Amimestrocel infusion to prevent severe mouth sores after allogeneic stem cell transplant

A Single-Arm, Single-Center Exploratory Study of Amimestrocel Injection for the Prevention of Gastrointestinal Mucositis Induced by Conditioning Regimens Containing TBI and/or Melphalan

This study will try a single IV infusion of Amimestrocel in adults receiving myeloablative allogeneic stem cell transplants with TBI or melphalan to see if it prevents severe mouth sores.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 22 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: The First Affiliated Hospital of Soochow University (Academic / other)
• Drugs / interventions: chemotherapy, radiation, cyclophosphamide
• Locations: 1 site (Suzhu, Jiangsu)
• Trial ID: NCT07400328 on ClinicalTrials.gov

What this trial studies

This is a single-arm, open-label Phase 2 study enrolling about 22 adults at one center to test a single fixed dose of Amimestrocel (6.0×10^7 cells IV) given 24–48 hours after the last cyclophosphamide dose and before stem cell infusion. Patients will be followed closely for oral mucositis severity and pain over the first 28 days, with additional safety monitoring up to 100 days. The trial focuses on those receiving total body irradiation and/or high‑dose melphalan as part of myeloablative conditioning. Outcomes include the rate of severe (Grade 3–4) oral mucositis, analgesic use, and treatment-related adverse events.

Who should consider this trial

Good fit: Adults aged 18–65 planned for myeloablative allogeneic HSCT with a conditioning regimen that includes TBI and/or melphalan, ECOG 0–1, and adequate organ function are the intended candidates.

Not a fit: Patients who are pregnant or lactating, have active uncontrolled infections, recent other malignancies, prior relevant cell therapies, or who are not receiving TBI/melphalan conditioning are unlikely to be eligible or to benefit from this intervention.

Why it matters

Potential benefit: If successful, the infusion could lower rates of severe oral mucositis, reduce pain and feeding difficulties, and decrease infection risk and hospital complications after transplant.

How similar studies have performed: Mesenchymal stem cell therapies have shown promise in preclinical models and early-phase clinical reports for reducing mucosal injury, but prophylactic umbilical-cord MSC infusion in this specific HSCT setting remains exploratory.

Eligibility criteria

Inclusion Criteria:

1. Aged 18 to 65 years.
2. Planned to undergo myeloablative allogeneic hematopoietic stem cell transplantation.
3. The conditioning regimen must contain total body irradiation (TBI) and/or melphalan.
4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
5. Adequate organ function defined as:Left ventricular ejection fraction (LVEF) ≥ 50%.Serum total bilirubin ≤ 1.5 × upper limit of normal (ULN).Serum creatinine ≤ 1.5 × ULN or creatinine clearance ≥ 60 mL/min.
6. Voluntarily signs the informed consent form.

Exclusion Criteria:

1. History of allergy to mesenchymal stem cells or any component of the Amimestrocel injection preparation.
2. Presence of active, uncontrolled bacterial, fungal, or viral infection.
3. History of other malignancies within the past 5 years, except for cured carcinoma in situ or basal cell skin cancer.
4. Pregnant or lactating women, or those planning pregnancy during the study period.
5. Previous receipt of any cell therapy product.
6. Severe psychiatric disorder or cognitive impairment that would limit the ability to provide informed consent or comply with study procedures.
7. Any condition that, in the investigator's judgment, makes the subject unsuitable for study participation.

Where this trial is running

Suzhu, Jiangsu

• First Affiliated Hospital of Soochow University — Suzhu, Jiangsu, China (Recruiting)

Study contacts

• Principal investigator: xiaojin Wu, Principal Investigator — The First Affiliated Hospital of Soochow University
• Study coordinator: depei Wu Study Coordinator

Conditions Oral Mucositis; Stomatitis; Gastrointestinal Mucositis; Hematopoietic Stem Cell Transplantation; Amimestrocel; Mesenchymal Stem Cells; Umbilical Cord; Mucositis Prevention