dose Level（Low/Medium/High). A dose limiting toxicity (DLT) is defined as the hAESCs-related adverse event (AE) greater level than 3（including grade 3) that occurs during the DLT period, graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE), Version 5.0.

    Measured: D0~D28

  2. Maximum Tolerated Dose (MTD)

    The Maximum Tolerated Dose (MTD) of hAESCs. The MTD is defined as the previous lower dose in the dose group in which ≥ 2/6 subjects developed DLT during the DLT observation period after infusion of hAESCs injection.

    Measured: D0~D28

  3. Incidence of adverse events

    Incidence of adverse events (AEs), serious adverse events (SAEs), adverse events of special interest (AESIs).

    Measured: D0~D100

Secondary outcomes (6)

  1. Overall response rate (ORR)

    Overall response rate (ORR) after hAESCs injection infusion. ORR rate: the proportion of the total number of subjects who obtained CR, VGPR or PR after receiving study treatment to the total number of evaluable subjects.

    Measured: D0~D100

  2. Complete response (CR) rate

    Complete response (CR) rate after hAESCs injection infusion. CR rate: the proportion of the total number of subjects who received a CR after receiving treatment to the total number of evaluable subjects.

    Measured: D0~D100

  3. Very good partial response (VGPR) rate

    Very good partial response (VGPR) rate after hAESCs injection. VGPR rate: the proportion of the total number of subjects who received VGPR after receiving treatment to the total number of evaluable subjects.

    Measured: D0~D100

  4. Partial response (PR) rate

    Partial response (PR) rate after hAESCs injection. PR rate: the proportion of the total number of subjects who received PR after receiving treatment to the total number of evaluable subjects.

    Measured: D0~D100

  5. Overall survival (OS)

    Overall survival (OS) after infusion of hAESCs injection. OS: It refers to the time which begins at the start of treatment and up to the time of death.

    Measured: The time of death caused by any reason from the date of receiving hAESCs injection

  6. Overall survival (OS) rate

    Overall survival (OS) rate after infusion of hAESCs injection. OS rate: It refers to the proportion of total subjects who survived on the 28th and 100th day to the total subjects who received treatment.

    Measured: D28、D100

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Dec 2023
  2. Study start19 Feb 2024
  3. Primary completion22 Mar 2025
  4. Study completion22 Mar 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06164288
Sponsor's protocol ID
HAESCS-GVHD-101
Acronym
aGVHD，hAESCs
Status last verified
Oct 2026
First submitted
21 Nov 2023
Last updated
15 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Human Amniotic Epithelial Stem Cell Injection in people with GVHD,Acute and aGVHD, run by Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Subjects with DLT at a Given Dose Level (time frame: D0~D28). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06164288.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2026.

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