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

Phase 2

Not yet recruiting

• Conditions: Acute-graft-versus-host Disease

• Registration Number: NCT04883918
• Lead Sponsor: ASC Therapeutics

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-05-07
• Study First Submitted QC: 2021-05-07
• Study First Posted: 2021-05-12
• Status Verified: 2023-01
• Last Update Submitted: 2023-07-18
• Last Update Posted: 2023-07-20
• Study Start: 2023-12
• Primary Completion: 2026-01
• Study Completion: 2026-04

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 60

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Complete Response (CR) at Day 28 and Day 180	28 and 180 days post-infusion
Duration of response (DOR) at DAY 180	180 days post-infusion
Overall survival (OS) rate at DAY 180	180 days post-infusion