Evaluating iMesenchymal Stromal Cells for Steroid-Refractory Acute Graft-versus-Host Disease

A Trial to Evaluate the Safety and Preliminary Efficacy of Human Induced Pluripotent Stem Cell Derived Mesenchymal Stromal Cells in Subjects With Steroid-refractory Acute Graft-Versus-Host Disease

This study is testing if a new type of stem cell treatment can help people with acute graft-versus-host disease who haven't responded to steroids.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 4 Years to 70 Years
• Sex: All
• Sponsor: Anhui Provincial Hospital (Government)
• Locations: 1 site (Hefei, Anhui)
• Trial ID: NCT06321198 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and preliminary efficacy of iMesenchymal Stromal Cells (iMSC) in patients suffering from steroid-refractory acute graft-versus-host disease (SR-aGVHD). It is a single-center, open-label, dose escalation study designed to assess how well iMSC can be tolerated and its potential effectiveness in treating this condition. Participants will be monitored for safety and response to treatment as part of the evaluation process.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals aged 4 to 70 years with a diagnosis of steroid-refractory acute graft-versus-host disease.

Not a fit: Patients with severe allergies to blood products, severe hepatic or lung diseases, or those with a very limited life expectancy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with SR-aGVHD who do not respond to standard steroid treatments.

How similar studies have performed: While the use of mesenchymal stem cells in various conditions has shown promise, this specific approach for SR-aGVHD is relatively novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

1.Subjects who understand and voluntarily sign the Informed Consent Form(ICF); 2.4-70 years; 3.Subjects with SR-aGVHD; 4.ECOG≤2; 5.Subjects with II to IV grades of steroid hormone resistance;

Exclusion Criteria:

1. Accepted systemic or local treatment of mesenchymal stem cells;
2. Have severe allergy to blood products or have allergy history of heterologous protein;
3. Expected survival period within 3 months;
4. Alanine transaminase(ALT)or Aspartate aminotransferase(AST)\>2\*upper limit of normal(ULN);Creatinine clearance rate≤30ml/min or Blood Urea Nitrogen(BUN)\>2\*upper limit of normal(ULN), International Normalized Ratio (INR)\>1.5\*upper limit of normal(ULN);
5. Have severe hepatic veno-occlusive disease(HVOD);
6. Have severe lung disease like severe lung infection;
7. Have history of severe acute myocardial infarction or have uncontrolled angina pectoris,arrhythmia and severe heart failure;
8. Proved having resistant hypertension within 6 months before enrollment;
9. Have active thrombus;
10. Have untreated or uncertain active solid tumors within 5 years;
11. Have alcohol or drug addiction or with a clear history of mental disorders or with a history of drug abuse or drug use of psychotropic substances;
12. Human immunodeficiency virus(HIV)antibody positive, treponema pallidum (TP) antibody positive;
13. Have active hepatitis B or hepatitis C;
14. Have gastrointestinal symptoms which not caused by GVHD
15. Pregnant or lactating female subjects, or subjects who are unable to comply with contraceptives from the study period to 6 months after the end of this study;
16. Subjects who have participated in other clinical trials and have used other study products within 12 weeks before screening;
17. Not suitable for this clinical trial for other reasons.

Where this trial is running

Hefei, Anhui

• Anhui Provincial Hospital — Hefei, Anhui, China (Recruiting)

Study contacts

• Principal investigator: Xiaoyu Zhu, PH.D — Anhui Provincial Hospital
• Study coordinator: Xiaoyu Zhu, phd

Conditions Steroid-refractory Acute Graft-versus-host Disease