ASC930 in Patients With Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD)

Study Overview
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Brief Summary
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Acute GVHD following allogeneic HCT is an immune-triggered process, leading to profound immune dysregulation and organ dysfunction. Despite pivotal advances, aGVHD is one of the leading causes of non-relapse mortality in patients undergoing HCT. Placenta-derived DSCs, isolated from the fetal membrane of maternal origin, are a type of stromal cells with well-characterized immunosuppressive properties. The current study is designed to assess the safety and efficacy of 4 intravenous (IV) doses of ASC930 DSC cells in aGVHD patients.

Official Title
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Evaluation of Safety and Efficacy of ASC930 in Patients With Steroid-Refractory Acute Graft Versus Host Disease

Conditions
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Acute-graft-versus-host Disease

Intervention / Treatment
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* Biological: ASC930


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participants ≥ 2 months of age Diagnosis of aGVHD grade II-IV following allogeneic HSCT according to standard criteria (Harris, 2016). Diagnosis of SR-aGVHD according to Mohty (2020) Meet one of the following criteria: Participants who are Ruxolitinib-refractory, according to Mohty (2020) Participants who are not eligible for SOC therapy Participants who agree to receive ASC930 as a second-line therapy Karnofsky/Lansky Performance Status of at least 30 at the time of study entry Minor participants must be capable of giving written assent as appropriate per the applicable age (per local regulatory requirements). Exclusion Criteria: Diagnosis of active Hepatic Sinusoidal Obstruction Syndrome (SOS) Presence of an active uncontrolled infection Active treatment for a hyprecoagulation disorder Evidence of diffuse alveolar hemorrhage or other active pulmonary disease Evidence of encephalopathy as defined by a change in mental status since the onset of aGVHD Evidence of relapse of malignancy Receival of agents other than steroids for primary treatment of aGVHD Severe allergic history to cell-based products

Ages Eligible for Study
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Minimum Age: 2 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ASC930<br>Experimental Arm | Biological: ASC930<br>* 4 intravenous doses of ASC930<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Response Rate (ORR) at Day 28 |  | 28 days post-infusion | 
| Number of adverse events, and serious AEs |  | 30 days post-infusion | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Duration of response (DOR) at DAY 180 |  | 180 days post-infusion | 
| Overall survival (OS) rate at DAY 180 |  | 180 days post-infusion | 
| Complete Response (CR) at Day 28 and Day 180 |  | 28 and 180 days post-infusion |