A Trial to Evaluate the Safety and Preliminary Efficacy of iMesenchymal Stromal Cells(iMSC) in Subjects With SR-aGVHD

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

A study to evaluate the safety and preliminary efficacy of iMSC in subjects with SR-aGVHD

Study Overview

• Status: Recruiting
• Conditions: Steroid-refractory Acute Graft-versus-host Disease(SR-aGVHD)
• Intervention / Treatment: Biological: iMSC

Detailed Description

A single center, open label, dose escalation study to evaluate the safety,tolerability and preliminary efficacy of iMSC in subjects with SR-aGVHD

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Xiaoyu Zhu, phd; Phone Number: +86 15255456091; Email: xiaoyuz@ustc.edu.cn

Study Locations

• China
  • Anhui
    • Hefei, Anhui, China, 230036
      • Recruiting
      • Anhui Provincial Hospital
      • Contact: Zhu Xiaoyu, Ph.D; Phone Number: +86 15255456091; Email: xiaoyuz@ustc.edu.cn
      • Contact: Sun Guangyu; Phone Number: +86 13956970687; Email: sunguangyu_vip@foxmail.com

Participation Criteria

Eligibility Criteria

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

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: iMSC injection; Low dose injection once a week; Low dose injection twice a week; High dose injection once a week; High dose injection twice a week;	Biological: iMSC; Subjects will receive 4 or more times of iMSC injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dose-Limiting Toxicity(DLT)	Number of Dose-limiting toxicity in 28 days after injection	4 weeks after initial infusion
Adverse Event(AE),Serious Adverse Event(SAE),Treatment Emergent Adverse Event(TEAE)and Treatment Related Adverse Event(TRAE)	Number of treatment-related adverse events,treatment emergent adverse event,treatment related adverse event or serious adverse events as assessed by CTCAE v5.0	From the date of initial infusion to 180 days after initial infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response Rate(ORR)	Percentage of participants achieved Complete Response(CR) or Partial Response(PR)	28 days after initial infusion
Overall Survival(OS)	The time from initial infusion to death	100 days after initial infusion

Collaborators and Investigators

• Sponsor: Anhui Provincial Hospital
• Investigators: Principal Investigator: Xiaoyu Zhu, PH.D, Anhui Provincial Hospital

Study record dates

Study Major Dates

• Study Start (Actual): February 10, 2024
• Primary Completion (Estimated): March 2, 2027
• Study Completion (Estimated): March 2, 2027

Study Registration Dates

• First Submitted: March 13, 2024
• First Submitted That Met QC Criteria: March 13, 2024
• First Posted (Actual): March 20, 2024

Study Record Updates

• Last Update Posted (Actual): March 20, 2024
• Last Update Submitted That Met QC Criteria: March 13, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: iMSC-2001

Drug and device information, study documents

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